Perspectives - HAE International

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Perspectives - HAE International
MARCH 2021

                                               HAEi MAGAZINE · ISSUE 1/2021

                                               93 Member countries

                                NEW APP! HAE TRACKR
                                BY PATIENTS FOR PATIENTS                      18

                                LAUNCH OF THE THIRD HAEi
                                – NOW IN LATIN AMERICA
Perspectives - HAE International
          Global Perspectives
             Issue 1/2021
              March 2021

             Cover photo
HAEi Youngsters visiting the National Civil
 Rights Museum during the 2019 Global
HAE youth advocacy workshop in Atlanta,

            Magazine staff
   Henrik Balle Boysen, Executive Vice        DEAR HAE INTERNATIONAL FRIENDS,
   President & Chief Operating Officer
Rikke Sørensen, Communication Design          Welcome to the first 2021 edition of Global            Importantly, TrackR strictly protects patient
        and Graphics Manager
                                              Perspectives. The extraordinary spirit and “can do”    privacy, is EU-GDPR compliant, and enables data
Steen Bjerre, Communications Manager          attitude of our global community remains evident       sharing with a physician. We will announce the
                                              as HAEi member organizations continue to               TrackR rollout on our social media platforms
  If you would like to subscribe to our       make remarkable progress, despite the ongoing          within the next several weeks.
   magazine, please send an email to          challenges posed by COVID-19 restrictions. or register directly        Also, as you will read in the pages that follow,       Survey data we have gathered over the years
                                              our Regional Patient Advocates (RPAs) continue         reveals a critical need for significant improvement
                                              to implement HAEi’s vision of a decentralized          in emergency room care for people with HAE.
                                              yet globally connected network by establishing         Education is clearly the key to upgrading
                                              Regional Advisory Groups and Regional Medical          emergency care, and that is why HAEi has
HAEi 2021 activities are supported by
                                              Advisory Panels. These initiatives are focused on      prepared a poster with important information
                                              encouraging information sharing, collaboration,        that a doctor must know to successfully treat
                                              and dissemination of effective and practical           an HAE emergency. The posters can be brought
                                              solutions to help member organizations expedite        to local emergency rooms where they can be
                                              improving diagnosis and obtaining access to            displayed in a prominent location. See more on
                                              modern HAE medicines.                                  how to obtain posters for your country in this
                                              2021 marks the 10th Anniversary of hae day :-)
                                              and HAEi has developed a fun and creative              Finally, we are pleased to announce the 2021 HAEi
                                              campaign for celebrating this important                Virtual Regional Workshop for South America,
                                              milestone. Consistent with the theme of ‘Let’s         Mexico, Central America and the Caribbean.
                                              Take the Next Step,’ HAEi will ask our friends from    The workshop has four tracks that include the
                                              all over the globe to take part in and record (on      international perspective, informative lectures
                                     time spent performing physical or          by expert HAE physicians, youngster programs,
                                              well-being activities. All recorded activities will    and member organizations’ updates. All videos
                                              be converted into steps to support a virtual walk      will be offered in the presenter’s local language,
                                              across the HAEi regions to visit with and learn        with subtitles in Spanish, Portuguese, and English.
                                              about our individual member organizations.             We look forward to a very successful event in this
                                                                                                     important part of the HAEi global network.
                                              Our contacts with member organizations revealed
                                              the need for a secure, user-friendly app that can      Wishing all in our global community the very best
                                              be used to track attacks, treatments, emergency        and look forward to meeting in person again,
                                              room visits and document how HAE affects               hopefully in the very near future.
        HAEi is registered as a               quality of life. We are excited to announce the
     non-profit organization in USA           forthcoming launch of TrackR, an app for patients,     Warmest regards and please stay safe,
                                              designed by patients, that fulfills the requirements
  HAEi is a global non-profit umbrella
organization dedicated to working with        requested by our member organizations.
  a network of national HAE member
organizations to raise awareness of HAE                                                              Anthony J. Castaldo
                                                                                                     President & CEO, HAEi

                                                                                                                             GLOBAL PERSPECTIVES · MARCH 2021 ⎮ 03
Perspectives - HAE International
IN THIS ISSUE                                                                                                                                                                                  IN THIS ISSUE


                                            06                                                       24
                                                                                                     14                                                                                  18


           23                                                                                        18

          06	News from the HAEi regional patient       20	Regional advisory groups define their   28	ACARE in action                             40	Presenting powerful free resources:
              advocates                                     top 5 challenges                                                                            HAEi Connect and website hosting
                                                                                                    30	Expanding global network of ACARE
          14	10 years anniversary of global HAE        22 H
                                                            AEi establishes Regional Medical           centers                                     42	News from member organizations
              awareness day                                Advisory Panels                                                                              around the globe
                                                                                                    32	Patient story: Once I was diagnosed
          16 	Novel prophylaxis therapies can lead     23 L
                                                            aunch of third virtual regional            with HAE, I felt very relieved that I had   54 Medical papers
               to significant improvements in quality      workshop                                     answers – and treatment
               of life                                                                                                                              58 Clinical trials
                                                        24	Meeting old and new friends via HAEi    37	HAEi staff news
          18 	HAEi launches new app – by patients,        Youngsters’ online meetups                                                               61 News from the industry
               for patients                                                                         38	The HAEi media
                                                        26	Emergency room posters help                                                             72 HAEi around the world
                                                            raise awareness
04 ⎮ GLOBAL PERSPECTIVES · MARCH 2021                                                                                                                                    GLOBAL PERSPECTIVES · MARCH 2021 ⎮ 05
Perspectives - HAE International

          NEWS FROM THE

                                                                                                                                                MICHAL RUTKOWSKI
                                                                                                                                                CENTRAL EASTERN EUROPE,
          What a busy start to the year! There has been a lot of hard work going on behind the scenes                                           BENELUX AND MIDDLE EAST
          with the Regional Patient Advocates (RPAs) and HAEi staff, and we look forward to bringing
          new and exciting projects and initiatives to our member organizations.

          Last year HAEi held two virtual regional workshops, and now we launch a third workshop , this
          time for Latin America. The 2021 HAEi Virtual Regional Workshop South America & Mexico,
          Central America & Caribbean features presentations from local expert physicians, member
          organizations, HAEi, including the two RPAs for South America and Mexico, and Central America    The recent months have been busy with plenty of               In a joint effort with HAE Belarus, the first modern
          and the Caribbean. We are planning a fourth virtual workshop to take place later in the year,    advocacy activities for patients in Central Eastern           therapy (plasma-derived C1-INH) for HAE patients has
          focusing on the Middle East, North Africa, and Sub Sahara Africa. Stay tuned for more details.   Europe, Benelux and the Middle East. This period was          been registered in Belarus. Furthermore, the president
                                                                                                           full of virtual meetings with member organizations,           of HAE Belarus, Irina Ausianik, presented the patients’
          The RPAs have also been extending invitations and information to the Regional Medical            both individual and group, during which we discussed          perspective during a virtual conference organized by
          Advisory Panel’s nominated by our member organizations. We are planning the first meeting        important projects delivered and implemented by               Takeda in February 2021. The conference was dedicated
          to take place very soon.                                                                         HAEi. It is truly amazing to be part of the activities that   to health care professionals from Belarus, Kazakhstan
                                                                                                           involve patient organizations, patients, caregivers and       and Ukraine.
          The hae day :-) campaign ‘Let’s Take the Next Steps’ has been a significant focus for the        physicians from so many countries.
          RPAs. They have been working with their member organizations to collect country specific                                                                       In collaboration, HAE Kazakhstan and Takeda have
          information that will be showcased on the website.                                    An important element of the HAEi strategy, related to         set up the diagnosing program for HAE patients in
                                                                                                           the decentralization of our activities, is the formation      the country. Also, the process of C1-INH registration
          Exciting, busy, and innovative times ahead.                                                      of the Regional Medical Advisory Panels in each of            is about to take place, and HAE Kazakhstan hopes it
                                                                                                           the regions supported and covered by the global               will soon have the medication registered. Also, HAE
          Fiona Wardman                                                                                    organization. I am delighted to inform you that thanks        Kazakhstan has organized a successful virtual meeting
          Chief Regional Patient Advocate                                                                  to the intensified activities, it was possible to invite      for HAE patients.
                                                                                                           and confirm the participation of many expert HAE
                                                                                                           specialists in the Regional Medical Advisory Panel for        HAE Lithuania is the first member organization from
                                                                                                           Central Eastern Europe, Benelux and Middle East. We           Central and Eastern Europe to implement the HAEi
                                                                                                           expect to have inaugural meetings very soon.                  Emergency Room Poster. The poster was translated into
                                                                                                                                                                         Lithuanian, and HAEi covered the costs of production.
                                                                                                           In addition to various virtual meetings, I am constantly
                                                                                                           working on implementing HAEi resources and                    HAE Egypt has taken steps to support the registration
                                                                                                           projects so that each patient has access to the latest        of plasma-derived C1-INH, the first HAE therapy in
                                                                                                           information regarding HAE. Also, we have taken the            the country for people living with HAE. Supported
                                                                                                           first steps related to the preparations of a website          by HAEi, the organization has sent an official letter
                                                                                                           dedicated to the Middle East and North Africa regions         to the Egyptian Ministry of Health, H.E. Dr. Hala
                                                                                                           and the organization of the 2021 HAEi Virtual Regional        Zayed, emphasizing how important access to modern
                                                                                                           Workshops for the Middle East, North and Sub Sahara           treatment options is for HAE patients.
                                                                                                           Africa. In these two projects, I enjoy working with my
                                                                                                           colleagues Maria Ferron and Patricia Karani.                  And finally, some excellent news from HAE United Arab
                                                                                                                                                                         Emirates: Thanks to the involvement of the member
                                                                                                           I am happy to announce that HAE Jordan has joined             organization, local health care professionals, the
                                                                                                           HAEi as member number 93. Mr. Bassam Mohammad                 hospital, CSL Behring and myself, it was after months of
                                                                                                           Alelimat represents the organization. Just after              shortages possible to deliver plasma-derived C1-INH
                                                                                                           entering the global organization, HAE Jordan launched         to the Mediclinic City Hospital in Dubai.
                                                                                                           its website hosted by HAEi.

