Factor Nine News The Coalition for Hemophilia B
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F actor N ine N ews
The Coalition for Hemophilia B
WINTER 2018 Topics in Hemophilia
• Leadership Through Service • After Hurricane Maria Puerto Rico
• Taekwondo Humanitarian Mission
• When Insurance Becomes a • Industry News
Challenge • Gettin’ In the Game, Swim Style!
• AIDS Grove Dedicated • Upcoming Events
• A Community of Care • Kidz Korner
GENERATION IX PROJECT
LEADERSHIP THROUGH SERVICE
OUR EXPERIENCES AT THE PIGEON KEY
GENERATION IX PROGRAM
BY MANUEL A. LOPEZ SERRANO, SHAD H. TULLEDGE AND EMILI LEE
The Generation IX Project gathers community members with Hemophilia B from all over
the United States and Puerto Rico. Generation IX Project Leadership was held in Pigeon
Key, Florida from January 2nd to the 7th of 2018 with the main educational theme of “Service
Leadership”. Gutmonkey, the facilitators of the project, created a space where each of the
mentors became submerged into the concept of service leadership, not just within the
bleeding disorders community, but also in society in general. Embracing their roles as
leaders, the Gen IX crew set out to make a difference.
Thank you to
Aptevo for
generously
sponsoring our
Gen IX Meeting!
2 Winter 2018 Factor Nine News
This leadership model brought the Marathon Key, home of the Dolphin This group of individuals is paying
group together to learn about charity Research Center, to help the center forward what they have received
and service and that this model in their efforts to recover from the and acting upon their commitment to
had three essential concepts at its hurricane. They were excited to make the world a better place, one
core: the acts of being compelled, receive the group of Hemophilia B act of kindness at a time.
courageous and committed; which mentors and were impacted to see ~ Manuel
come into play in their advocacy members of a community that might
and leadership efforts. During their normally be labeled as “physically Hurricane Irma devastated the
time in the Keys, the mentors were limited,” to actually come and do the Florida coast and unfortunately
given the opportunity to bring service dirty work and heavy lifting, for the had a great impact on the Florida
leadership into practice. They first simple reason to give back and put keys in September of 2017. The
helped the Pigeon Key Foundation service leadership into practice. Florida Pigeon Key is home to our
to restore part of the landscape The group of amazing individuals Generation IX Leadership program
of the Key. It was amazing to see that made part of the Generation IX and has been held there the past
the impact of what a simple act of team, had an amazing time at this years. This January, we attended
kindness, support and service can program and will most likely say, the program and GutMonkey’s
do. that their lives were changed for the team taught us as leaders there
better. Service Leadership is more are many ways we can be drawn
The mentors were also given the than just a concept; it’s an act of to service work. Sometimes we are
opportunity to put service leadership love, compassion, commitment or compelled by an experience like
into practice. They made their way to just the simple act of being human. Hurricane Irma, however we have
www.hemob.org Factor Nine News 3
choices and so by accepting Being a new member in such a
to become service leaders we close knit community can be a
are courageous enough to offer little scary, but I also believe it
help, ask where our skills can can be detrimental in some ways
be utilized and step up to reflect if you don’t put yourself out there
our commitment to doing work to learn valuable information
that may sometimes make us when it personally pertains to you
uncomfortable. and your children. So this year,
as a new mom of a child with
On Pigeon Key, we rebuilt the fire hemophilia, I decided to attend
pit which had been destroyed by the Generation IX Program with
Hurricane Irma. We first had to GutMonkey on Pigeon Key. This
remove large rocks and slabs of experience was so amazing and
concrete. After we had removed a fantastic way to start the new
all the rocks we started to dig a year – and not just because I
10-foot circle to the depths of 3 had freedom from my children
feet. Once all the sand was out for six days! I didn’t just gain
we started to line the hole with more knowledge and insight than
the rocks and slabs of concrete I had prior to the event – I also
to keep the shape of the fire pit. gained experience, perspective,
This helped us put our service friendship and leadership skills.
leadership in practice.
This program gave me the
We spent time networking and opportunity to meet other
creating connections amongst strong and amazing people
the Pigeon Key staff, the with hemophilia, both men and
GutMonkey team and the Gen women, from all over the country
IX participants. There were that I would probably never have
times when we as a group felt had the opportunity to cross
uncomfortable doing certain work paths with. I assure you that after
however, we learned to step- those six fun filled days in the
up by showing we are willing Florida Keys, I not only became
to do the dirty work without the a better mom to my child with
reward and learning as leaders hemophilia, but I also became
to step aside to create space for a better leader and advocate
new ideas, emerging leaders, for myself, my children and the
and celebrating others. Overall bleeding disorders community.
the Generation IX Pigeon Key I highly encourage everyone to
Leadership Program was a great attend a GenIX program and I
learning experience and I hope to am already looking forward to
use the skills I learned in Service future Generation IX events!
