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Bloodline VO LU M E 4 8 N U M B E R 2 J U LY 2 0 2 0
Bloodline
Magazine of the Haemophilia Foundation of New Zealand. Volume 47 Number 3
CONTENTS CONTACTS
The H Word ......................................................................................03 Website Lynne Campbell
www.haemophilia.org.nz Central Outreach Worker
CEO Report .......................................................................................04 PO Box 24014
National Office
Manners Street
WFH Virtual Summit ..................................................................05 PO Box 7647
Central Wellington 6142
Sydenham
You’re invited to the 2020 HFNZ Adult Weekend!......14 04 382 8442
Christchurch 8240
lynne@haemophilia.org.nz
03 371 7477
Outreach and Member Details................................................15
info@haemophilia.org.nz Amber Maihi
Looking after ourselves ...............................................................16 President Northern Outreach Worker
Deon York PO Box 41-062
Revised schedule for Port Hills walks 2020 ....................17 St Lukes, Mt Albert
president@haemophilia.org.nz
Auckland 1346
Region and Group Reports .......................................................18 Chief Executive
09 845 4658
Sue Ellis
Gene Therapy Shows Promise For Hemophilia, amber@haemophilia.org.nz
sue@haemophilia.org.nz
But Could Be Most Expensive U.S. Drug Ever...............20
Editor & Communications Manager
#ISTH2020 – FLT180a Gene Therapy Phil Constable
Shows Promise for Hemophilia B Patients
phil@haemophilia.org.nz
in Phase 1/2 Trial ..........................................................................22
Administrator
Managing Chaos as a Family...................................................23 admin@haemophilia.org.nz
03 371 7477
We Are the Experts of Our Own Stories ............................24
Ross Paterson
The Year Ahead ...............................................................................25 Southern Outreach Worker
PO Box 7647, Sydenham
Christchurch 8240
03 371 7485
ross@haemophilia.org.nz
OUTREACH FREEPHONE
Disclaimer: The information contained in this magazine is 0508 322 867
not intended to take the place of medical advice from your GP,
haematologist, or specialist. Opinions expressed are not necessarily HAEMOPHILIANZ
those of HFNZ.
The purpose of this magazine is to provide a wide range of accurate
Bank Details
and timely information on all aspects of haemophilia and related
disorders. Haemophilia is a dynamic specialty and therefore opinion Acct Name: Haemophilia Foundation of New Zealand
may change or be varied from time to time. Acct Number: 02 0828 0102656 000
2 Bloodline July 2020
THE H WORD
I hope you are all keeping warm during these winter
months. As New Zealand has progressively opened, so
too has HFNZ. Planning for regional and national events
is now well underway. We also have the results of our
member survey, and the national council will be taking a
good look at this to make sure HFNZ is delivering what
members need.
As a council, we continue to pay close attention to the
financial health of the organisation, for now and the
future. We are so fortunate to have the continued
support of the New Zealand public via Kiwifirst,
generous benefactors, donors, companies, trusts and
other organisations willing to support us financially.
Times are tough out there for many, and I cannot
emphasis enough our gratitude for this support. It really
does make a difference to our community. I would also
like to acknowledge the time of our volunteers. It is
priceless in more ways than one!
Since the last edition of Bloodline, there have been
opportunities to continue to connect with our global
community despite the challenges of a global pandemic.
I hope that many of you were able to attend the World
Federation of Hemophilia’s first global summit. Most of
the content is still available to access free-of-charge
online. There is also an opportunity to participate in the
National Hemophilia Foundation’s (NHF) bleeding
disorder conference 2020. The NHF is the (much larger)
equivalent of HFNZ in the United States. As you can see,
events have gone borderless this year, so now is the
perfect time to acquaint yourself with the global
community and have direct access to the latest research
and developments in care for people with inherited
bleeding disorders.
Deon York
HFNZ President
Bloodline July 2020 3
From the CEO
BY SUE ELLIS
The last few months have been testing for us all as we Planning is also underway for the Youth Camp 28 April
navigated our way through the Covid-19 levels with – 2 May 2021, again led by Ross, with support from the
Outreach Workers unable to meet members face-to-face. team and valuable advice from Colleen McKay. This camp
Instead, we’ve made more of an effort to check with will be held at Blue Skies in Kaiapoi, North Canterbury.
members via technology – Zoom, phone calls, Facebook,
As you may be aware, following the financial disruption of
and emails. With a return to some normality under Level 1,
Covid-19, a decision was made by the National Council to
we can now look forward to reconnecting with members
postpone recruitment of an Outreach Worker for the
via face-to-face visits.
Midland Region. In the meantime, Lynne and Amber are
As we moved through the Covid-19 alert levels, we worked covering the Midland region, with the region split between
to maintain connections with members and with our them. For more information on this, check out the article
supporters, and to plan events we knew could provide later in Bloodline.
support under these unusual circumstances.
I want to acknowledge Amber and Lynne for taking on this
It was lovely to get so many responses to the HFNZ extra work and the difficulty they have had tracking down
Member Survey. Congratulations to members Kathryn members in the Midland region. I am very appreciative of
MacGregor and Brendan Lee for winning our Prezzy Card them both for being quick to offer support for the Midland
draw. A quick snapshot of the data shows that 98% of members, while continuing to look after their own regions,
members are well aware of what HFNZ does to meet their until we are in a better position to recruit an OR Worker for
diverse needs, both regionally and nationally, and value our the Midland region again.
national events. I was pleased to see that the Outreach
While it has been a difficult time for all of us to manage our
service scored highly in providing access to services,
work through the Covid-19 alert levels, the most important
up-to-date information, the amount of contact, and benefit
focus has been on ensuring members received the support
for those contacts. Members are also satisfied with the way
and care they need. It is a great relief that we are now able
information gets to them via the website, Bloodline, Pānui,
to meet with members face-to-face and to enjoy a more
and Facebook with almost 100% of responses happy that
relaxed environment to work in.
the information is relevant, up-to-date, and reflects our
community. However, there appears to be less satisfaction
with our website.
CSL Behring Pharmaceuticals has kindly provided funding
to help improve health outcomes for members through a
redesigned and refreshed website. HFNZ has contracted a
very experienced Wellington company to complete the
update, and work is already underway.
In other funding news, I was very pleased to attend the
national conference of the Buffalo Lodge, where the
Franklin Lodge very generously donated $20,000 to
HFNZ. This was a lovely surprise, and very useful at this
difficult time.
Planning for the Adult Weekend Workshop in
Christchurch 6 – 8 November 2020 is well advanced.
Thanks to Southern OR Worker Ross for taking the lead in
the planning of this event with support from the team. The
programme looks like it will provide an interesting and
enjoyable experience for all, including workshop
discussions, outside activities and the NZ premiere of the HFNZ CEO Sue Ellis attended the Buffalo Lodge National Conference
movie Bombardier Blood. For more info, see the piece later to accept a generous donation
in Bloodline.