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Perspectives - HAE International
NEWS FROM THE HAEi REGIONAL PATIENT ADVOCATES                                                                                                                                                              NEWS FROM THE HAEi REGIONAL PATIENT ADVOCATES

                                              MARIA FERRON                                                                                                             FERNANDA DE OLIVEIRA MARTINS
                                              MEDITERRANEAN, NORTH AFRICA AND BRITISH ISLES                                                                            SOUTH AMERICA AND MEXICO

                                                                                                                                  Over the past few months, I have been working with            Most recently, I have been organizing and co-ordinating
          Initially, I am happy to inform you that the HAEi           30 hospitals with HAE modern treatment. Furthermore,        the member organizations in my region and informing           presentations and recordings for the first virtual
          Emergency Room poster is now translated into Spanish,       the annual meeting included a presentation on self-         them about new resources like the Emergency Room              regional meeting in Latin America. I have also been
          French and Arabic.                                          administration. Here one of the nurses dealing with         Poster and the new HAE TrackR app.                            creating the scripts and timed transcripts for the videos
                                                                      HAE gave feedback on self-administration using a                                                                          as they will be available in Spanish, Portuguese and
          On 5 December 2020, HAE Portugal (ADAH) held its            video to show how to do it.                                 There has been a lot of work and communications               English. Each video needs to be checked for errors, and
          annual meeting, this time in a virtual format. Around                                                                   on collecting information for the hae day :-) 10th            this process takes many hours.
          45 participants took part, and Raquel Oliveira, the         HAE France is in the initial phase of having its website    anniversary campaign.
          President of HAE Brazil (Abranghe) and I were invited       hosted under the HAEi umbrella.                                                                                           Since the most recent issue of Global Perspectives, there
          as speakers. After a word of welcome from President                                                                     HAEi Connect is currently being rolled out in Colombia        have been many Instagram posts uploaded on the
          Ines Pessoa, there was a scientific update with Professor   HAE Algeria has been quite active regarding visiting        and Peru.                                                     South American and Mexico Instagram page.
          Branco as moderator. Here four Portuguese doctors           the Ministry of Health to create HAE awareness and
          talked about updates on new HAE forms, diagnosis and        try to improve HAE patients’ lives. The local authorities
          clinical manifestations, long and short terms therapies,    are now looking into the possibility of registering HAE
          as well as new therapies and questionnaire results          modern treatment in the country. In February, HAE
          on Quality of Life. Then followed a presentation from       Algeria was on tv and radio and in newspapers to talk
          Raquel Oliveira on the current Brazilian situation while    about HAE; this included some interviews with HAE
          I, among other things, presented some of the HAEi           patients.
          resources available to the member organizations. Ines
                                                                                                                                                                       JØRN SCHULTZ-BOYSEN
          Pessoa gave some ADAH data and updates of which             Speaking of North Africa: I am still looking for some
                                                                                                                                                                       NORDICS, GERMANY, AUSTRIA AND SWITZERLAND
          we can highlight these: There are an estimated 205          local contacts for pharmaceutical companies. Hopefully,
          people with HAE in Portugal, 40 members of ADAH and         I will have good news on this soon.

                                                                                                                                  It has been a pleasure starting as the Regional Patient       The Finnish website is in the middle of the migration
                                                                                                                                  Advocate for the Nordics and Germany, Austria and             to the HAEi hosted environment. In Austria, the first
                                                                                                                                  Switzerland. It is fantastic to meet – mostly online – with   steps have been taken to this as well.
                                                                                                                                  many of the member organizations’ representatives as
                                                                                                                                  there are so many things to talk about. And not least for     The regional meetings in Norway, Sweden and Denmark
                                                                                                                                  me to learn from these amazing people.                        are currently being planned as online meetings instead
                                                                                                                                                                                                of physical ones.
                                                                                                                                  We have had some very nice and tangible successes. The
                                                                                                                                  emergency card has now been translated to German,             And like last time, a callout: if you are a patient,
                                                                                                                                  covering Germany, Austria and Switzerland. Also, it has       caregiver or physician treating HAE patients in Iceland,
                                                                                                                                  been translated to the national languages in Finland,         the Faroe Islands or Greenland and would like to get
                                                                                                                                  Sweden, Norway and Denmark. Once the world will               in touch, please do not hesitate to reach out to me. It
                                                                                                                                  open more up again, and we can start to travel, this will     would be great to connect with you.
                                                                                                                                  undoubtedly be beneficial to HAE patients and their
                                                                                                                                  families. Also, the HAE Companion app holds these new
                                                                                                                                  translations to make it even easier to bring along the
                                                                                                                                  emergency cards on your trips.

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Perspectives - HAE International
NEWS FROM THE HAEi REGIONAL PATIENT ADVOCATES                                                                                                                                                        NEWS FROM THE HAEi REGIONAL PATIENT ADVOCATES

                                             JAVIER SANTANA                                                                                                       PATRICIA KARANI
                                             CENTRAL AMERICA AND CARIBBEAN                                                                                        SUB SAHARA AFRICA