Leadership to further give back in ~ Emili
my community.
~ Shad
4 Winter 2018 Factor Nine News
TAEKWONDO
BY TRISTAN MARTIN - 11 years-old
Taekwondo has a positive effect
on my life in the following ways:
FOCUS: GOAL SETTINGS:
Since I’ve started Taekondo (TKY), TKD teaches me about short
I am more focused when I do my term and long term goal
homework. In school, I found that I setting. For example, a short
pay more attention to the teachers term goal is working on the
too. next technique, working on
the forms, practicing 1-step
ENTHUSIASM: sparring and getting the
Being part of the TKD family inspires testing tip. A long term goal is
me to help Children’s of Alabama by getting to the next belt. TKD
participating in a fundraising activity is teaching me that to grow, I
and the Miracles Board Break-A- must set goals and work hard
Thon. Children’s of Alabama holds a towards them. It’s not always
special meaning for me. I was born easy, but that’s what makes it
with severe hemophilia B. That’s even better when you achieve
a bleeding disorder that impacts the goals you set for yourself.
the clotting of my blood. I don’t clot I now have confidence to show my
like a normal child unless I get this COOPERATION: sister and others what the moves
special medicine through my port, TKD teaches me to make friends are, answer the questions they
which is under my skin (like a cancer with people outside of my school or might ask, and help if I see that their
patient). I go to Children’s Hospital church or my neighborhood. Black technique or posture needs to be
at least once a year for my annual belt preparation is the ultimate corrected. We must always do our
check-up - I get a blood test, see the cooperation so far because I get to best.
hematologist, a dentist, a physical practice choreography with a partner,
therapist, a social worker and a which is super fun.
RESPECT:
TKD constantly reminds me to not
nutritionist. I get to experience first-
only respect our master, instructors
hand the hard work of our dollars SELF-CONTROL: and peers, but also our parents,
from our fundraising. What we do Self-control is doing the right thing, grandparents, siblings, pastor,
in TKD is important, we do make a doing something good without teachers, doctors, nurses, police
difference in the lives of children. being asked and doing it right. TKD officers and others in the community.
teaches me the basis for saying Yes,
sir, Yes, ma’am. It teaches me to
stay at attention and not fidget.
RESPONSIBILITY:
TKD teaches me to make sure
my TKD bag has my uniform, my
PERSEVERANCE: belt and my protective gear. It is
TKD helps me to never give up. my responsibility, not my parents’
Some days I don’t want to go to responsibility. I need to better use
practice, but I go anyway because this lesson and apply it toward my
I know I want to reach my goal, be school papers, homework and my
reliable and not let down my partner percussion instrument so I don’t
down, and have fun. accidently leave them at home.
CONFIDENCE: LEADERSHIP:
TKD gives me confidence in myself. TKD teaches me that in order to
I look at my little sister and it reminds become a good leader, one must
me of when I first started in TKD and have all the above attributes,
how I didn’t know the moves and practice them daily and teach them
how much I relied on my instructor. to others.
www.hemob.org Factor Nine News 5
HemMobile® Striiv Wearable—
GET THE
MOST OUT
OF ACTIVITY
TRACKING
HemMobile® App Striiv Wearable
TRACK ACTIVITY
Track your heart rate, steps, distance,
calories, and duration
TRACK BLEEDS
Photograph, map, and log each bleed
TRACK INFUSIONS
Record the date, time, and location of
every infusion
SHARE REPORTS
Create consolidated reports to share
with your treatment team
To begin tracking, the HemMobile® Striiv Wearable
must be paired with the HemMobile® app.
Claim your FREE HemMobile® Striiv Wearable —
visit www.HemMobileWearable.com
Pfizer will not have access to any personal information you enter into HemMobile®.
HemMobile® is not intended for curing, treating, seeking treatment for, managing
or diagnosing a specific disease or disorder, or any specific health condition.
PP-HEM-USA-0796-01 © 2017 Pfizer Inc. All rights reserved.
Printed in USA/May 2017
WHEN
INSURANCE
BECOMES A
CHALLENGE
At first, when Laura received letters from her health insurance company
asking for the weights of her three sons, Billy, Adam and Josh, who have
severe hemophilia B, she did not find it suspicious, and she responded to
their requests. However, when she told her doctor and pharmacist about
the requests, they told Laura these requests were strange.
Shortly thereafter, the insurance company that has been While all this was going on, Laura felt overwhelmed
supplying the factor for Laura’s boys for over a year and alone. Living in rural Oregon, her family was a six-
sent her a letter, which said that they would be lowering hour drive from their hematologist. They had to visit the
the factor dosages. They claimed the dosages the boys emergency room frequently and bring their own factor,
were getting were not in compliance with the factor because the hospital did not carry it. Laura reached
package insert guidelines. When Laura’s pharmacy out to her local governmental insurance commission
preauthorized the prescriptions in the dosages they and they carried out several reviews of her family’s
had been receiving, they were denied. Instead, the situation. However after each review, they sided with the
insurance company altered the scripts and cut the insurance company and no relief seemed in sight.
doses for all three boys by 80 percent.