4 Bloodline July 2020
WFH Virtual Summit
On 14 – 19 June 2020 the World Federation of Hemophilia WFH EDUCATIONAL: UNCERTAINTY IN AN ERA
(WFH) ran their Virtual Summit. This online event replaced OF TRANSFORMATIVE THERAPY FOR
the World Congress, which had been planned for Kuala HEMOPHILIA: ADDRESSING THE UNKNOWNS
Lumpur. Unfortunately, the live event had to be cancelled
due to the worldwide Covid-19 situation. Instead, WFH put Hemophilia is at the dawn of a new era in therapeutic
together a fantastic online summit, featuring experts from management, one that can generate greater protection from
around the world. bleeding and a functional cure in some individuals. The
use of gene transfer is emerging as an effective long-term
HFNZ President Deon York played a big part in getting the treatment for a variety of diseases. While clinical progress
Virtual Summit up and running. He was part of the team who has been definitive, many questions remain unanswered
organised the multidisciplinary programme, as well as being a as pre-licensure phase 3 clinical trials are underway. These
speaker. We also had HFNZ Youth delegate Lauren Phillips on unanswered questions translate into a state of uncertainty
one of the speaking panels. It’s fantastic to see HFNZ people about the known unknowns and unknown unknowns
performing on the world stage. intrinsic to any new therapeutic platform. This pre-summit
session will examine the evolution gene therapy while
One of the advantages of running an event like this online addressing the unknowns that need to be answered.
is that you can keep the sessions available after the live
presentation has finished. This is particularly useful for us SPEAKERS
down here in NZ, as the sessions all started between 1am and Glenn Pierce - WFH
4am local time. Now you can access nearly every session from Megan Del Grosso - WFH
the comfort of your own home, at a time that suits you. WFH GUIDELINES FOR THE MANAGEMENT OF
HEMOPHILIA
Once you’re registered, you’ll be directed to the login page,
and from there to the Virtual Summit website. This WFH session highlights what is new in the hot-off-
the-press, 2020 WFH Guidelines for the Management of
The quickest way to find all the different sessions is to hover
Hemophilia. Informed by the best available evidence and
over the Sessions tab, and select Personal Agenda. Any
supplemented with expert opinion and patient preference, 4
session that has On Demand next to it is available to stream
key chapters of the WFH treatment guidelines will be featured
right now.
in this session: hemostatic agents; prophylaxis; inhibitors; and
Here’s a summary of all the sessions available, in the order treatment of specific hemorrhages. Come hear as the experts
they appear on the site: present updated recommendations and guidance!
SPEAKERS
Glenn Pierce - Vice-President Medical, World Federation
of Hemophilia
To access the sessions, first you need to register. Just go here:
https://onlinexperiences.com/scripts/Server.nxp?LASCmd=L:0&AI=1&InitialDisplay=1&ShowKey=94278&ClientBrowser=0
and hit the Register Now button.
Bloodline July 2020 5
Cedric Hermans - Hematologist, UCL Saint Luc SPEAKERS
Alok Srivastava - Professor of Medicine, Department of Glenn Pierce - Vice-President Medical, World Federation of
Haemotology, Christian Medical College Hemophilia
Steve Pipe - Professor of Pediatrics and Pathology, Steve Pipe - Professor of Pediatrics and Pathology,
University of Michigan University of Michigan
Manuel Carcao - Co-Director, Hemophilia Clinic, Hospital Luigi Naldini - Director, San Raffaele Telethon Institute for
for Sick Children Gene Therapy
Margaret Ragni - Tenured Professor of Medicine and MULTIDISCIPLINARY PLENARY: WOMEN
Clinical and Translational Science, Department of Medicine, AGEING GRACEFULLY
Division of Hematology/ Oncology, University of Pittsburgh
Johnny Mahlangu - Professor of Haematology, University This plenary, with special emphasis on women and those
of the Witwatersrand and NHLS who care for them, aims to identify the issues that women
with bleeding disorders may face as they age; this may
WFH GENE THERAPY AND WORLD BLEEDING include arthropathy associated with joint bleeds, increased
DISORDERS REGISTRIES fracture risk due to osteoporosis, gynaecological malignancy
Two key WFH data programs are highlighted during this and the need for surgical intervention for cardiac disease.
session: The WFH Gene Therapy Registry (GTR) and the This remarkable panel of experts including, Dr. Michelle
WFH World Bleeding Disorders Registry (WBDR). With gene Lavin, Prof. Rezan Kadir, Dr. Jameela Sathar and Latifa
therapy for hemophilia on the horizon, there is an urgent need Lamhene addresses womens’ long-term health issues and
to develop a global database to monitor the safety and efficacy improve screening, as well as answer any questions from the
of all patients who receive gene therapy. The WFH Gene virtual audience.
Therapy Registry is being developed to fill this need. This SPEAKERS
session focuses on the core data set of this registry and the Kate Khair - Director of Research, Haemnet
global implementation plan. Additionally, a 2-year update of Michelle Lavin - Clinical Research Fellow, Irish Centre for
the WBDR is provided, along with an exciting announcement Vascular Biology at the Royal College of Surgeons in Ireland
planned for 2020. Rezan Abdul-Kadir - Consultant Gynaecologist, Royal Free
SPEAKERS London NHS Foundation Trust
Barbara Konkle - Associate Chief Scientific Officer, WFH Latifa Lamhene - Association Algérienne des Hémophiles
Alfonso Iorio - Professor, McMaster University Jameela Sathar - Senior Consultant - Haemotology,
Catherine Lambert - Cliniques Universitaires Saint-Luc, Ampang Hospital
Haemophilia Centre DENTAL TRACK: NEW TECHNOLOGIES,
OPENING PLENARY PSYCHOSOCIAL ASPECTS, AND
Please join us for the official opening of the WFH Virtual INTERDISCIPLINARY DENTAL CARE
Summit: Connecting the Global Bleeding Disorders The Dental Committee is proud to present the dental track at
Community. The WFH President, Mr. Alain Weill, presents the WFH Virtual Summit with a truly global representation of
welcoming remarks, along with the WFH VP (Medical), Dr. presenters, speaking on a broad range of topics related to the
Glenn Pierce, and the WFH’s CEO, Mr. Alain Baumann, oral health of individuals with inherited bleeding disorders.