          I participated in an interview with Dr. Olga Barrera in    I have been in communication with a new doctor from        In Ghana, we have been able to start discussions on       Furthermore, in Lesotho, we have established an
          Panama for a national newspaper to highlight HAE           the Dominican Republic who treats patients with HAE        HAE with the Immunology Society of Ghana (ISG). We        awareness campaign through the Rare Disease Lesotho
          patients’ situation in the country.                        and is willing to help identify more patients in the       have also been able to liaise with Rare Disease Ghana     group who have shared HAE awareness materials on
                                                                     country and ensure that treatments are registered in       for purposes of raising awareness amongst the general     their platforms, which has elicited a good public
          In Puerto Rico, I have had several communications with     the future.                                                public.                                                   response.
          Dr. Rafael Zaragoza, representatives of pharmaceutical
          companies, lawmakers, and legislative advisers about       I am working with my member organizations on media         We have established new doctor contacts in Tanzania.      In South Africa, we have introduced the Brady Club
          the reconsideration of the law that would give HAE         training and have provided a list of the most important    We are in the process of holding further discussions as   activity book for the youngsters. This is a wonderful
          patients access to treatment through the government’s      media contacts in their countries.                         regards HAE awareness and how we can improve HAE          publication that assists kids to better understand HAE
          public health insurance.                                                                                              knowledge amongst the health care professionals and       and how to manage themselves better. It also provides
                                                                     There have been some communications with CSL               the general public.                                       an opportunity to engage in fun activities and games
          During the past few months, I have also been working       Behring Latin America, who informed me that the                                                                      for youngsters.
          with my member organizations and doctors in the            pharmacy is taking steps to enter its treatments for       In Kenya, we are now working on spreading HAE
          region on the 2021 virtual conference project for Latin    HAE in Panama and Costa Rica.                              awareness around the different major referral hospitals
          America, with recording videos and developing scripts                                                                 through doctor webinars. I have presented on HAE
          and transcripts and checking videos. Furthermore, I        It should be noted that many countries are still in        and started ongoing conversations with the National
          have provided videos for the introductions for the         lockdown due to COVID-19, which is why it has made         Council of Persons Living with Disability (NCPWD) as a
          presentations.                                             it difficult for the leaders of the member organizations   way of ensuring that HAE being a debilitating condition
                                                                     to hold events, including being able to attend their       is included in being supported as a disability. I have
          I wrote a letter to the Ministry of Health in Costa Rica   medical appointments. Let’s hope in God that these         also presented on HAE as a speaker at the Africa Health
          in support of patients gaining access to treatments        challenges end soon.                                       Agenda International Conference (AHAIC) hosted in
          following the decision to reject drugs for HAE patients                                                               Kenya by AMREF and officially opened by the President
          because treatments were not approved in other                                                                         of the Republic of Kenya as regards driving momentum
          countries. I worked with the patients on media training                                                               to achieve Universal Health Coverage (UHC) in Africa.
          and interviews to highlight the issue.                                                                                Rare diseases were given a platform to discuss their
                                                                                                                                challenges and call for inclusivity in the UHC campaign
                                                                                                                                for Africa.

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Perspectives - HAE International
NEWS FROM THE HAEi REGIONAL PATIENT ADVOCATES                                                                                                                                                            NEWS FROM THE HAEi REGIONAL PATIENT ADVOCATES

                                             NATASA ANGJELESKA                                                                                                        FIONA WARDMAN
                                             SOUTH EASTERN EUROPE                                                                                                     ASIA PACIFIC

          In the course of December 2020 and January 2021, I         On 28 February, I had a presentation entitled “Act locally,   During the past few months, I have been working            Dr. Jindal Ankur from the Post Graduate Institute of
          held online meetings with patient representatives          think globally” at the patient meeting organized by           with member organizations in my region to nominate         Medical Education & Research in Chandigarh, India,
          from Montenegro, Greece, Albania and Bosnia and            HAE Serbia on the occasion of the organization’s fifth        physicians for the Regional Medical Advisory Panel         has created and registered a Physicians Association to
          Herzegovina. Some of the patients wanted to hear           anniversary. I was asked to present different treatment       (RMAP) project. I am pleased with the confirmation         assist with HAE education and awareness in India.
          details about the vaccines for COVID-19 because they       options across countries in South Eastern Europe and          from the nominated doctors who have accepted to
          feel insecure about the safety of people with HAE. I       discussed some of the challenges of HAE patients              be panelists. I look forward to holding the first RMAP     The Ministry of Health and Welfare in South Korea has
          have re-shared the link to the video of Professor Marcus   and their family members. Furthermore, I highlighted          meeting in the next few weeks.                             expanded reimbursement for HAE patients to have two
          Maurer, as well as explained the official advice issued    the part of the HAEi website where information and                                                                       vials of icatibant. Previously patients were only allowed
          from HAEi regarding vaccination.                           video discussions about HAE and COVID-19 (including           In late December 2020, I presented ‘International          one prefilled syringe at a time for self-administration.
                                                                     vaccination) are posted. I also presented the HAEi            experience on emergency management of larynx edema
          The University Clinic of Respiratory and Allergic          Advocacy Academy opportunities via online courses and         caused by Hereditary Angioedema’ during a webinar in       I have been working with HAE Japan on a webinar on
          Diseases in Golnik has become the first ACARE Center       encouraged patients to start using the HAE Companion          China to help patients and physicians understand the       HAE with Normal C1. We hope this webinar will answer
          in Slovenia.                                               app. I also talked about the brand new HAE TrackR app         severity of swelling of the throat and face. The Chinese   a lot of questions that patients and physicians may
                                                                     that will hopefully replace the paper documentation           Organization for Rare Disorders (CORD) and HAE China       have.
          In February, I used a great deal of my time in             that patients are using to track their attacks.               coordinated the webinar.
          communication with member organizations in my                                                                                                                                       I contributed to a paper on ‘Mitigating Disparity
          region regarding the hae day :-) anniversary initiative.                                                                 I have been assisting HAE Taiwan with the emergency        in Health-care Resources Between Countries for
                                                                                                                                   department poster and emergency cards translated           Management of Hereditary Angioedema’. This paper
          I was engaged in planning an event to mark Rare                                                                          with Dr. Shyur and Takeda’s assistance. The patients in    has been accepted and will be published soon.
          Disease Day in North Macedonia. I gave an interview                                                                      Taiwan now have access to icatibant and have had a
          about the situation of rare disease patients’ care and                                                                   training session on administration.                        I have been working with the member organizations on
          access to therapies for a Macedonian Information                                                                                                                                    submitting their information for the 2021 hae day :-)
          Agency that was published on 28 February. Also, I have                                                                   Awareness of HAE is low in Singapore, and not many         campaign ‘Let’s Take the Next Step’.
          been invited to an official event organized by the Prime                                                                 patients have been diagnosed or are not in touch with
          Minister and the Minister of Health of the Republic of                                                                   other patients. The head of the member organization
          North Macedonia. We discussed the present situation                                                                      and I have been working on her story to be shared with
          and our demands and challenges as patients with rare                                                                     the Rare Disorders Singapore Society (RDSS) on their
          diseases and had a chance to present our suggestions                                                                     website and social media to help locate more patients
          for improvement of the overall conditions and quality                                                                    in this country.
          of life of patients, caregivers and families.
                                                                                                                                   Together with HAE India, we have held two informal
          I was engaged with the HAE Macedonia team, Natasha                                                                       Zoom meetups with patients and doctors. These
          Jovanovska Popovska and Verce Jovanovska Jankovska                                                                       meetups are an excellent way for patients and their
          and initiated negotiations on getting prophylactic                                                                       family members to learn more about HAE and ask
          subcutaneous treatment in two youngsters who are                                                                         questions in a supportive environment. For the
          dealing with frequent attacks.                                                                                           February meet-up, we had a guest – Arouba, a mental
                                                                                                                                   health therapist and counsellor. Arouba also has a
                                                                                                                                   rare disease and gave practical advice and answered
                                                                                                                                   questions from the group. We will continue to hold
                                                                                                                                   virtual meetups each month.

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Perspectives - HAE International
HAE DAY :-) 10 YEAR ANNIVERSARY                                                                                                                                                                                                HAE DAY :-) 10 YEAR ANNIVERSARY

                                                                                                                                     workout classes, or you are reading, meditating or just    Updated campaign website
                                                                                                                                     find yourself pottering around the garden a bit more,      The updated website has lots of helpful
                                                                                                                                     you can enter the time for this activity.                  information about the campaign, inspiration for
                                                                                                                                                                                                activities and how you can get involved. As you add
                                                                                                                                     Regularly adding activities is important                   your activities to the website, you can share pictures
                                                                                                                                     “Adding your activities to regularly is         of what you are doing on hae day : -) 2021. If you also
                                                                                                                                     really important as this is what will help us visit our    share these via your social media channels, using the
                                                                                                                                     regions and member organizations. When we get to           hashtags #active4hae and #haeday10 others will be
                                                                                                                                     each region, everyone will be able to read more about      able to find you.
                                                                                                                                     the HAEi member organizations in that region. For
                                                                                                                                     example, what are their success stories, achievements,     “To make activity entering easier, we have introduced
                                                                                                      YEAR                           proudest moments as a community and what are their         the option to create a profile on If you
                                                                                                      ANNIVERSARY                    hopes for the future of HAE – as well as some fun          create a profile, you have the chance to earn our
                                                                                                                                     information about their country. This will be in both      limited-edition regional badges, one for each region
                                                                                                                                     English and local language. You’ll also get to hear from   where you enter an activity. Your profile will show a
                                                                                                                                     our Regional Patient Advocates and, hopefully, HAEi if     clear overview of all your activities entered and badges
                                                                                                                                     we make it around the world in time for hae day :-)        earned. For those of you who don’t want to create a
                                                                                                                                     2021”, Deborah Corcoran explains.                          profile but still want to enter activities and take part
                                                                                                                                                                                                in the challenge, don’t worry, you can still do that on
                                                                                                                                                                                      ”, says Nevena Tsutsumanova.