Finally, after six months Laura found someone in the
This couldn’t have come at a worse time since the insurance commission office who cared. Together, they
family’s hematologist was actually talking about raising insisted the insurance commission involve the family’s
the dosage for Laura’s youngest son, Josh, who was hematologist in the review process. This time, after the
experiencing nosebleeds. Laura was adamant to get hematologist shared all the family’s records with the
answers from the insurance company as to why they commission, they came out in favor of Laura and her
were unlawfully changing the scripts for her family. boys. In fact, they actually recommended the doses for
She called them continuously and was sent from one all three boys be increased.
department to another, only to having them repeat
the same answer they had sent in the letter – that the When Laura reflects on the trying times she experienced
dosages they had been filling for over a year were now not being able to prevent her kids from constant bleeds
considered by them as “experimental” and therefore and the repercussions that came with them, she still
not covered. After some time, the insurance company feels afraid if such a situation should repeat itself. Since
stopped taking Laura’s calls altogether. her sons began receiving their properly prescribed
dosages, all the bleeds have stopped. The insurance
Laura had to take off from work to attend to this sudden company however, still tries to alter the scripts every
change within her family and deal with the insurance now and then. When asked why she doesn’t switch to
company. Meanwhile, Josh developed a bleed in his a different health insurance company, she was quick to
knee, and all three boys began having daily nosebleeds, reply, “Why? So they could do the same thing to another
sometimes lasting hours. One night, Laura woke to find poor family?” Additionally she stated, “The insurance
Adam in the shower with his nose bleeding profusely. company would love it if we left. As a family, we cost
“There was so much blood in the tub, it looked like there them millions of dollars each year.”
had just been a massacre,” she said. Shortly thereafter,
Billy developed a bleed in his elbow. Josh’s knee bleed From her story, Laura wants others in the bleeding
became worse and he had to use crutches. With the disorders community to come away with the
kids having consistent nosebleeds, they felt weak and understanding that they should always stand up for
sick because of the blood loss. themselves. “Don’t back off!” she says. “Persistence in
fighting for your rights will eventually pay off.”
By John King Sep 16, 2017|San Francisco Chronical
AIDS GROVE Article link: http://m.sfgate.com/bayarea/article/AIDS-Grove-
dedicates-new-section-to-hemophiliacs-12203299.php#photo-
DEDICATES NEW 1809232?cmpid=email-mobile
SECTION TO There’s a new place of solace within
HEMOPHILIACS KILLED the National AIDS Memorial Grove: a
memorial circle devoted to victims of
BY THE DISEASE the disease who were hemophiliacs.
About 300 people attended a ceremony Saturday
morning in Golden Gate Park to dedicate the space,
which has several benches inscribed with the names
of victims, most of whom died between 1983 and the
mid-’90s. Shrubs were planted along the circle and then
family members read the names of victims, each name
accompanied by the ringing of a Tibetan bell.
The victims had been infected in the early 1980s by the
injection of blood-clotting products for hemophiliacs that
were contaminated by plasma tainted with HIV.
The best-known victim was Ryan White, an Indiana
middle-school student who at first was banned from
his school after being diagnosed. His case received
international attention and the teenager eventually was
befriended by celebrities including President Ronald Photo: Jason Henry
Reagan, basketball star Kareem Abdul-Jabbar and
singer Elton John. National AIDS Memorial Grove volunteer, Jesse Dimond
arranges flowers while preparing for the December 1st 20th
“It took the innocence of a young boy to begin to anniversary of the Grove. In Golden Gate Park, Nov. 28, 2011.
shake the bigotry and the hatred” directed by many
people toward many of the men, women and children “The hemophiliac victims are such a unique community,
affected by the disease, said John Cunningham, in that they were affected by the tainted blood supply,”
executive director of the National AIDS Memorial Grove. Cunningham said. “It’s important that we all unite” in
An estimated 10,000 hemophiliacs eventually died, overall remembrance of the roughly 750,000 Americans
Cunningham said. who are estimated to have died of AIDS.
The grove’s first redwood was planted in 1991, and it John King is a San Francisco Chronicle staff writer.
was declared a national memorial in 1996. There are Email: jking@sfchronicle.com
several circles of contemplation, but this is the first one Twitter: johnkingsfchron
dedicated to a specific set of victims.