launching the first and exciting new initiative in bringing our SPEAKERS
bleeding disorders community together virtually. Lochana Nanayakkara - Part time Consultant, Restorative
SPEAKERS Dentistry at The Royal London Dental Hospital
Alain Weill - President, WFH Rebecca Schaffer - Adjunct Professor, A.T. Still University
Glenn Pierce - Vice-President, Medical, WFH Arizona School of Dentistry & Oral Health (ATSU-ASDOH)
Alain Baumann - Chief Executive Officer, WFH Zikra Alkhayal - Consultant Pediatric Dentist, King Faisal
MEDICAL PLENARY: GENE THERAPY Specialist Hospital & Research Center
Laura Olan - Professor, Universidad Juárez Autónoma de
The promise of gene therapy is a single treatment (‘one and Tabasco
done’) that leads to steady-state expression of endogenous Elvira Correa - Chair of the Oral Medicine/Dental
factor VIII or factor IX sufficient to achieve a functional cure Ambulatory, Hematology and Blood Transfusion Center,
(free of recurrent haemophilic bleeding) if not normalized University of Campinas
haemostasis. Dr. Steve Pipe explores that, although we
currently stand on the threshold of this achievement, REDEFINING PROPHYLAXIS IN THE
delivering on this promise will require broad-based multi- MODERN ERA
stakeholder preparation with a focused emphasis on The currently evolving novel therapies for the management
education, approval of safe and effective therapies, removal of of hemophilia has ushered in a new era characterized by
barriers to access and excellence in clinical delivery. improved prophylaxis targets and outcomes. In this session, the
Dr. Luigi Naldini continues to investigate new strategies speakers redefine prophylaxis in the modern era by revisiting
to overcome the major hurdles to safe and effective gene its definition, presenting data to support higher trough levels
transfer, and translate them into new therapeutic strategies for to achieve through prophylaxis, and introducing steady-state
genetic disease and cancer. This has generated new insights hemostasis as a possible new target for prophylaxis. The
into hematopoietic stem cell function, and the induction of benefits of low-dose prophylaxis is also discussed.
immunological tolerance and tumor angiogenesis. Naldini SPEAKERS
presents his data regarding the further development of Johnny Mahlangu - Professor of Haematology, University
lentiviral vectors as a systemic gene delivery vehicle to of the Witwatersrand and NHLS
complement the reach of AAV vectors. Victor Blanchette - Hematologist, Hospital for Sick Children
6 Bloodline July 2020
Robert Klamroth - Director, Comprehensive Care SPEAKERS
Haemophilia Treatment Center & the Haemostasis & Andra James - Maternal-Fetal Medicine Specialist, Duke
Thrombosis UnitVivantes Klinikum im Friedrichshain University Medical Center
Emna Gouider - Hematologist, Hopital Aziza Othmana Mandy Yap - MBBS(IMU), MRCP(UK), Clinical
VWD, A CLOSER LOOK Haematologist of Ampang Hospital, Ministry of Health
Malaysia
Learn more about VWD and the perception of the condition Mario von Depka Prondzinski - Director, Werlhof-
throughout the world, how the WFH is working on it, Institute for Haemostasis & Thrombosis
psychosocial issues, the differences between men and women, Lubna Zafar - Consultant Haematologist, Fauji Foundation
and the treatment options available. Hospital, Rawalpindi
The session aims to answer the following questions: how is Robert Sidonio - Pediatric Hematologist/Oncologist, Emory
VWD perceived worldwide? What is the perspective of those University
who suffer it? Is there a difference between men and women? Roseline d’Oiron - Clinician Investigator, Centre Hospitalier
SPEAKERS de Bictre
Dawn Rotellini - Chief Operating Officer, National
TRM-201 (ROFECOXIB) FOR THE TREATMENT OF
Hemophilia Foundation
HEMOPHILIC ARTHROPATHY
Paula James - Professor, Queen’s University
Jeannette Cesta - VWD Connect Foundation Inc. Tremeau is focused on developing non-opioid pain therapy for
Cody Kester - Board Member, Hemophilia Foundation of patients with significant unmet need.
Arkansas In this session you will learn more about our lead product,
Baiba Ziemele - President, Latvia Hemophilia Society TRM-201 (rofecoxib), a COX-2 selective NSAID and a non-
opioid analgesic. We also discuss our upcoming Phase III trial
Dental track: Dental Management and Treatment of TRM-201 for Hemophilic Arthropathy, which we plan to
The Dental Committee is proud to present the dental track at initiate later this year.
the WFH Virtual Summit with a truly global representation of
SPEAKER
presenters, speaking on a broad range of topics related to the
Judith Boice - Tremeau Pharmaceuticals
oral health of individuals with inherited bleeding disorders.
SPEAKERS ELEVATING PATIENT CARE IN HAEMOPHILIA –
Lochana Nanayakkara - Part-time Consultant, Restorative WHAT DO WE NEED TO KNOW AND WHY?
Dentistry at The Royal London Dental Hospital - BY ROCHE
Norjehan Yahaya - Specialist in Special Needs Dentistry Our first symposium provides perspectives on what’s involved
(SND), Specialty Needs Dentistry Unit, Kuala Lumpur in elevating patient care in haemophilia and why improving
Hospital the level of care is necessary for the haemophilia community.
Miryam Parreira - Dentist - Specialist in Surgery and We focus on the changing conversations around patient
Traumatology Bucomaxilofacial, Fundación de la Hemofilia focused outcomes, the risk–benefit assessments of care, and
Daniel Sundaresan - Specialist, Special Needs Dentistry, recognising how stakeholders can facilitate joint decision
Fiona Stanley Hospital making with patients. The symposium is comprised of pre-
Mathew Lim - Alfred Health recorded presentations culminating in an open panel discussion
INHIBITORS - THINK DIFFERENTLY illustrated with real-life examples of what can be achieved.
SPEAKERS
The development of inhibitory antibodies to therapeutic
Mark Skinner - President, Institute for Policy
factor VIII (FVIII) in up to 40% of severe haemophilia
Advancement Ltd.
A (HA) patients is the major complication in treatment/
Declan Noone - President, European Haemophlia
prevention of hemorrhages. The reasons some HA patients
Consortium
develop inhibitors while others do not remain unclear. This
Johnny Mahlangu - Professor of Haematology, University
session summarises our understanding of anti-FVIII immune
of the Witwatersrand and NHLS
responses, the roles of T cells, both effector and regulatory,
Jameela Sathar - Senior Consultant - Haemotology,
and generally discusses the interplay between FVIII and the
Ampang Hospital
immune system, both in the case of replacement therapy and
in gene therapy. INVESTIGATIONAL AAV GENE THERAPY IN
SPEAKERS HEMOPHILIA: WOULD YOU BE READY?
Sébastien Lacroix-Desmazes - Team Leader, Centre de - BY BIOMARIN
Recherches des Cordeliers, Universit Pierre et Marie Curie BioMarin organized a symposium at the WFH 2020
Kathleen Pratt - Associate Professor, Uniformed Services Virtual Summit to foster discussion on potential clinical
University of the Health Sciences considerations for Adeno-Associated Virus (AAV)-based
Valder R. Arruda - Associate Professor of Pediatrics, gene therapy for hemophilia, currently under clinical trial
University of Pennsylvania School of Medicine investigation.
MEDICAL FREE PAPERS: VWD & WOMEN’S The objectives of the symposium are to: discuss the science
ISSUES behind AAV-based gene therapy and potential practical
Five of the best scoring abstract authors in category of the considerations (including eligibility and follow-up after
VWD and Women’s issues have been selected to present their treatment) and to give insight into the perspectives of a
research. Each presenter is available for a Question & Answer patient advocate and a nurse through a live interview. Q&A is
discussion at the end of the presentations. included at the end.