          16 May 2021 marks the 10th anniversary of the hae day       Take part in physical and wellbeing activities
          :-) awareness day. For the last ten years, HAEi and its     “We are asking HAE friends all over the globe to take
          member organizations worldwide have used 16 May as          part in physical or wellbeing activities and regularly
          a focus for raising awareness: not just on the day itself   record the time you spend on each activity, on our
          but in the weeks leading up to it.                          campaign website at Any and all activity
                                                                      reported here will be converted into steps. Our goal is
          Let’s Take the Next Step                                    to generate enough steps for a virtual walk that will
          “We want to celebrate this anniversary with a special       take us to all the HAEi regions, where our member
          campaign to recognize the work of our HAE Community         organizations will showcase their history, achievements
          over the last ten years. The theme of our 10th              and hopes for the future”, HAEi Chief Specialist Projects
          anniversary campaign is ‘Let’s Take the Next Step’.         and Research Deborah Corcoran explains:

          We’re looking back at HAE community achievements
          over the past ten years and shining a light on what
                                                                      “We are taking a walk around the world, and we are
                                                                      doing this via participation in lots of different activities
                                                                                                                                                              Let’s Take the Next Step for HAE advocacy
          we want to achieve through the power of advocacy in         both physical and wellbeing focused, not only walking.                                 and see what we can achieve when we work
          the future. There has been lots of positive progress, but   People with HAE know that stress is a trigger for
          there is still a long way to go before reaching our goals   attacks, which is why staying healthy both mentally                                      together for people with HAE across the
          of improved time to diagnosis and consistent access to
          lifesaving therapies for everyone with HAE around the
                                                                      and physically can help managing HAE. We’re also
                                                                      very aware that we continue to live with restrictions
                                                                                                                                                              globe – Let’s Take the Next Step together!
          world. Our work is far from over!”, says HAEi Operations    to help manage the COVID-19 pandemic, so we are
          Manager Nevena Tsutsumanova.                                encouraging participation in activities that can be
                                                                      done both outside and inside.”
          The campaign launches on 1 April with the activity
          challenge of stepping around the world in time for hae      Whether you are able to be out walking, running or
          day :-) on 16 May 2021.                                     cycling, or you have recently started following online

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Perspectives - HAE International
IMPROVEMENTS IN QoL                                                                                                                           IMPROVEMENTS IN QoL

                                        Prophylaxis therapies can lead
                                        to significant improvements in
                                        quality of life
                                                                                                   Therefore, we decided to assess the overall economic
                                                                                                   burden of on-demand-only treatment for HAE and
                                                                                                   compare patient quality of life with patients who
                                                                                                   received novel subcutaneous prophylactic therapies.”

                                                                                                   For the study, members of the US HAEA were invited
                                                                                                   to complete an anonymous online survey to profile
                                                                                                   attack frequency, treatment use, and the presence of
                                                                                                   comorbidities as well as economic and socioeconomic
                                                                                                   variables. The authors modelled on-demand treatment
                                                                                                   costs by using net pricing of medications in 2018, indirect
                                                                                                   patient and caregiver costs, and attack-related direct
                                                                                                   billed costs for emergency department admissions,
                                        According to HAEi President & CEO Anthony J. Castaldo,     physician office visits, and/or hospitalizations. Quality
                                        “A study of HAE medicines published by a group of          of life was assessed by using the Angioedema Quality
                                        economists who receive funding from the insurance          of Life questionnaire.
                                        industry concluded that new preventive HAE medicines
                                        are not cost-effective. The US HAE Association and         The study shows that per patient/year, direct costs
                                        HAEi joined forces in questioning the methods and          associated with modeled on-demand-only treatment
                                        conclusions of this analysis and responded by initiating   totalled 363,795 USD, with additional indirect
                                        a comprehensive study to determine the financial and       socioeconomic costs of 52,576 USD per patient/year.
                                        quality of life impact of the new preventive medicines.”   The most significant improvement in quality of life
                                                                                                   was seen in patients who used novel subcutaneous
                                        Almost 800 people with HAE participated in the study,      prophylactic therapies, as there was a 59.5 percent
                                        and the results point to a drastically different reality   improvement in median impairment scores versus on-
                                        from the insurance industry economists’ study. The         demand-only treatment. In addition, patients who used
                                        data reveals that the new preventive therapies yield       novel subcutaneous prophylactic therapies reported
                                        (1) substantial economic value in light of remarkable      a 77 percent reduction in the number of attacks each
                                        decreases in attack frequency and (2) statistically        year compared with those who used on-demand-only
                                        significant improvements in patient quality of life.       treatment.
                                        After an extensive peer review, the study ‘Assessing
                                        the cost and quality-of-life impact of on-demand-only      The authors concluded that the use of novel
                                        medications for adults with hereditary angioedema’ is      subcutaneous prophylaxis can lead to sizeable
                                        now published and available in print and free online at    reductions in attack frequency and statistically
                                                               significant and clinically relevant improvements in
                                                                                                   quality of life. In addition, the study noted that these
                                        Co-authors Anthony J. Castaldo, Deborah Corcoran,          data could be useful to clinicians and patients as they
                                        Henrik Balle Boysen from HAEi, Christian Jervelund         consider therapy options.
                                        from Copenhagen Economics, along with Bruce L.
                                        Zuraw and Sandra C. Christiansen from the Department
                                        of Medicine at the University of California San Diego,        The study ‘Assessing the cost and
                                        noted that, “Novel subcutaneous prophylactic therapies        quality-of-life impact of on-demand-only
                                        are transforming the treatment landscape of HAE.              medications for adults with hereditary
                                        Although questions are being raised about their cost,         angioedema’ is available in print and
                                        little attention has been paid to the cost and quality        free online at
                                        of life impact of using on-demand-only medications.

16 ⎮ GLOBAL PERSPECTIVES · MARCH 2021                                                                                         GLOBAL PERSPECTIVES · MARCH 2021 ⎮ 17
Perspectives - HAE International

                                        HAEi launches new app
                                        – by patients, for patients
                                        Very soon HAEi will launch an app by patients, for         “In addition, the app helps the user keep track of
                                        patients. The name of the new app is HAE TrackR and        prophylactic treatment just as it gathers useful data
                                        that is just what it is: An easy way for people with       that can be shared with the physician or other health
                                        HAE to track their condition, their attacks, and their     care professionals. It is important to note that the data
                                        treatments.                                                gathered by the app is the sole property of the user and
                                                                                                   only to be shared if he or she decides so. Furthermore,
                                        “Many HAE friends around the globe have encouraged         HAE TrackR has been designed by patients for patients
                                        us to introduce an app with an electronic diary to         with an emphazis on protecting the privacy of the users
                                        support people with HAE – no matter where they are,        data.
                                        what medication they have access to and what kind
                                        of electronic device they are using. This has led to the   “Both Apple’s App Store and Google Play will have the
                                        design of HAE TrackR. While providing both patients        app available for download. Furtermore, HAE TrackR
                                        and physicians with a better decision foundation for       can be accessed from it´s own website,
                                        planning the management of the individual’s HAE, the       allowing the user to also open HAE TrackR from their
                                        app still protects the privacy and data of the user. We    computer.”
                                        believe this will be an extremely valuable help for
                                        men and women with HAE”, says Henrik Balle Boysen,         In the words of HAEi’s President & CEO Anthony J.
                                        the HAEi Executive Vice President & Chief Operating        Castaldo:
                                                                                                   “HAE TrackR brings a new and very exciting tool to
                                        HAE TrackR will not only help users keep track of          our global HAE community. The data stored in the app
                                        their HAE attacks with and without treatment. HAEi’s       provides all the information the patient and physician
                                        Enterprise Technology Manager Ole Frølich explains:        needs to create an individualized HAE management