10 Winter 2018 Factor Nine NewsA COMMUNITY OF CARE BY JILL LATHROP
In June 2016, my son suffered a psychotic break The decision to take him to the emergency department
resulting in an inpatient hospitalization. As I sat on the at our local hospital turned into a 6-day stay in the
floor in the hallway of the emergency department, I felt emergency room and another 4 days in a psychiatric
helpless, hopeless and terrified; the feeling was terribly unit 200 miles from our home. Despite our efforts to
familiar. I was taken back to our very first emergency explain hemophilia, our son injured himself early in his
room visit for an infusion of clotting factor. I remember stay. We had difficulty accessing medical attention for
feeling overwhelmed, even leaving the room as the the hemophilia related injury. We were also dismayed
nurses struggled to find a vein in the arms of my to learn virtually all the mental health facilities available
screaming six-week old baby. That was also the night to help my son refused to accept him because of his
I realized my husband and I were going to have to be bleeding disorder.
our kids’ strongest advocates. I couldn’t run away from
hemophilia; I certainly couldn’t run away from my 18- Once our hematologist was notified, he was able to
year old’s psychosis. assist in finding an suitable placement. An appropriate
course of treatment was initiated and my son was
Looking back, I can see there were many subtle things discharged 3 days later. His recovery from psychosis
that led up to my son’s psychosis. We noticed changes hasn’t been easy; however, the tools that helped us
in his routine, but attributed them to excitement and navigate hemophilia are the same tools we’re relying
stress surrounding his high school graduation. upon now.
The morning I took my son to the hospital began When I needed help 20 years ago, the bleeding
uneventfully. I think I asked him if he had breakfast disorders community stood shoulder to shoulder with
and his response was along the lines of “I don’t need me, encouraging and teaching me. 20 years later,
to eat anymore.” Something about the way he made my blood brothers and sisters have been a source of
this statement made me stop and really look at him. comfort and encouragement through this leg of our
He was disheveled and a little disoriented…something journey.
was definitely off. Reassuring my son I wanted to help
him and that I thought he needed to see a doctor, we Truly, though, if you’re reading this, you’re stronger than
headed for the hospital. you know and you are not alone.
www.hemob.org Factor Nine News 11In hemophilia B
TAKE CONTROL TO A
HIGH LEVEL
WITH REBINYN ®
NOW AVAILABLE Clayton, 34 years
old, is a pilot wh
o hikes and cam
ps in his spare
time. Clayton live
s with hemoph
ilia B.
Rebinyn elevates factor levels above normal levels
® a
+ 94 % Factor IX (FIX) levels achieved
immediately after an infusionb 17 % FIX levels sustained
after 7 daysa
With a single dose of Rebinyn® 40 IU/kg in adults with ≤2% FIX levelsa
a
In two phase 3 studies, factor levels were evaluated for 1 week after the first dose of Rebinyn® b
Based upon a 2.34% increase in factor levels per IU/kg infused in adults.
40 IU/kg. The average levels after 7 days were 16.8% in 6 adults, 14.6% in 3 adolescents,
10.9% in 13 children ages 7 to 12 years, and 8.4% in 12 children up to age 6 years.
Image of hemophilia B patient shown is for illustrative purposes only.
INDICATIONS AND USAGE How should I use Rebinyn®?
What is Rebinyn® Coagulation Factor IX (Recombinant), • Rebinyn® is given as an infusion into the vein.
GlycoPEGylated? • Call your healthcare provider right away if your
Rebinyn® is an injectable medicine used to replace clotting bleeding does not stop after taking Rebinyn®.
Factor IX that is missing in patients with hemophilia B. • Do not stop using Rebinyn® without consulting
Rebinyn® is used to treat and control bleeding in people your healthcare provider.
with hemophilia B. Your healthcare provider may give you
What are the possible side effects of Rebinyn®?
Rebinyn® when you have surgery. Rebinyn® is not used for
routine prophylaxis or for immune tolerance therapy. • Common side effects include swelling, pain, rash
or redness at the location of the infusion, and itching.
IMPORTANT SAFETY INFORMATION
• Call your healthcare provider right away or get
What is the most important information I need emergency treatment right away if you get any of
to know about Rebinyn®? the following signs of an allergic reaction: hives, chest
• Do not attempt to do an infusion yourself unless you tightness, wheezing, difficulty breathing, and/or swelling
have been taught how by your healthcare provider of the face.
or hemophilia treatment center. Carefully follow your • Tell your healthcare provider about any side effect
healthcare provider’s instructions regarding the dose and that bothers you or that does not go away.
schedule for infusing Rebinyn®.
• Animals given repeat doses of Rebinyn® showed
Who should not use Rebinyn®? Polyethylene Glycol (PEG) inside cells lining blood vessels
Do not use Rebinyn® if you: in the choroid plexus, which makes the fluid that cushions
• are allergic to Factor IX or any of the other ingredients the brain. The potential human implications of these
of Rebinyn®. animal tests are unknown.
• are allergic to hamster proteins. Please see Brief Summary of Prescribing Information
What should I tell my health care provider before on the following page.
using Rebinyn®? Rebinyn® is a prescription medication.