Bloodline July 2020 7
SPEAKERS by regulators for post marketing surveillance and additional
Wing Yen Wong - Vice President Clinical Sciences and data needed to enhance our understanding of gene therapy
Global Medical Affairs, BioMarin Pharmaceutical Inc. outcomes and their impact on the lives of people with
Glenn Pierce - Vice-President Medical, World Federation of haemophilia. The aim of this session is to describe efforts
Hemophilia, Independent Consultant, La Jolla, CA, USA underway by the American Thrombosis and Hemostasis
Debra Pollard - Lead Nurse, Specialist Practice, Network and the World Federation of Hemophilia to collect
Haemophilia, Katharine Dormandy Haemophilia & long-term harmonized data and considerations of the
Thrombosis Centre, Royal Free Hospital, London, UK European and US regulatory agencies, which will inform
Matthew Jajeh - Active Member, Hemophilia Foundation of ongoing data collection.
Northern California, CA, USA SPEAKERS
MEDICAL PLENARY: DIAGNOSIS Barbara Konkle - Associate Chief Scientific Officer,
Bloodworks Northwest
The combination of high BAT score followed by NGS-based
Michael Recht - Chief Science Officer, American Thrombosis
genetic diagnosis could be the new paradigm for the primary
and Hemostasis Network
diagnosis of IBDs. This needs to be followed by confirmation
Caroline Voltz - Product Lead, Oncology, Haematology &
with functional haemostasis tests, as required. This approach
Diagnostics Office, European Medicines Agency
will increase rates of detection of the known common
Peter Marks - Director, Center for Biologics Evaluation and
disorders many folds and reduce the burden on these families
Research United States Food and Drug Administration
towards accessing facilities for accurate diagnosis and
appropriate treatment based on that. EMERGING FROM COVID - DEFINING A NEW
SPEAKERS NORMAL
Steve Kitchen - Clinical Scientist, Sheffield Teaching This session explores what patients are most concerned about
Hospitals and what we should expect living in a Covid-19 world from
Alok Srivastava - Professor of Medicine, Department of the perspective of multidisciplinary healthcare professionals.
Haemotology, Christian Medical College SPEAKERS
Pierre Toulon - Hemostasis and Thrombosis Laboratory, Declan Noone - President, European Haemophlia
Pasteur University Hospital Consortium
MULTIDISCIPLINARY PLENARY: Len Valentino - Chief Executive Officer, National
QUALITY OF LIFE Hemophilia Foundation
Kate Khair - Director of Research, Haemnet
This plenary seeks to
Ed Kuebler - Advocacy & Leadership Programs Manager,
explore and explain
Global Blood Disorder Foundation
the role of pain in our
lives. While pain is not LABORATORY SCIENCES TRACK:
something we generally LABORATORY ISSUES AND TESTING
wish to experience, it has The advent of novel nonfactor therapies poses a new level of
an essential role in making difficulty on the laboratory monitoring of these patients. It
us who we are. Lorimer is important to understand the correct assays and the mode
Moseley will explore of action of these nonfactor agents. The WFH Laboratory
the new understanding Sciences Committee addresses these issues as well as
of pain, and how that monitoring replacement therapy.
understanding can change
SPEAKERS
one’s perspectives of
Steve Kitchen - Clinical Scientist, Sheffield Teaching
dealing with pain. “Sleep:
Hospitals
Why we need it” gives an
Stefan Tiefenbacher - Vice-President, Technical Director,
overview of what sleep is,
Colorado Coagulation
how it happens and why
HFNZ President Deon York spoke at Anna Lowe - Advanced Biomedical Scientist, Scientific Lead
‘good’ sleep is so important
the summit and helped organise the Haemophilia Programmes, UK NEQAD BC
to being healthy and how multidisciplinary programme. Annette Bowyer - Senior Biomedical Scientist, Sheffield
pain influences sleep
Teaching Hospital NHS Foundation Trust
patterns. Dr. Bajan Singh also offers ideas on how to promote
Sukesh Nair - Professor, Christian Medical College and
good habits for better sleep.
Hospital Vellore
SPEAKERS
LABORATORY SCIENCES TRACK:
Deon York - Board Member, World Federation of Hemophilia
REPLACEMENT THERAPY AND TESTING
Lorimer Moseley - Clinical Neurosciences, School of Health
Sciences, University of South Australia For decades the clinical laboratory has predominantly
Bhajan Singh - Clinical Professor, University of performed either manual or automated one stage (OS)
Western Australia factor VIII assays as a reliable means for monitoring
factor VIII (F8) replacement therapy in hemophilia. The
CRITICAL NEED FOR HARMONIZED POST-
emergence of chromogenic factor VIII testing illuminated
MARKETING SURVEILLANCE IN GENE THERAPY
both discrepancies between testing methods and potential
With regulatory approval of the first gene therapy product issues associated with product potency assessment. However,
expected as early as mid-2020, there is an urgent need for a the creation of B-domain deleted recombinant (BDDR) F8
mechanism to collect long-term data on safety and variability replacement product initiated the first real challenge for
and durability of efficacy. There will be elements required clinical laboratories to accurately monitor the advances in
8 Bloodline July 2020
hemophilia replacement therapy. Currently, the escalation of between disease severity and bleeding phenotype is reviewed.
hemophilia therapeutic advances, including extended half- Defining sub-groups in the hemophilia B population who
life products (EHL), gene therapy and non-factor bridging are currently undertreated will shed a light on the needs of
therapies, have generated significant improvements for the whole community. Additionally, the importance of high
hemophilia management. However, the clinical laboratory circulating FIX trough activity levels is discussed. Finally,
may not possess the ideal laboratory test(s) for monitoring utilizing digital tools based on robust pharmacokinetic
these new-age therapies, and it may be that a single F8 testing principles has the potential to guide treatment strategies.
platform may not be adequate. A description of the EHL New electronic tools will allow precision dosing that address
products and the optimal or acceptable laboratory F8 testing the goals of both health care providers and patients to
platforms is discussed. optimize outcomes.
SPEAKERS SPEAKERS
Steve Kitchen - Clinical Scientist, Sheffield Teaching Flora Peyvandi - Professor of Internal Medicine, Angelo
Hospitals Bianchi Bonomi hemophilia and Thrombosis Center,
Emmanuel Favaloro - Principal Hospital Scientist, Institute Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico,
of Clinical Pathology & Medical Research Universit degli Studi di Milano
Robert Gosselin - Clinical Laboratory Scientist, University David Lillicrap - Professor, Department of Pathology &
of California at Davis Hemostasis & Thrombosis Center Molecular Medicine,Richardson Laboratory,
Geoffrey Kershaw - Senior Hospital Scientist, Institute of Queen’s University
Haemotology, Royal Prince Alfred Hospital Cedric Hermans - Hematologist, UCL Saint Luc
SUBSTITUTION THERAPY Alfonso Iorio - Professor, McMaster University
Despite significant paradigm shifts in the medical WFH EDUCATIONAL: TREATMENT CHOICE IN
management of hemophilia A with inhibitors and the AN ERA OF CHANGE
expanding use of novel non-factor therapies, FVIII-based This session explores the patient and provider perceptions on
immune tolerance induction (ITI) is still the only effective treatment choices: how does each make a choice and process
means to inhibitor eradication. With the recently introduced the risk-benefit analysis.