                                                                                                      Read more about the
                                                                                                      EU-GDPR compliant app

18 ⎮ GLOBAL PERSPECTIVES · MARCH 2021                                                                                        GLOBAL PERSPECTIVES · MARCH 2021 ⎮ 19
Regional Advisory Groups
          Define Their Top 5 Challenges
          Last year the Regional Patient Advocates rolled out a
          new HAEi initiative called Regional Advisory Groups
                                                                   The RAGs are made up of patients or family members
                                                                   from each member organization who the member
                                                                                                                           This growth, achieved in a short amount of time
                                                                                                                           highlights the importance and need for useful and
                                                                                                                                                                                   Top 5 Challenges
          (RAGs) in each of the regions they represent.            organization nominated as the Advisor for their         meaningful tools and resources to assist our member
                                                                                                                                                                                   1. Lengthy time to
                                                                   country. The appointed Advisor from each member         organizations to best serve their patient community”,
          The initiative followed an unanimous vote by the         organization meets several times a year with their      says Fiona Wardman, HAEi Chief Regional Patient            diagnosis
          HAEi General Assembly during the 2018 HAE Global         Regional Patient Advocate to discuss each country and   Advocate.                                               2. No modern treatment
          Conference in Vienna, Austria where it was resolved to   region’s challenges and unmet needs. The information
          broaden HAEi Governance participation by establishing    and outcomes from these meetings provide the HAEi       It is not surprising that there were five unmet needs
                                                                                                                                                                                      access or home therapy
          the RAGs. This initiative is a key component of the      Leadership with the basis for adjusting existing        and challenges consistently identified by all of the    3. Lack of HAE awareness
          decentralized approach to advocacy and support that      programs and services or establishing new projects      RAGs.
          HAEi has adopted.                                        and initiatives to best serve the needs expressed by
                                                                                                                                                                                   4. Motivating patients and
                                                                   the Advisors.                                           HAEi plans to hold the next Regional Advisory Group        care givers
          The decentralized approach means that HAEi can                                                                   meeting in the coming months.                           5. Lack of resources
          focus on each country and region’s specific needs more   “The HAEi’s global footprint has grown substantially
          precisely.                                               through the work of our Regional Patient Advocates.

20 ⎮ GLOBAL PERSPECTIVES · MARCH 2021                                                                                                                                                          GLOBAL PERSPECTIVES · MARCH 2021 ⎮ 21
South America & Mexico
                                                                                                                                                                                                                          Central America & Caribbean

                                                                                                                                     WELCOME TO THE 2021 HAEi
                                                                                                                                    VIRTUAL REGIONAL WORKSHOP
                                                                                                                                      SOUTH AMERICA & MEXICO
                                                                                                                                   CENTRAL AMERICA & CARIBBEAN

                                                                                                                             Launch of third virtual regional workshop
          HAEi establishes Regional                                                                                          After the successful virtual regional workshops in          of HAEi services such as emergency card, hosted

          Medical Advisory Panels                                                                                            South Eastern Europe and Central Eastern Europe and
                                                                                                                             Benelux, HAEi is now focused on Latin America.
                                                                                                                                                                                         websites and HAEi Connect.

                                                                                                                                                                                         The expert presentations are delivered by Dr. Olivera,
          The HAEi Regional Medical Advisory Panel initiative       “Once a RMAP has been established, a chairperson is      “The Latin American workshop is the first one for this      Panama (Diagnosis), Dr. Olivares, Colombia (Symptoms),
          is the next step in a decentralized approach to serve     appointed, and every panel member is encouraged to       region where the working language is the presenters’        Dr. Grumach, Brazil (Treatment), and Dr. Calderón, Peru
          the global HAE community. The objective of the            collaborate, network, share information, and exchange    local language. The workshop brings together the            (ACARE), while the Youngsters are represented by
          Regional Medical Advisory Panels (RMAP) is to obtain      ideas”, Fiona Wardman explains.                          Regional Patient Advocates Fernanda de Oliveira             Nathan Galarza and Isabel Brunkan.
          the physician viewpoint regarding challenges that                                                                  Martins from South America & Mexico and Javier
          countries (within a region) are facing in terms of        Each RMAP will meet twice a year via Zoom or             Santana from Central America & Caribbean”, says HAEi        All 17 member organizations in Latin America – that
          diagnosis, education, awareness, and access to modern     teleconference with the RPA to organize and take part    Chief Regional Patient Advocate Fiona Wardman.              is Argentina, Brazil, Chile, Colombia, Costa Rica, Cuba,
          treatments. HAEi’s member organizations will work         in the call to take minutes.                                                                                         Dominican Republic, Guatemala, Ecuador, El Salvador,
          with their Regional Patient Advocate (RPA) to nominate                                                             The workshop features 34 videos in the presenter’s          Mexico, Panama, Paraguay, Peru, Puerto Rico, Uruguay,
          physicians to be part of RMAPs.                           “Furthermore, the RMAPs will have an opportunity to      local language with subtitles in Spanish, Portuguese,       and Venezuela – are presented through short videos on
                                                                    meet and interact with the HAEi Regional Advisory        and English.                                                the present situation in each country.
          Each RMAP will gather physicians’ perspectives on         Groups and RPAs during our regional conferences
          circumstances and unique needs in their country and       and the HAEi Global Leadership Workshops”, Fiona         The workshop is divided into four tracks: HAEi,             The workshop’s final part is a Q&A video with the two
          region and share this information with the member         Wardman says.                                            Experts, Youngsters, and updates from the member            Regional Patient Advocates teaming up with Dr. Olivera,
          organizations and RPAs.                                                                                            organizations.                                              Dr. Olivares, Dr. Grumach, and Dr. Calderón.
                                                                    Additionally, Advisors will have access to the HAEi’s
          “This important initiative will enable us to adapt        Chief Medical Advisor, and the global organization       The workshop begins with brief introductions from HAEi
          HAEi’s existing programs, activities, and services, and   encourages each Advisor’s clinic/hospital to become an   Leadership and the two Regional Patient Advocates.
          implement new ones to address the local and regional      accredited ACARE Center.                                 The first part of workshop covers ‘Building foundations
          issues and concerns raised by the Regional Medical                                                                 for a better future’ (President & CEO Anthony J. Castaldo
          Advisory Panels,” says Fiona Wardman, HAEi’s Chief                                                                 and Executive Vice President & Chief Operating Officer         VIDEOS IN SPANISH, PORTUGUESE
          Regional Patient Advocate.                                                                                         Henrik Balle Boysen), ‘Decentralized Approach in               AND ENGLISH
                                                                                                                             Practice’ (Fiona Wardman), ‘ACARE’ (Anthony J. Castaldo
                                                                                                                             and Professor Marcus Maurer) as well as a presentation         You have access to all the videos of the Latin
                                                                                                                                                                                            American workshop at

22 ⎮ GLOBAL PERSPECTIVES · MARCH 2021                                                                                                                                                                             GLOBAL PERSPECTIVES · MARCH 2021 ⎮ 23

                                                          Meeting old and new friends
                                                          via HAEi Youngsters’ online meetups
                                                          By Hana Faulds

                                                          Hey Youngsters!                                               in December we were all about the holiday spirit and
                                                                                                                        what people were most excited about this holiday
                                                          What a crazy year 2020 was, right! And to that note we        season.
                                                          send our love to all of you around the globe.
                                                                                                                        It was wonderful to meet new people and reconnect
                                                          2020 was the year of changes and for many that meant          with old friends and it was definitely the highlight of
                                                          finding different ways to connect. Meeting online has a       our 2020! We are so grateful for this global community
                                                          whole new and different meaning, hasn’t it?                   and we look forward to many more meetups to come
                                                                                                                        in 2021!
                                                          We are very sad we didn’t get the chance to see
                                                          everyone at the 2020 HAE Global Conference, however           If you want to say hello, keep a look out on our
                                                          that pushed us to think outside the box and find a new        Instagram for more meetups that you could join!
                                                          way to stay in touch.