Tell your health care provider if you: You are encouraged to report negative side effects
• have or have had any medical conditions. of prescription drugs to the FDA.
• take any medicines, including non-prescription medicines Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
and dietary supplements.
• are nursing, pregnant, or plan to become pregnant.
• have been told you have inhibitors to Factor IX. Learn more at rebinyn.com
Novo Nordisk Inc., 800 Scudders Mill Road, Plainsboro, New Jersey 08536 U.S.A.
Rebinyn® is a registered trademark of Novo Nordisk Health Care AG.
Novo Nordisk is a registered trademark of Novo Nordisk A/S.
© 2018 Novo Nordisk All rights reserved. USA17BIO02796 February 2018infuse the medicine by themselves or with the help of a Cap Color Indicator Nominal Strength
family member.
Red 500 IU per vial
Your healthcare provider will tell you how much
REBINYN ® to use based on your weight, the severity of Green 1000 IU per vial
your hemophilia B, and where you are bleeding. Your Yellow 2000 IU per vial
dose will be calculated in international units, IU.
Brief Summary Information about: Always check the actual dosage strength printed on
Call your healthcare provider right away if your
REBINYN® Coagulation Factor IX bleeding does not stop after taking REBINYN ®. the label to make sure you are using the strength
(Recombinant), GlycoPEGylated prescribed by your healthcare provider.
If your bleeding is not adequately controlled, it could
Rx Only be due to the development of Factor IX inhibitors. How should I store REBINYN ®?
This information is not comprehensive. This should be checked by your healthcare provider. Prior to Reconstitution (mixing the dry powder in
You might need a higher dose of REBINYN ® or even a the vial with the diluent):
• Talk to your healthcare provider or pharmacist
different product to control bleeding. Do not increase Store in original package in order to protect from light.
• Visit www.novo-pi.com/REBINYN.pdf to obtain the total dose of REBINYN ® to control your bleeding
FDA-approved product labeling Do not freeze REBINYN ®.
without consulting your healthcare provider.
• Call 1-844-REB-INYN REBINYN ® vials can be stored in the refrigerator
Use in children (36-46°F [2°C-8°C]) for up to 24 months until the
Read the Patient Product Information and the REBINYN ® can be used in children. Your healthcare expiration date, or at room temperature (up to 86°F
Instructions For Use that come with REBINYN ® provider will decide the dose of REBINYN ® you will [30°C]) for a single period not more than 6 months.
before you start taking this medicine and receive. If you choose to store REBINYN ® at room temperature:
each time you get a refill. There may be new • Note the date that the product is removed from
information. If you forget to use REBINYN ® refrigeration on the box.
This Patient Product Information does not take If you forget a dose, infuse the missed dose when you • The total time of storage at room temperature
the place of talking with your healthcare provider discover the mistake. Do not infuse a double dose to should not be more than 6 months. Do not return
about your medical condition or treatment. If you make up for a forgotten dose. Proceed with the next the product to the refrigerator.
have questions about REBINYN ® after reading this infusions as scheduled and continue as advised by • Do not use after 6 months from this date or the
information, ask your healthcare provider. your healthcare provider. expiration date listed on the vial, whichever is
earlier.
What is the most important information I need If you stop using REBINYN ® Do not use this medicine after the expiration date which
to know about REBINYN ®? Do not stop using REBINYN ® without consulting your is on the outer carton and the vial. The expiration date
Do not attempt to do an infusion yourself healthcare provider. refers to the last day of that month.
unless you have been taught how by your If you have any further questions on the use of this
healthcare provider or hemophilia treatment After Reconstitution:
product, ask your healthcare provider. The reconstituted (the final product once the powder is
center.
You must carefully follow your healthcare provider’s What if I take too much REBINYN ®? mixed with the diluent) REBINYN ® should appear clear
instructions regarding the dose and schedule for without visible particles.
Always take REBINYN® exactly as your healthcare
infusing REBINYN ® so that your treatment will work provider has told you. You should check with your The reconstituted REBINYN ® should be used
best for you. healthcare provider if you are not sure. If you infuse immediately.
What is REBINYN ®? more REBINYN ® than recommended, tell your If you cannot use the reconstituted REBINYN ®
healthcare provider as soon as possible. immediately, it should be used within 4 hours when
REBINYN ® is an injectable medicine used to replace stored at or below 86ºF (30°C). Store the reconstituted
clotting Factor IX that is missing in patients with What are the possible side effects of product in the vial.
hemophilia B. Hemophilia B is an inherited bleeding REBINYN®?
disorder in all age groups that prevents blood from Keep this medicine out of the sight and out of reach of
clotting normally. Common Side Effects Include: children.
REBINYN ® is used to treat and control bleeding in • swelling, pain, rash or redness at the location of What else should I know about REBINYN ® and
people with hemophilia B. infusion hemophilia B?