Atlanta protocol, emicizumab prophylaxis is given in SPEAKERS
combination with 50-100 IU/kg FVIII three times a week. Glenn Pierce - Vice-President Medical, World Federation
The session explores the impact of emicizumab prophylaxis of Hemophilia
for non-inhibitor patients and examine the current status of Johnny Mahlangu - Professor of Haematology, University of
clinical studies regarding emicizumab prophylaxis. the Witwatersrand and NHLS
SPEAKERS David Page - National Director of Health Policy, Canadian
Alok Srivastava - Professor of Medicine, Department of Hemophilia Society
Haemotology, Christian Medical College Brian O’Mahony - Irish Haemophilia Society
Midori Shima - Professor of the Department of Pediatrics, PHYSICAL ACTIVITY FOR JOINT HEALTH AND
Nara Medical University WELLNESS
Robert Sidonio - Pediatric Hematologist/Oncologist, Emory
University Exercise joint and muscle can reduce spontaneous bleeding
Gallia Levy - Vice President and Global Head of Rare Blood in the long term. Many patients focus only getting enough
Disorders, Roche Pharmaceuticals treatment products, which is impossible for developing
Steve Pipe - Professor of Pediatrics and Pathology, countries. Musculoskeletal care is one solution. This
University of Michigan session aims to demonstrate easy and practical methods in
musculoskeletal care from orthopedist, physiotherapist and
WFH EDUCATIONAL: PATIENT FOCUS: ASH, patient perspectives, including bone health without surgery,
ISTH, NHF, WFH GUIDELINES ON THE rehabilitation vs wellness, best patient practices.
DIAGNOSIS AND MANAGEMENT OF VWD
SPEAKERS
Join us for this patient-focused preview of two of the Paul McLaughlin - Haemophilia - Physiotherapist, Royal
recommendations resulting from the international Free Hospital
collaboration to develop guidelines for the diagnosis and Adolfo Llinas - Orthopedic Surgeon, Fundación Santa Fe de
management of von Willebrand disease (VWD), ahead of Bogotá
publication later this year. Learn why international clinical Pamela Narayan - Executive Committee Member, Medical
guidelines are particularly important to this community, Advisory Board Member, Hemophilia Federation of India (HFI)
how they were developed, and the important contributions of Chavez Edgecombe - Bahamas Hemophilia Foundation
various stakeholders throughout this process.
WFH EDUCATIONAL: BETTER DATA, BETTER
SPEAKERS PATIENT OUTCOMES: 20 YEARS OF DATA
Paula James - Professor, Queen’s University COLLECTION
Nathan Connell - Chief of Hematology, Brigham and
Women’s Faulkner Hospital This workshop is intended for healthcare professionals
Mark Skinner - President, Institute for Policy engaged in clinical research and patient leaders and
Advancement Ltd. organizations who have an active interest in using health data
in their work.
HEMOPHILIA B: CARE ACROSS THE SPECTRUM
- BY CSL BEHRING SPEAKERS
Part 1: Data Collection at WFH - Welcome
This symposium highlights the paradigm shift in hemophilia Ellia Tootoonchian - Data Specialist, World Federation
B treatment and management. Specifically, the discordance of Hemophilia
Bloodline July 2020 9
Donna Coffin - Director, Research & Public Policy, World despite a large variability in phenotype expression, the joint
Federation of Hemophilia health and the way to secure both maternal and fetal bleeding
Mark Skinner - President, Institute for Policy risks during pregnancy, delivery and the post-partum period.
Advancement Ltd. SPEAKERS
Part 2: 20th Anniversary of the Annual Global Survey Roseline d’Oiron - Clinician Investigator, Centre Hospitalier
Ellia Tootoonchian - Data Specialist, World Federation of de Bictre
Hemophilia Sarah O’Brien - Pediatric Hematologist-Oncologist,
Alfonso Iorio - Professor, McMaster University Nationwide Children’s Hospital
Part 3: Improvements/New Approaches in Metrics Andra James - Maternal-Fetal Medicine Specialist, Duke
Ellia Tootoonchian - Data Specialist, World Federation University Medical Center
of Hemophilia
Jeff Stonebraker - Associate Professor, North Carolina State EXPLORING THE WFH ELEARNING PLATFORM:
University’s Poole College of Management OVER 700 FREE RESOURCES AT YOUR
Emna Gouider - Hematologist, Hopital Aziza Othmana FINGERTIPS
MUSCULOSKELETAL TRACK: SYNOVITIS AND A Guided Tour of the WFH eLearning Platform: Join WFH
SYNOVECTOMY Educational Materials Manager, Fiona Robinson, as she
shows you how to get the most out of the >700 resources
This session addresses the current understanding of synovitis and 24 languages on the WFH eLearning Platform. Together
and its treatment options with specific emphasis on chemical you will navigate the bleeding disorders themed eLearning
and radioactive synovectomy, surgical options, rehabilitation Centres, explore the enhanced Featured Tools including many
and non-surgical management. of your favourite well-loved WFH publications, and master
SPEAKERS the searching and sharing functions of the Platform.
Sébastien Lobet - Physiotherapist and Researcher, Clinique SPEAKER
Universitaire Saint-Luc Fiona Robinson - Education Materials Manager, World
Eline van Bergen - MD PhD Candidate, Van Creveldkliniek Federation of Hemophilia
Sylvia Thomas - Hematologist, Federal University of
Rio de Janeiro HEALTHY LIVING
Mauricio Silva - Medical Director, Orthopaedic Institute This session discusses the strategies for healthy living in all
for Children developmental stages and share advice on nutrition, fitness,
WHAT DOES QUALITY OF LIFE MEAN TO YOU? getting a good night’s sleep, building self-confidence/self-
LIVING WITH (AN INHERITED) BLEEDING esteem, healthy aging, and sexuality.
DISORDER IN A CHANGING WORLD SPEAKERS
Sabrina Farina - Social Worker, Gulf States Hemophilia &
Quality of life for patients
Thrombophilia Center
is a topic often discussed
Hariyati Shahrima Abdul Majid - Chief Health
among clinicians. This
Psychologist, Naluri Life
session focuses on what
Richa Mohan - Consultant Clinical Psychologist & Director,
quality of life means
Empowering Minds Society for Research and Development
from the perspective
Mina Nguyen-Driver - Associate Professor, Oregon Health
of a person living with
and Science University (OHSU)/ The Hemophilia Center
a bleeding disorder.
of Oregon
This session asks you
to consider: what does MUSCULOSKELETAL TRACK: HOT TOPIC
quality of life mean to you, DEBATES
especially during This session features three controversial topics debated by
a pandemic? experts in their field: POCUS image capture by patients; Ankle
SPEAKERS Arthrodesis or Arthroplasty; HJHS vs. functional measurement.