                                        HAEi YOUNGSTERS   Chatting with our international friends is what we miss
                                                          the most and that is why the HAEi Youngsters Advocacy

            CONNECTED BY HAE.
                                                          Group launched the HAEi Youngsters online meetups in
                                                          November 2020, and it was a huge success! Our hope
                                                          is that through those meetups we get the chance to
                                                          catch up, have fun, talk about HAE and support each
                                                          other. We are happy we found a way to do this with the

            UNITED AS A FAMILY.
                                                          help of HAEi.

                                                          The idea is simple. At the end of each month, we invite
                                                          you to join us on a Zoom call and say hello. On our
                                                          Instagram you can always find more information about
                                                          the theme of the meetup, the date and time as well
                                                          as a registration link. (Psst.. If you’re not already doing
                                                          so, make sure to follow our Instagram account @
                                                          haeiyoungsters so you never miss out on any updates
                                                          from us)

                                                          We want to make sure that everyone on the call gets
                                                          a chance to share their story and that is why we have
                                                          a limited space of 15 people per call. If you are new
                                                          to the community and have never joined a youngsters
                                                          meeting, don’t worry! We always start the meetups with
                                                          a (virtual) roundtable introduction to break the ice.

                                                          Our first two meetups in November and December 2020
                                                          and were a lot of fun! In November we introduced the
                                                          theme ‘Meet around the world’ and we were overjoyed
                                                          to say it was fully booked. We had youngsters joining
                                                          us from six different countries! Some had never met
                                                          young people with HAE before, and some had never
                                                          been to a global conference, but they were all excited
                                                          to chat with HAE friends and share their stories. And

24 ⎮ GLOBAL PERSPECTIVES · MARCH 2021                                                                                                            GLOBAL PERSPECTIVES · MARCH 2021 ⎮ 25
EMERGENCY ROOM POSTER                                                                                                                                                                                                                                                                                                                                                                                                                                        EMERGENCY ROOM POSTER

                                                                                                                                                                                                                                                                                                                                                                    !       IMMEDIATE CARE REQUIRED                                         !

                                                                                                                                                                                                                                                                                                                                      NOT ALL
          Emergency room posters help raise awareness                                                                                                                                                                                                                                                                                 ANGIOEDEMA
          HAEi friends in many countries cite the need for             Established member organizations with funding
                                                                                                                                                                                                                                                                                                                                      IS ALLERGIC
          significant improvement in emergency room care for           sources are requested to fund printing, postage or                                                                                                                                                                                                             Hereditary Angioedema (HAE) presents with
          people with HAE. Because education is the key to             delivery. In contrast, patient groups without a funding                                                                                                                                                                                                        recurrent cutaneous swelling, abdominal
                                                                                                                                                                                                                                                                                                                                      pain, and/or airway obstruction due to
          better emergency care, HAEi has prepared a poster            source can have their print expenses covered by HAEi.
          with important information on recognizing HAE and            However, if version three of the poster is chosen, the                                                                                                                                                                                                         HAE is often misdiagnosed as allergic or
          successfully treating an emergency.                          member organization will be covering all costs for                                                                                                                                                                                                             histamine-mediated angioedema.
                                                                       customization, printing and distribution.
                                                                                                                                                                                                                                                                                                                                      HAE SWELLING IS CAUSED
          To ensure the most widespread use of the poster, it is
                                                                                                                                                                                                                                                                                                                                      BY EXCESSIVE BRADYKININ
          to be translated into as many languages as possible. At      The emergency room poster files are available in three
                                                                                                                                                                                                                                                                                                                                      PRODUCTION TYPICALLY DUE TO
          this point, it exists in for instance English, Spanish and   sizes:                                                                                                                                                                                                                                                         LOW C1-INH PROTEIN FUNCTION.
          Arabic – and over the coming months, it will turn up
                                                                                                                                                                                                                                                                                                                                      Allergy medications (antihistamines,
          around the world in many local languages.                    • A2 (420mm x 594mm)
                                                                                                                                                                                                                                                                                                                                      corticosteroids, epinephrine) are ineffective
                                                                       • B1 (500mm x 700mm)                                                                                                                                                                                                                                           in treating angioedema due to HAE.
          There are three versions of the poster. The first is a       • Poster Size Large 609.6mm x 914.4mm                                                                                                                                                                                                                          Swelling of the airway is dangerous and
          standard poster with HAEi contact information and                                                                                                                                                                                                                                                                           can lead to death by asphyxiation.
          logo, while the second has the same information with         Option 1: The original standard poster with HAEi
          space to include the national member organization’s          contact information and logo.
          logo and website or other contact details. Thirdly                                                                                                                                                                                                                                                                           TREATMENT OF HAE                                SYMPTOMS OF HAE
          there is a fully customizable poster. This version is        Option 2: The original standard poster with space to                                                                                                                                                                                                           • Drugs approved for treating acute HAE
                                                                                                                                                                                                                                                                                                                                                                                       Face                               Larynx, glottis
                                                                                                                                                                                                                                                                                                                                        attacks (where available)
          without the HAEi watermark and logo and can thus be          include the member organization’s logo and website                                                                                                                                                                                                                                                             • Feeling of tension in the skin    • Difficulty swallowing
                                                                                                                                                                                                                                                                                                                                      • Fresh Frozen Plasma (FFP) (when no
          customized by the member organization.                       or contact details. The example is with the HAE                                                                                                                                                                                                                  approved HAE treatments are available)        • Swelling of face and/or lips      • Hoarseness
                                                                       Scandinavia logo and information.                                                                                                                                                                                                                                                                              • Erythematous, non-pruritic        • Changes in the voice
                                                                                                                                                                                                                                                                                                                                      Airway swelling is particularly                   rash in 25% of patients           • Shortness of breath
          Over the last few months, member organizations have
                                                                                                                                                                                                                                                                                                                                      dangerous and can lead to death                                                     • In extreme cases, suffocation
          been in contact with hospitals and clinics in their          Option 3: A fully customizable poster without the HAEi                                                                                                                                                                                                                                                          Abdomen
                                                                                                                                                                                                                                                                                                                                      if not treated immediately.
          country to present the poster and encourage them to          and logo. The poster can be fully customized by the                                                                                                                                                                                                                                                            • Vomiting                           Extremities
                                                                                                                                                                                                                                                                                                                                                                                      • Diarrhea                          • Feeling of tensions in the skin
          use the poster within the emergency rooms.                   member organization.
                                                                                                                                                                                                                                                                                                                                                                                      • Colic-like pain, often severe     • Swelling
                                                                                                                                                                                                                                                                                                                                                                                      • Nausea                            • Restricted movement
          Production of the poster takes place locally as HAEi                                             !       IMMEDIATE CARE REQUIRED                                         !
                                                                                                                                                                                                                                                                                                                                                                                                                          • Erythematous, non-pruritic
          emails a print-ready file for the poster to the member             NOT ALL                                                                                                                                                                                                                                                                      Mortality rate:                                                   rash in 25% of patients
          organization, who then sources a local company to print                                                                                                                                                                                   !

                                                                                                                                                                                                                                                                                                                                                          30 %
                                                                                                                                                                                                                                                          IMMEDIATE CARE REQUIRED
                                                                             ANGIOEDEMA                                                                                                                                                                                                                                  !

                                                                             IS ALLERGIC
          the poster. Distribution is managed either as hand-
          delivered or by mail, in both cases via the member           1     Hereditary Angioedema (HAE) presents with
                                                                             recurrent cutaneous swelling, abdominal
                                                                             pain, and/or airway obstruction due to
                                                                             HAE is often misdiagnosed as allergic or
                                                                             histamine-mediated angioedema.