• itching Medicines are sometimes prescribed for purposes
Your healthcare provider may give you REBINYN ®
when you have surgery. Other Possible Side Effects: other than those listed here. Do not use REBINYN ® for
REBINYN ® is not used for routine prophylaxis or for You could have an allergic reaction to coagulation a condition for which it is not prescribed. Do not share
immune tolerance therapy. Factor IX products. Call your healthcare provider REBINYN ® with other people, even if they have the
right away or get emergency treatment right same symptoms that you have.
Who should not use REBINYN ®? away if you get any of the following signs of an
You should not use REBINYN ® if you allergic reaction: hives, chest tightness, wheezing,
• are allergic to Factor IX or any of the other difficulty breathing, and/or swelling of the face.
ingredients of REBINYN ® Your body can also make antibodies called “inhibitors”
• if you are allergic to hamster proteins against REBINYN ®, which may stop REBINYN ® from
If you are not sure, talk to your healthcare provider working properly. Your healthcare provider may need to
before using this medicine. test your blood for inhibitors from time to time.
Tell your healthcare provider if you are pregnant or You may be at an increased risk of forming blood
nursing because REBINYN ® might not be right for you. clots in your body, especially if you have risk factors
What should I tell my healthcare provider for developing blood clots. Call your healthcare
before I use REBINYN ®? provider if you have chest pain, difficulty breathing, leg More detailed information is available upon
You should tell your healthcare provider if you tenderness or swelling. request.
• Have or have had any medical conditions. Animals given repeat doses of REBINYN ® showed Available by prescription only.
• Take any medicines, including non-prescription Polyethylene Glycol (PEG) inside cells lining blood For more information about REBINYN®, please call
medicines and dietary supplements. vessels in the choroid plexus, which makes the Novo Nordisk at 1-844-REB-INYN.
• Are nursing. fluid that cushions the brain. The potential human Revised: 11/2017
• Are pregnant or planning to become pregnant. implications of these animal tests are unknown.
REBINYN® is a trademark of Novo Nordisk A/S.
• Have been told that you have inhibitors to Factor IX. These are not all of the possible side effects from
For Patent Information, refer to: http://novonordisk-us.
REBINYN®. Ask your healthcare provider for more com/patients/products/product-patents.html
How should I use REBINYN ®? information. You are encouraged to report side effects
Treatment with REBINYN ® should be started by a to FDA at 1-800-FDA-1088. Manufactured by:
healthcare provider who is experienced in the care of Novo Nordisk A/S
Tell your healthcare provider about any side effect that Novo Allé, DK-2880 Bagsværd, Denmark
patients with hemophilia B. bothers you or that does not go away.
REBINYN ® is given as an infusion into the vein. For information about REBINYN ® contact:
You may infuse REBINYN ® at a hemophilia treatment What are the REBINYN® dosage strengths? Novo Nordisk Inc.
center, at your healthcare provider’s office or in your REBINYN ® comes in three different dosage strengths. 800 Scudders Mill Road
home. You should be trained on how to do infusions The actual number of international units (IU) of Factor Plainsboro, NJ 08536, USA
by your hemophilia treatment center or healthcare IX in the vial will be imprinted on the label and on the © 2017 Novo Nordisk
provider. Many people with hemophilia B learn to box. The three different strengths are as follows: USA17BIO03951 12/2017AFTER HURRICANE MARIA
PUERTO RICO
HUMANITARIAN MISSION
On September 20, 2017, Hurricane Maria, a powerful
Category 4 hurricane with winds in excess of 15O
mph made direct landfall on Puerto Rico. From
BY KIM PHELAN
south to north, with drenching rain, storm surges
and devastating winds, the storm ripped through the
island resulting in complete power outage, total loss of In October, William Patsakos - a father of 4
communications and no running water. sons, 3 with hemophilia B, a pharmacist and
an FDNY Lieutenant began his two-week
journey to deliver much needed relief to the
bleeding disorder community of Puerto Rico.
Before his departure, a conference call was
had,which included William and myself,
Puerto Rico HTC’s Dr. Enid Riviera, Dr.
Van Heise, Dr. Peter Kourides, Erik Iglwski
of Rochester Regional, Joe Pugliese of
Hemophilia Alliance, Dominick Dellamarco
of the New York Blood Center, Dawn
Rotellini of NHF, Dr. Christopher Walsh
and Susan Miller both of Mt. Sinai Hospital,
Laurie Kelley of Kelley Communications,
Linda Mugford of Hemophilia Association
of New York (HANY), Brett Spitale of NHF,
Mariam Voutsis with The Consortium,
Kathryn McLaughlin of HRSA, Becky
Dudley of American and Thrombosis and
To aid in the humanitarian efforts for hurricane ravaged
Hemostasis Network (ATHN), Dr. David De
Puerto Rico, The Coalition received over $35,000 Angel Sola, and many additional bleeding
of medical supplies in just 8 days from community disorder treatment center, chapter and
members and organization representatives – all working
organizations together to assist the people in Puerto Rico.