Deon York - Board SPEAKERS
Member, World HFNZ Youth Delegate Lauren Phillips Adolfo Llinas - Orthopedic Surgeon, Fundación Santa Fe de
Federation of Hemophilia spoke at the Summit. Bogotá
Nathalie Roussel - Hortensia de la Corte Rodriguez - University Hospital La
Assistant Professor, University of Antwerp Paz, Madrid, Spain
Cathy Harrison - Advance Nurse Practitioner, Sheffield Annette von Drygalski - Director, Hemophilia & Thrombosis
Haemostasis & Thrombosis Centre Treatment Center, University of California San Diego
Lauren Phillips - Youth Committee Member, Haemophilia Mauricio Silva - Medical Director, Orthopaedic Institute
Foundation of New Zealand for Children
Randall Curtis - Hemophilia Utilization Group Study (HUGS) David Dalstrom - Orthopedic Surgeon, UC San Diego Health
WOMEN AND GIRLS WITH HEMOPHILIA Janaina Bosso S. Ricciardi - Physiotherapist, Hemocentro
UNICAMP
The aim of this session is to address issues on women and
David Stephensen - Physiotherapist, Kent Haemophilia
girls with hemophilia (WGWH) integrating here female
Centre / Royal London Hospital
carriers with low levels of FVIII or FIX and/or bleeding
experiences. The key points that will be covered are the VWD
importance of early diagnosis during childhood of affected Von Willebrand disease (VWD) is the most common inherited
girls, the management and treatment of the bleeding risk bleeding disorder. This session provides an overview of VWD
10 Bloodline July 2020diagnosis and treatment, special considerations in treating WFH EDUCATIONAL: DIGITAL TECHNOLOGIES
women with VWD, and current genomic approaches to AND PERSONALIZED CARE TO MANAGE
VWD. Diagnosis of VWD begins with the clinical assessment JOINT HEALTH
for the bleeding phenotype, and then made by laboratory This session provides an overview of the cellular effects of
investigation. Interdisciplinary management of childbirth and bleeding in a joint and discusses the importance of individual
prophylaxis in the postpartum period are needed to reduce monitoring plans for the preservation of joint health in
the risk of postpartum haemorrhage. Genomic approaches patients with haemophilia. It also conveys the concept that
to VWD can inform VWD diagnosis, treatment, test assay every bleed matters and that treatment should be without
selection, reproductive planning, and family counseling. Next delay and provided examples for how digital tools can enable
generation sequencing is rapidly being adopted to provide education and collaboration with patients and the wider
more comprehensive VWF sequence information for patients multi-disciplinary team to strive for zero bleeds.
with known or suspected VWD.
SPEAKERS
SPEAKERS Angela Forsyth - Director, REBUILD Program, Diplomat
Jill Johnsen - Associate Member, Bloodworks Northwest Specialty Infusion Group
Research Institute Len Valentino - Chief Executive Officer, National
Michael Laffan - Professor of Haemostasis and Thrombosis, Hemophilia Foundation
Centre for Haematology, Department of Medicine Deon York - Board Member, World Federation of Hemophilia
Imperial College Alfonso Iorio - Professor, McMaster University
Jameela Sathar - Senior Consultant - Haemotology,
Ampang Hospital MUSCOLOSKELETAL FREE PAPERS:
ORTHOPEDIC ISSUES
ELEVATING PATIENT CARE – FIND OUT HOW TO
CREATE YOUR ‘NEW NORMAL’ - BY ROCHE Four researchers will present their findings in the
arthroplasties.
Our second symposium provides practical examples of
SPEAKERS
how to apply the theory from symposium #1 in improving
Adolfo Llinas - Orthopedic Surgeon, Fundación Santa Fe
patient care and the realistic expectations of the community.
de Bogotá
Our expert panel engages in discussion focused on the
Cedric Hermans - Hematologist, UCL Saint Luc
evolving needs of patients, effective collaboration between all
Jenny Y. Zhou
stakeholders, and the importance of shared decision making
Ozgür Mert Bakan
when selecting treatments. Throughout the programme the
Kumiko Ono
panel highlights the tangible opportunities that exist for
enabling patients to improve the level of care they receive. WFH EDUCATIONAL: CONNECTING THE
SPEAKERS GLOBAL COMMUNITY THROUGH
Mark Skinner - President, Institute for Policy PARTNERSHIPS: WFH TWINNING PROGRAM
Advancement Ltd. SPEAKERS
Declan Noone - Professor of Haematology, University of the Deniece Chevannes - Director of Health Promotion and
Witwatersrand and NHLS Evaluation, Hemophilia of Georgia
Johnny Mahlangu - Senior Consultant - Haemotology, Jayson Stoffman
Ampang Hospital Niamh O’Connell
Jameela Sathar - Senior Consultant - Haemotology, John Sarmenta
Ampang Hospital Luisa Durante - Director, Programs and Education, World
MANAGEMENT OF HEMOPHILIA IN TIMES OF Federation of Hemophilia
UNCERTAINTY: ADAPT, INNOVATE, EVOLVE GENE THERAPY: THE UNFOLDING STORY
-BY PFIZER
We are still learning about Gene Therapy. Each patient
We are living in an age of uncertainty due to the current experience is different, and gene therapy is not for everyone
global crisis. There is an increased need to adapt the way – either by choice or by exclusion because of the antibodies.
we work, develop innovate new therapies and to evolve our What can we expect if we ARE eligible – what is it like to
approaches to the delivery of care. participate in the gene therapy trials? What is it like to be
Now more than ever, in an era of transformative medicine, it a patient once you have gone through the treatment? What
is fundamental that information is shared with patients and if it does not work for me (one and done) – can I try other
patient communities, in order to support shared decision- future therapies?
making based on open, and transparent education.
SPEAKERS
Through a basic overview of the science underpinning gene
Debra Pollard - Lead Nurse, Specialist Practice,
therapy, this session explores the evolution of the patient-
Haemophilia, Katharine Dormandy Haemophilia &
physician relationship.
Thrombosis Centre
SPEAKERS John Pasi - Professor of Haemostasis and Thrombosis, Barts
Thierry VandenDriessche - Professor, Vrije and the London, Queen Mary, University of London
Universiteit Brussel David Page - National Director of Health Policy, Canadian
Wolfgang Miesbach - Professor, University Hospital Hemophilia Society
Frankfurt, Haemophilia Centre / Department of Garrett Hayes - National Youth Leadership Institute (NYLI)
Haemostaseology, Medical Clinic 2 / Institute of Representative, National Hemophilia Foundation
Transfusion Medicine Rob Schroeder - Senior Executive Rep, Oncology-Prostate,
Laurence Woollard - Director, On The Pulse Consultancy Astellas
Bloodline July 2020 11Enrique David Preza Hernandez - Federación de Tien-Truong Dang - Ozlem Turan
Hemofilia de la República Mexicana Glaivy Batsuli - Assistant Professor of Pediatrics,
PSYCHOSOCIAL TRACK: TRAINING AND Emory University
CAREER GUIDANCE FOR YOUTH WITH PSYCHOSOCIAL TRACK: NEW
BLEEDING DISORDERS COMMUNICATION STRATEGIES FOR THE
This session explores the ways in which social workers can BLEEDING DISORDERS COMMUNITY
assist youth with bleeding disorders explore careers and This session explores challenges that affect communication
develop skills that will prepare them for employment. The between HTC team members and the patients and families
presentation also addresses the economic disparity in countries whom they serve. As providers, we know what we want
around the world, which significantly impacts the opportunities our patients to understand, but sometimes, it seems that
that are available for youth with bleeding disorders, discusses we just cannot get our message across effectively. These
how the bleeding disorders community has addressed this experiences can be frustrating and, in some cases, can result
issue, and suggests ways that we can expand our efforts. in strained relationships between patients and providers. In
SPEAKERS this session, we discuss common communication barriers
Susan Cutter - Social Worker, Hospital of the University of and ask participants to identify situations that they personally
Pennsylvania, Penn Comprehensive Hemophilia Center have found difficult or frustrating. By the end of the session,
Claude Bartholomew - Social Worker, Providence Health participants specify one or two communication skills that
Care (Adult Hemophilia Program) they want to utilize or improve and identify resources to assist
Annie Bole - Lectures and Programming Assistant, Faith in this process.