                                                                             HAE SWELLING IS CAUSED
                                                                                                                                                                                                                 NOT ALL
                                                                                                                                                                                                                 IS ALLERGIC                                                                                                                                                           KNOW MORE ABOUT HAE
                                                                             BY EXCESSIVE BRADYKININ                                                                                                            Hereditary Angioedema (HAE) presents with
                                                                             PRODUCTION TYPICALLY DUE TO                                                                                                        recurrent cutaneous swelling, abdominal
                                                                             LOW C1-INH PROTEIN FUNCTION.                                                                                                       pain, and/or airway obstruction due to
                                                                             Allergy medications (antihistamines,                                                                                               HAE is often misdiagnosed as allergic or
                                                                             corticosteroids, epinephrine) are ineffective                                                                                      histamine-mediated angioedema.
                                                                             in treating angioedema due to HAE.
                                                                             Swelling of the airway is dangerous and                                                                                            HAE SWELLING IS CAUSED
                                                                             can lead to death by asphyxiation.                                                                                                 BY EXCESSIVE BRADYKININ
                                                                                                                                                                                                                PRODUCTION TYPICALLY DUE TO
                                                                                                                                                                                                                LOW C1-INH PROTEIN FUNCTION.
                                                                              TREATMENT OF HAE                                 SYMPTOMS OF HAE
                                                                                                                                                                                                               Allergy medications (antihistamines,
                                                                             • Drugs approved for treating acute HAE                                                                                           corticosteroids, epinephrine) are ineffective
                                                                                                                               Face                              Larynx, glottis
                                                                               attacks (where available)                                                                                                       in treating angioedema due to HAE.
                                                                                                                              • Feeling of tension in the skin   • Difficulty swallowing
                                                                             • Fresh Frozen Plasma (FFP) (when no                                                                                              Swelling of the airway is dangerous and
                                                                               approved HAE treatments are available)         • Swelling of face and/or lips     • Hoarseness
                                                                                                                                                                                                               can lead to death by asphyxiation.
                                                                                                                              • Erythematous, non-pruritic       • Changes in the voice
                                                                             Airway swelling is particularly                    rash in 25% of patients          • Shortness of breath
                                                                             dangerous and can lead to death                                                     • In extreme cases, suffocation
                                                                                                                               Abdomen                                                                         TREATMENT OF HAE
                                                                             if not treated immediately.                                                                                                                                                         SYMPTOMS OF HAE
                                                                                                                              • Vomiting                          Extremities

                                                                                                                                                                                                               • Drugs approved for treating acute HAE
                                                                                                                              • Diarrhea                         • Feeling of tensions in the skin
                                                                                                                                                                                                                 attacks (where available)                      Face                               Larynx, glottis
                                                                                                                              • Colic-like pain, often severe    • Swelling
                                                                                                                                                                                                               • Fresh Frozen Plasma (FFP) (when no            • Feeling of tension in the skin   • Difficulty swallowing
                                                                                                                              • Nausea                           • Restricted movement                           approved HAE treatments are available)        • Swelling of face and/or lips     • Hoarseness
                                                                                                                                                                 • Erythematous, non-pruritic                                                                  • Erythematous, non-pruritic

                                                                                                                                                                                                                                                                                                                                               HAE Scandinavia is the national patient organization         HAE International is the global umbrella organization
                                                                                                                                                                                                                                                                                                  • Changes in the voice
                                                                                                 Mortality rate:                                                   rash in 25% of patients                     Airway swelling is particularly                   rash in 25% of patients
                                                                                                                                                                                                                                                                                                  • Shortness of breath
                                                                                                                                                                                                               dangerous and can lead to death
                                                                                                 30 %                                                                                                          if not treated immediately.                      Abdomen                          • In extreme cases, suffocation
                                                                                                                                                                                                                                                               • Vomiting

                                                                                                                                                                                                                                                                                                                                                for HAE patients in Denmark, Norway and Sweden.                   for HAE patient groups around the world.
                                                                                                                                                                                                                                                                                                   Extremities
                                                                                                                                                                                                                                                               • Diarrhea
                                                                                                                                                                                                                                                                                                  • Feeling of tensions in the skin
                                                                                                                                                                                                                                                               • Colic-like pain, often severe
                                                                                                                                                                                                                                                                                                  • Swelling
                                                                                                                                                                                                                                                               • Nausea
                                                                                                                                                                                                                                                                                                  • Restricted movement
                                                                                                                                                                      Website:
                                                                                             KNOW MORE ABOUT HAE                                                                                                                                                                                  • Erythematous, non-pruritic
                                                                                                                                                                                                                                  Mortality rate:
                                                                            HAE International is the global umbrella organization                                                                                                                                 rash in 25% of patients
                                                                              for HAE patient groups around the world. Find more
                                                                           information on our website and social media channels.                                     
                                                                                                                                                                                                                                  30 %

                                                                                                                                                                                                                                                                                                                                                                        SUPPORTED BY HAE INTERNATIONAL · HAEI.ORG
                                                                                                                         HAE INTERNATIONAL · HAEI.ORG

                                                                                                                                                                                                                                              SUPPORTED BY HAE INTERNATIONAL · HAEI.ORG

26 ⎮ GLOBAL PERSPECTIVES · MARCH 2021                                                                                                                                                                                                                                                                                                                                                                                                        GLOBAL PERSPECTIVES · MARCH 2021 ⎮ 27


                                                                The network of ACARE centers continues to grow            Balle Boysen, HAEi. Here the reach was between 116          insights and treatment options that have recently
                                                                despite the many challenges created by COVID-19.          and 172 attendees representing almost 40 countries.         become available. Each preceptorship will include
                                                                                                                                                                                      interactive elements, case discussions, and lectures,
                                                                ACARE is a joint venture with GA²LEN and HAEi that        In 2019, live ACARE preceptorships on recurrent             with additional material provided online for download”,
                                                                fulfills HAEi’s longstanding goal of establishing a       angioedema were held in Beijing, China and Muscat,          HAEi’s Chief Medical Advisor and GA2LEN/HAEi ACARE
                                                                worldwide network of accredited angioedema care           Oman. More live ACARE preceptorships will happen            Coordinator Marcus Maurer explains.
                                                                centers.                                                  once this is possible again, the next one in Hiroshima,
                                                                                                                          Japan.                                                      As for ‘Make a Difference’, it is a direct continuation
                                                                In 2020 and 2021, the ACARE Network launched, hosted                                                                  of the ‘The Power of Prophylaxis’ series of webinars.
                                                                and aired two webinar series.                             ACARE is currently working on two scientific projects:      Planned to be held during the two last quarters of
                                                                                                                                                                                      2021 and the first quarter of next year, this series of five
                                                                The first was ‘Hereditary Angioedema in Times of          •   IMAGINE – with the pilot phase ongoing – is             webinars will explore how physicians can best aim to
                                                                COVID-19’, two webinars on the pandemic and its effects       focused on the identification of mutations in genes     provide the optimal diagnostic work-up and therapy to
                                                                in dermatology and allergy, with a particular focus on        of patients with recurrent idiopathic and hereditary    all HAE patients as early as possible. The webinars will
                                                                the impact of the pandemic on patients with recurrent         angioedema. The aim is to identify known and            cover the strategy of an earlier and broader diagnostic
                                                                angioedema, including HAE. Faculty were Professor             novel mutations in patients with bradykinin-            strategy that reduces delay in diagnosis and of a close-
                                                                Maurer, Dr. Melba Munoz-Roldan and Professor Markus           mediated recurrent angioedema using dried blood         meshed therapy control that allows optimizing therapy
                                                                Magerl, all from the Angioedema Center of Reference           spot analysis. Project leads are Markus Magerl,         methods continuously.
                                                                and Excellence at Charité Universitätsmedizin,                Thomas Buttgereit and Marcus Maurer.
                                                                Berlin, Germany. Both webinars were well attended                                                                     Regarding future projects, Professor Maurer says that
                                                                and received with more than 100 participants, each        •   SHAERPA – a project in development – focuses            ACARE is currently working to implement a platform
                                                                representing over 30 countries.                               on stopping androgen treatment in HAE patients          and program for physician education and information
                                                                                                                              with characterization of reasons and protocols and      on recurrent angioedema under the name ‘ACARE
                                                                The second webinar with the title ‘The power of               development of advice for patients and physicians.      LevelUp’:
                                                                prophylaxis’ was a three episodes webinar series on           Here the aim is to develop guidance on how to
                                   Professor Marcus Maurer      the burden of HAE, the aims of treatment, and the role        discontinue androgen treatment based on patient         “ACARE LevelUp will be a comprehensive and long-
                                 HAEi’s Chief Medical Advisor   and relevance of prophylaxis. This series’ objective          data. Project leads are Carolina Vera, Marcus Maurer    term educational platform and program for healthcare
                         and GA2LEN/HAEi ACARE Coordinator      was to review the impact of HAE on patients and their         and Markus Magerl.                                      professionals to learn, network, and share best clinical
                                                                families, discuss the aims of HAE management and                                                                      practices in recurrent angioedema, worldwide. ACARE
                                                                guideline recommendations, talk about strategies for      Furthermore, the network is currently working on the        LevelUp will be interactive, virtual and aim to update
                                                                assessing treatment needs and responses, and the tools    INTERACT preceptorship, and the ‘Make a Difference’         physicians on recent developments in angioedema and
                                                                to help with this. Furthermore, the webinars reviewed     webinar series.                                             establish a platform for interaction among physicians
                                                                the efficacy and safety of prophylactic treatment                                                                     who treat patients with recurrent angioedema.
                                                                options and shared real-life experience. For these        With the INTERACT preceptorship series, to be held          Furthermore, the aim is to help health care professionals
                                                                webinars, the faculty was Chief of Service Margarida      during the second, third and fourth quarters of 2021,       involved in the management of angioedema to
                                                                Goncalo from the University of Coimbra in Portugal,       ACARE is reaching out to physicians who treat patients      network on a regional, national and global level and
                                                                Associate Professor Maria Staevska from Sofia Medical     with recurrent angioedema with a series of region-          to improve the interaction of general practitioners and
                                                                University in Bulgaria, Consultant Clinical Immunology    specific, web-based educational training programs,          specialists, including ACAREs, to streamline the patient
                                                                Sorena Kiani from Royal London Hospital in the United     focusing on the differential diagnosis, diagnostic work-    journey to disease control. ACARE LevelUp will feature
                                                                Kingdom, Dr. Hilary Longhurst from Auckland District      up and treatment of recurrent angioedema.                   several formats and activities, including webinars,
                                                                Health Board, New Zealand, Dr. Anna Sala-Cunill from                                                                  informational websites, podcasts, poster sessions and
                                                                Universitat Ramon Llull in Barcelona, Spain, as well as   “The four-hours-long INTERACT preceptorships will not       more.”
                                                                Professor Magerl and Professor Maurer from Charité        only focus on the clinical spectrum, burden, differential
                                                                Universitätsmedizin, Berlin, Germany. Furthermore,        diagnosis, and the diagnostic work-up in patients with
                                                                featured guests were President & CEO Anthony J.           recurrent angioedema but also the management of
                                                                Castaldo and Executive Vice President & COO Henrik        recurrent angioedema, with a special focus on new