across the
USA. Bill From medical supplies to beneficial funding,
Patsakos generosity came in many forms from many
departed on HTCs, hemophilia organizations, homecare
October 8th companies throughout the bleeding
disorders community. Over $35,000 of
to deliver the
supplies were donated and sent to the
much-needed Coalition for Hemophilia B for William to
supplies to take to assist the people in Puerto Rico.
the Puerto
Rico Chapter In addition to collecting medical supplies,
of NHF. The Coalition donated monies to several
relief organizations. The Hemophilia
14 Winter 2018 Factor Nine NewsAlliance donated monies to HFA’s Behring provided coagulation more wonderful people from great
(Hemophilia Federation of America) therapies while Novo Nordisk organizations not listed here working
Disaster Relief for proper allocation. donated money and life-saving just as hard! The tireless efforts of
Funds donated to HFA’s program medicine through AmeriCares. our community and their giving of
were administered by their Helping Pfizer activated a comprehensive time and resources is outstanding.
Hands team. Many Puerto Rican and coordinated plan at a company- It is times like this we are able to
bleeding disorder community wide level to provide cash donations see how fortunate we are to be part
members have come to stay with through the Pfizer Foundation. such a strong community.
family in the continental United They shipped thousands of factor
States on a permanent basis or doses through Direct Relief and
until the situation in Puerto Rico AmeriCares, a special one-to-one
improves. These donated funds match for donations made by Pfizer
also assisted the families in these colleagues, activated disaster relief
circumstances; and NHF and HFA protocols for its Patient Assistance
are matching donations while the Program waiving financial
Hemophilia Association of New York documentation requirements and
is standing by to help relocated allowing existing patients early
families. HFA continues to accept refills and created voluntary events
donations for this worthy cause on across the company for colleagues
their website. to assemble hygiene kits for people
in hurricane affected areas. Shire
The outstanding response from donated financially to hurricane-
pharmaceutical companies has related medical relief efforts through
also been impressive: Aptevo has their NGO partners Direct relief
made available 10 percent of the and AmeriCares, donating over
units they sell for access by US- $155K, which includes matching
based HTC blood banks managed their employees’ $55,000 generous
by AmeriCares. Bioverativ delivered donation.
emergency supplies to hemophilia
patients through Direct Relief and This is a small list of a lot of very
sent a two-week supply of factor good people from all organizations
for 70 hemophilia patients. CSL doing their part! There are many
www.hemob.org Factor Nine News 15PUERTO RICO
HUMANITARIAN MISSION
A RECAP
BY WILLIAM PATSAKOS
am an Army Veteran Medical Service There have been so many other
Corp officer, CVS Pharmacist, agencies local and federal under
and FDNY Lieutenant. In addition, FEMA and the military that have
I have 3 sons with hemophilia. I worked tirelessly to rescue people
have worked in Puerto Rico with and clear roads for access, restore
the bleeding disorder community in power and communications,
Puerto Rico’s Governor the past and have a connection to deliver supplies and medicine and
Ricardo Rosselló said, patients in hematology and oncology, volunteered.
“Make no mistake - this is their families, and members of the
a humanitarian disaster medical community. The hurricane It is important to note that prior to
generated great concern from the Hurricane Maria, the economic
involving 3.4 million US
bleeding disorder community to act situation on Puerto Rico was dire.
citizens.” immediately and decisively. I want to Prior to the hurricane, Puerto Rico
highlight several groups that acted filed for the equivalent of federal
San Juan Mayor, Carmen swiftly and generously to serve the bankruptcy protection in May 2017,
Yulin Cruz proclaimed, “Help people of Puerto Rico and share my for 74 billion in debt and 53 billion
us! Without a robust and profound gratitude and respect for in unfunded pensions. Maria has
consistent help, we will die.” the following groups: escalated Puerto Rico’s financial
chaos and damage with estimates
The official death toll of Hurricane • New York City Fire Department ranging from 40 to 80 billion. The
Maria is 64, though within 42 days Incident Management Team electric grid, and water and sewer
following the hurricane, 1052 more (Commissioner Nigro and Chief of infrastructure were experiencing
lives than usual life were claimed* Department James Leonard) daily interruptions in service.
with many more at risk due to • NYC Urban Search and Rescue
disease, lack of infrastructure and Task Force (Fire and Police The focus has been initially to
access to basic necessities and Departments) restore power and communication
medical care and medicines. • The FDNY Disaster Assistance capabilities to critical facilities -
Response Team firehouses, police stations and
The tragedy in Puerto Rico has hospitals. I have been able to assist
• Puerto Rico Fire and Police
deeply affected many Americans and coordinate medical shipments
Departments
both on the island and in the to 4 strategic pods located around
continental United States. In a 2012 • Centro Medico Medical Center the island and link up with medical
census, there are approximately 5.4 P.R. HTC and Mount Sinai HTC- volunteers to triage and deliver
million Puerto Ricans living in the US Zone 2( Dr.Rivera, Dr. Walsh, care to rural areas that are heavily
and approximately 120,000 veterans Susan Miller and staff) affected.