Justice Institute SPEAKERS
Tony Roberts - Registered Educational Psychologist, South Ed Kuebler - Advocacy & Leadership Programs Manager,
African Haemophilia Foundation Global Blood Disorder Foundation
Santosh Manivannan - Chairman, Youth Group of Silvina Grana - Psychologist, Fundación de la Hemofilia de
Hemophilia Federation (India) la Argentina
MANAGEMENT OF COMORBIDITIES IN Diane Standish - Faculty, Partners in Bleeding Disorders
BLEEDING DISORDERS Education Program of the Indiana Hemophilia and
Thrombosis Center
With the introduction of clotting factor concentrates in the David Silva - Spanish Federation of Hemophilia
early 1970s, significant improvements in quality of life and Patricia Cabré - Therapeutic Pedagogue, Catalan
life expectancy of persons with hemophilia (PWH) were Association of Hemophilia
realized. Unfortunately, as a result of transmission of HIV and
hepatitis C virus (HCV) by contaminated concentrates in the MEDICAL FREE PAPERS: LATE-BREAKING 2
1980s, many of these gains were lost. Now with four decades Four non-scientific abstracts were selected to present on
of PWH unexposed to contaminated factor products and research in registries, psychosocial, prevalence and
current treatments capable of suppressing and eliminating tele-dentistry.
HIV and HCV, respectively, the survival rate is once again SPEAKERS
increasing. In addition to the usual comorbidities associated Saliou Diop - Board Member, World Federation
with advanced age in the general population, several specific of Hemophilia
issues occur in patients with bleeding disorders. This session Ampaiwan Chuansumrit - Director, International
explores the incidence and management of the comorbidities Hemophilia Training Center - Bangkok
of the ageing PWH with a focus on cardiovascular disease Silvia Riva - Senior Lecturer in Psychology,
and osteoporosis. St. Mary’s University
SPEAKERS Ahmad Tarawah - President, Middle East and North Africa
Mike Makris - Professor of Haemostasis and Thrombosis, Hematology League
Sheffield Haemophilia and Thrombosis Centre Royal Alison Dougall - Academic and Clinical Dental Consultant,
Hallamshire Hospital Dublin Dental Hospital
Pal André Holme - Professor, Senior Consultant, Oslo MULTIDISCIPLINARY FREE PAPERS: DATA
University Hospital COLLECTION AND PATIENT OUTCOMES
Christine Kempton - Associate Professor, Hematology &
Medical Oncology, Emory University Hospital Midtown Three speakers from the WFH Research & Public Policy
department present on three key projects: The World
MEDICAL FREE PAPERS: YOUNG RESEARCHERS Bleeding Disorders Registry; Data Quality; and the Annual
The top six abstracts from young researchers as selected by Global Survey.
the Abstract Review Committee are presented and compete SPEAKERS
for the Christine Lee Haemophilia Journal Award in this Mayss Naccache - Gene Therapy Program Manager, World
Young Researchers Free Paper Session. Federation of Hemophilia
SPEAKERS Emily Ayoub - Data & Research Manager, World Federation
Cedric Hermans - Hematologist, UCL Saint Luc of Hemophilia
Geertje Goedhart - Epidemiologist, HemoNED Foundation Toong Youttananukorn - Data & Research Coordinator,
Patricio Cesar Gatti - Assistant Professor, Surgery World Federation of Hemophilia
Department, University of Buenos Aires Ellia Tootoonchian - Data Specialist, World Federation
Sandeep Albert - Associate Professor, Department of of Hemophilia
Orthopaedics Unit-1, Christian Medical College and Johannes Oldenburg - President, German Society of
Hospital Vellore Thrombosis and Haemostasis Research
12 Bloodline July 2020GENERAL PLENARY: MANAGING COVID-19 AND Michelle Lavin - Clinical Research Fellow, Irish Centre for
BLEEDING DISORDERS Vascular Biology at the Royal College of Surgeons in Ireland
Since the outbreak of the coronavirus disease 2019 Tahira Zafar - Chairperson, Medical Advisory Board,
(COVID-19) there has been an unprecedented global public Pakistan Haemophila Patients Welfare Society
health challenge. This session - developed as an educational Robert Sidonio - Pediatric Hematologist/Oncologist, Emory
response for the global bleeding disorders community - University
presents four main topics: WFH EDUCATIONAL: TREATMENTS FOR
• Radoslaw Kaczmarek focuses on the pathophysiology of BLEEDING DISORDERS: CURRENT
COVID-19 ENVIRONMENT AND PIPELINE PRODUCTS
• Flora Peyvandi, MD, explains the clinical manifestations There are novel products newly arrived on the market (longer
of COVID-19 half-life, non-factor products) that are very different from
• Cedric Hermans, MD, provides practical advice on the traditional clotting factor concentrates which pose new
management of COVID-19 questions about safety and clinical use. Furthermore, gene
• Mark Skinner concludes the session with information on therapies may revolutionize hemophilia care. Finally, these
the safety and supply of plasma derived products during new technologies mean the global supply of all treatment
the pandemic. products continues to grow, opening up the possibility for
SPEAKERS greater access to treatment in under-served areas of the world.
Cedric Hermans - Hematologist, UCL Saint Luc SPEAKERS
Radoslaw Kaczmarek - Postdoctoral Research Associate, Radoslaw Kaczmarek - Postdoctoral Research Associate,
Hirszfeld Institute of Immunology Hirszfeld Institute of Immunology
Flora Peyvandi - Professor of Internal Medicine, Angelo Flora Peyvandi - Professor of Internal Medicine, Angelo
Bianchi Bonomi hemophilia and Thrombosis Center, Bianchi Bonomi hemophilia and Thrombosis Center,
Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico,
Universit degli Studi di Milano Universit degli Studi di Milano
Mark Skinner - President, Institute for Policy Dan Hart - Consultant Haematologist, The Royal London
Advancement Ltd. Hospital Haemophilia Centre
NURSES TRACK: UNMET AREAS OF EVIDENCE Johnny Mahlangu - Professor of Haematology, University
BASED NURSING IN BLEEDING DISORDER CARE of the Witwatersrand and NHLS
As the role of hemophilia nurses continues to expand globally, NURSES TRACK: EMERGING HEMOPHILIA
they are practicing at ever-more advanced levels, delivering NURSING SKILLS
totally nurse-led care. The sessions cover best evidence-based The session covers best evidence-based practices and looks
practices, and look at how the role of the nurse in delivering at how the role of the nurse in delivering care will change
care will change dramatically over the coming years. dramatically over the coming years.