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          Today the global network of ACARE centers consists of more than 50 members spread over most of the globe.
          Please see for contact details of all ACARE centers. Presently the centers are:

          Argentina:                                               France:                                                  Japan:                                                    Russia:
          • Hospital Italiano de Buenos Aires, Buenos Aires        • CREAK: National french reference center for            • Department of Dermatology, Graduate School of           • Moscow Center of Allergy and Immunology, City
          • Instituto de Alergia e Inmunologia del Sur, Hospital     angioedema, Grenoble                                     Biomedical and Health Sciences, and Hiroshima             Clinical Hospital № 52, Moscow
            Italiano Regional del Sur, Buenos Aires                • Montpellier University Hospital, Montpellier             University Hospital, Hiroshima                          • NRC Institute of lmmunology FMBA Russia, Moscow
                                                                   • Service de dermatologie et allergologie, Hôpital       • Division of Dermatology, Kobe University Graduate
          Brazil:                                                    Tenon, APHP, Paris                                       School of Medicine, Kobe                                 Saudi Arabia:
          • Alergoalpha/CPAlpha Allergy Clinic and Clinical                                                                 • Yokohama City University Hospital, Yokohama             • King Faisal Specialist Hospital & Research Centre,
            Research Center, Barueri                               Georgia:                                                                                                             Riyadh
          • Clementino Fraga Filho University Hospital Federal     • Center of Allergy and lmmunology Tbilisi, Tbilisi      Kuwait:
            University of Rio de Janeiro, Internal Medicine                                                                 • Al-Rashed Allergy Center Kuwait, Kuwait City            Slovenia:
            Department, Immunology Service, Rio de Janeiro         Germany:                                                                                                           • University Clinic of Respiratory and Allergic Diseases
          • Clinical Hospital of Ribeirão Preto Medical School,    • Department of Dermatology, Venereology and             Netherlands:                                                Golnik, Golnik
            Division of Allergy and Immunology, University of        Allergology, Universität Leipzig, Leipzig              • Amsterdam UMC, location AMC, Amsterdam
            São Paulo, Ribeirão Preto                              • Elbeklinikum Buxtehude, Klinik für Dermatologie,                                                                 South Africa:
          • Division of Clinical lmmunology and Allergy, São         Buxtehude                                              Oman:                                                     • UCT Lung Institute, Cape Town
            Paulo University, São Paulo                            • Hautklinik und Poliklinik Universitätsmedizin der      • Immunology and Allergy Unit, Royal Hospital
          • Federal University of São Paulo, São Paulo               Johannes Gutenberg-Universität Mainz, Mainz              Muscat, Muscat                                          Spain:
          • Servico de Alergia e Imunologia, Complexo Hospital     • Klinik für Dermatologie, Venerologie und                                                                         • Department of Dermatology Hospital del Mar,
            de Clinicas Universidade, Curitiba                       Allergologie, Charité – Universitätsmedizin Berlin,    Peru:                                                       Barcelona
          • Serviço de Referência em Angioedema do Hospital          Berlin                                                 • Allergy Unit at Clinica San Felipe, Lima
            Santa Casa de Misericórdia de Vitória, Vitória         • Occupational Dermatology Department of                 • SANNA / Clínica el Golf, Lima                           Thailand:
          • University Center Health ABC, Medical School, São        Dermatology, Universitiy Hospital Carl Gustav Carus,                                                             • Siriraj Urticaria and Angioedema Center, Bangkok
            Paulo                                                    Technical University Dresden, Dresden                  Poland:
                                                                   • Universitätsmedizin Götting, Göttingen                 • Centralny Ośrodek Wrodzonego Obrzęku                    Turkey:
          Bulgaria:                                                                                                           Naczynioruchowgo, Centrum Alergologii Klinicznej,       • Division of Immunology and Allergic Diseases,
          • Clinic of Allergology, University Hospital„            Greece:                                                    Szpital Uniwersytecki w Krakowie, Krakow                  Department of Internal Medicine, Istanbul Faculty of
            Alexandrovska“, Medical University of Sofia, Sofia     • Referral Center of Occupational Dermatological         • Department of Dermatology, Venerology and                 Medicine, Istanbul
          • Medical Center Excelsior, Sofia                          Diseases, Athens                                         Pediatric Dermatology, Samodzielny Publiczny            • Hacettepe University Faculty of Medicine, Ankara
                                                                                                                              Szpital Kliniczny Nr.1, Lublin                          • Kayseri City Education and Research Hospital,
          China:                                                   Hungary:                                                 • European Center for Diagnosis and Treatment of            Kayseri
          • First Hospital, Peking University, Beijing             • Hungarian Angioedema Reference Center, Budapest          Urticaria, Zabrze
          • Sun Yat-sen Memorial Hospital, Guangzhou                                                                                                                                  United Arab Emirates:
                                                                   India:                                                   Portugal:                                                 • Immunology & Allergy Clinic, Cleveland Clinic, Abu Dhabi
          Denmark:                                                 • Allergy Immunology Unit, Department of Pediatrics,     • Clínica de Dermatologia Centro Hospitalar e
          • Bispebjerg Hospital, Department of Dermatology,          Postgraduate Institute of Medical Education and          Universitário de Coimbra, Coimbra                       United Kingdom:
            Copenhagen                                               Research, Chandigarh                                                                                             • Barts Health NHS Trust, London
                                                                   • Department of Dermatology at Postgraduate              Qatar:                                                    • The London Allergy & Immunology Center, London
                                                                     Institute of Medical Education and Research,           • Allergy and Immunology Section, Hamad General
                                                                     Chandigarh                                               Hospital, Doha
                                                                   • Dr. D.Y. Patil Medical College & Hospital, Mumbai

                                                                                                                                      If you would like to become an ACARE center, please use this link for further information:
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