SPEAKERS SPEAKERS
Kate Khair - Director of Research, Haemnet Kate Khair - Director of Research, Haemnet
Chris Guelcher - Pediatric Nurse Practitioner, Children’s Amber Federizo - Medical Director, Hemostasis &
National Medical Center Thrombosis Center of Nevada
Debra Pollard - Lead Nurse, Specialist Practice, Michelle Witkop - Head of Research, National Hemophilia
Haemophilia, Katharine Dormandy Haemophilia & Foundation
Thrombosis Centre Regina Butler - Clinical Manager, Childrens Hospital of
Kelly Tickle - Pediatric Nurse Practitioner, Childrens Philadelphia
Healthcare of Atlanta Cathy Harrison - Advance Nurse Practitioner, Sheffield
Mahmoud Abu-Riash - Senior Clinical Specialist, King Haemostasis & Thrombosis Centre
Faisal Specialist Hospital and Research Centre
WFH EDUCATIONAL: WFH GLOBAL INITIATIVE
WFH EDUCATIONAL: IMPACT OF CHALLENGES FOR BETTER CARE OF VWD
OF BLEEDING DISORDERS IN WOMEN AND GIRLS
VWD is the most common bleeding disorder yet it is greatly
The session highlights the challenges women and girls undiagnosed. It is challenging to diagnose particularly or
experience in getting proper diagnosis, care, and treatment developing countries. The Committee shares practical ways
for their bleeding disorder. What are the global and personal to diagnose that would work in all contexts around the world
impacts of having a bleeding disorder for women and girls? and educate the community on management.
The session addresses key issues such as reproductive
challenges, diagnostic dilemmas, and treatment options for SPEAKERS
heavy menstrual bleeding. The session also shares examples Margaret Ragni - Tenured Professor of Medicine and
of what actions are being taken to improve the lives of women Clinical and Translational Science, Department of Medicine,
and girls worldwide with bleeding disorders. The session Division of Hematology/ Oncology, University of Pittsburgh
includes a presentation on the impact of the COVID-19 Augusto Federici - Associate Professor of Hematology,
pandemic on women and girls with bleeding disorders. University of Milan
Roshni Kulkarni - Professor Emerita of Pediatrics and
SPEAKERS Human Development, Director, Michigan State University
Roshni Kulkarni - Professor Emerita of Pediatrics and Rezan Abdul-Khadir - Consultant Gynaecologist, Royal
Human Development, Director, Michigan State University Free London NHS Foundation Trust
Rezan Abdul-Kadir - Consultant Gynaecologist, Royal Free
London NHS Foundation Trust
Bloodline July 2020 13You’re invited to the 2020 HFNZ
Adult Weekend!
If you’re 25 or over, and you’ve been hanging out for some on Sunday afternoon. This is a great opportunity to really
HFNZ education and social time, then we have some great engage with the running of your foundation.
news for you!
The registration fee for the weekend, including flights and
Coming up in Christchurch on 6-8 November 2020 is the accommodation is $100 per person or $150 per couple. You
first HFNZ national event since lock-down: The 2020 HFNZ can choose to pay via internet banking as soon as you like, or
Adult Weekend. you will be invoiced closer to the date.
Living with a bleeding disorder, or living with a person Please use your family name as a reference.
with a bleeding disorder, is challenging. Adult Weekend is
an opportunity for adults with bleeding disorders to openly Account Name: Haemophilia Foundation of New Zealand
discuss their experiences, challenges, and concerns about Account: 02 0828 0102656 000
living and aging with a bleeding disorder. The focus of the
weekend is to increase knowledge about bleeding disorders, To make any other arrangements please contact Leanne at
upcoming trends and treatments, aging with a bleeding head office: admin@haemophilia.org.nz
disorder, and the provision of health care in New Zealand, as
well as learning about HFNZ and its activities. So, sign up today:
https://2020-hfnz-adult-weekend.lilregie.com/
Southern Outreach Worker, Ross, is working on the
programme, and so far it’s looking fantastic! We have an The link will take you to the sign-up page, hosted by Lil Regie,
expert HFNZ panel lined up, a haematologist, a nurse, and where you’ll be able to give us all your details, and sign up
a psychologist, all concentrating on the theme that ‘life is a your other half too. Then all you have to do is sit back and wait
highway’. That means talking about the challenges you might for November.
encounter along the way, as well as the good things.
We’re looking forward to seeing you then!
Along with the education, there’ll be plenty of chances to enjoy
some social time, as well as a couple of interesting outings.
To top it all off we’re aiming to finish up with the HFNZ AGM
14 Bloodline July 2020Outreach and Member Details
In order to increase the reach of HFNZ communications, and That means, for some people, it may still be more appropriate
improve member outcomes, Outreach have begun updating to connect with you via phone, text, email, or video. Please
member details in the database. Over the coming weeks contact your OR Worker to organise the best way for you to
members will be contacted to add missing details. The most connect with them:
important details are email addresses and dates of birth.
Southern: Ross Paterson - ross@haemophilia.org.nz,
Having a current email address for you and your whānau is 021 656 804
important as we try to manage costs. Posting invitations and
information to members is an expensive business, and where
possible we would prefer to make contact electronically. If you
have an email address, but you’re not sure that we have it,
please let your Outreach Worker know.
The other essential detail is date of birth. We need this so that
we can be sure you’re on the list for age-restricted events, like
Adult Weekend and Women’s Weekend. If we have no DOB
for you, the database won’t filter you in to age-based mailing
lists. Again, if you’re not sure whether we have your DOB,
please let your OR Worker know.
Please find your OR contact details below.
As many of you already know, the National Council has
decided not to seek a new Midland OR Worker until 2021.
Ross Paterson - Southern OR Worker
During this time, members in the Midland region will
continue to be supported by either Northern OR Worker Central: Lynne Campbell - lynne@haemophilia.org.nz,
Amber Maihi or Central OR Worker Lynne Campbell. 027 273 3443
Northern: Amber Maihi - amber@haemophilia.org.nz,
027 512 1114
Amber Maihi - Northern OR Worker Lynne Campbell - Central OR Worker
If you live in the Waikato DHB catchment your OR contact is
Amber Maihi at amber@haemophilia.org.nz or on
027 512 1114.
If you live in the Bay of Plenty or Lakes DHB catchments, then
your OR contact is Lynne Campbell at lynne@haemophilia.
org.nz or on 027 273 3443.
Please refer to the image below to figure out the DHB you’re
in, and which OR Worker currently supports you.
Since the Covid-19 lockdown, Outreach staff were advised
not to undertake any travel. We are pleased to say that there
is now the ability for OR Workers to travel to meet with
HTCs and members. With reduced funding due to the Covid
situation, OR Workers still need to manage their travel well.
Bloodline July 2020 15You can also read