and many living in the NYC area. I • CVS Heath Specialty Pharmacy
am one of those American citizens (Nicole Jackson P.R. and Joel We have been able to visit patients
with family living in Puerto Rico. Helle) that doctors were concerned about
• The Coalition for Hemophilia B and deliver medications. To date,
I am writing this article to detail the (Kim Phelan) we were able to collect medical
conditions encountered during my • National Hemophilia Foundation supplies, surgical supplies and over
humanitarian trip to Puerto Rico (Val Bias, Puerto Rico chapter the counter meds and approximately
and to plead for continued support and NYC chapter Jeremy Griffin) $350,000 in factor product and
from our fellow citizens by personal injectable drugs, refrigeration, and
• Hemophilia Federation of America
volunteerism, both monetarily and storage. We have been able to assist
(Kimberly Haugstad)
with reconstruction aid workers. major distributors and pharmacies
• LA Kelley Communications and in gaining assistance from FEMA for
I want to convey messages from
“Save one Life” (Laurie Kelley)
residents, first responders, and fuel and delivery logistics. In addition
members of the medical community. I • Heart 911 (Numerous members of we have communicated needs to
the first responder community) volunteer organizations in obtaining
16 Winter 2018 Factor Nine News5 days before they were able to
return home. They found their home
completely flooded. Her cousin’s
home just near hers was completely
destroyed. Now 8 members of the
family had to stay in a space of
a living room/bedroom with only
2 beds, no water and no electric
power.
Joanne sleeps in one bed with her
grandmother, who had open heart
surgery, is insulin dependent and
has asthma. They have a very small
tarps for temporary roofing and, in Our first is Osman - a beautiful ten generator to refrigerate the insulin,
some cases, replace roofs of some month old baby boy with hemophilia but with the gas shortage, they
residences. A. His 21-year-old mother and have to wake at 4 am to wait in line
grandmother are bleeders, but were to buy gas. It’s been very difficult
We visited many hospitals and never officially diagnosed. Osman to purchase drinking water, and in
in particular Centro Medico San has a newborn baby sister, who has their area, there is almost no food
Juan University, which is the major been diagnosed with a bleeding available in stores.
medical institution on the island. I disorder. Osman’s mom, Maria,
had the opportunity to round with had to stop working as a security Communications are very difficult,
attending physicians and department guard since she is frequently taking which worries Joanne in the event
head, Dr. Enid Rivera, and CVS her kids to doctor’s appointments. of a health emergency. She tried
territory executive, Nicole Jackson. The children’s father currently to smile, but seemed very sad and
We worked with CVS Health works daily12-hour shifts, also as a hopeless. Receiving their blessings
Specialty to assist pharmacists with security guard, to provide at least the for our help, we left Dr. Rivera, Dr.
shipments for all complicated and basic needs for the family. During Amy Lee Cabrera (Hema-Oncology
chronic disease states. There is a the hurricane, their house was fellow), and Yue Sai Jao (4th year
rare genetic disease that afflicts completely flooded. All their furniture, med student), and traveled to the
Puerto Ricans in greater incidence clothes, and babies articles were town of Canovanas looking for what
than any other population in the damaged, and three weeks after the used to be the home of Victoria
world. This condition is known as hurricane, only had water a couple of Pereira, a patient who left the island
Hermanski-Pudlak syndrome which hours a day with no electric power. and is currently receiving help
is an oculocutaneous albinism Maria had been struggling to find from the HFA. As we arrived to the
with bleeding problems and access to the medication Amicar. address provided, it was sad to see
platelet abnormalities. There are The pharmacies contracted with the house completely destroyed.
approximately 100 hemophiliacs Medicaid didn’t have it available.
and another 150 von Willebrand’s Maria was very thankful she would Hurricane Maria was a very powerful
Disease patients. These and other receive it as part of donations to the hurricane that left no part of the
oncology patients rely on continuous Pediatric Hospital. It’s heartbreaking island untouched. The people of
infusions and access, which is how Osman’s mom says they are Puerto Rico are very strong, resilient
complicated by no electricity or clean okay while everything is going so and with tremendous dignity. They
running water. bad for them. are Americans living in third world
conditions. We must work to restore
Together with Lilly Gomez and We also met Joanne, a 20-year- Puerto Rico’s infrastructure while
Nicole Jackson, we are compiling old that had been diagnosed with simultaneously assisting their
testimonials and patient stories severe Factor 5 deficiency (with economy and jobs, or the island
to share with the international a prevalence of about one in a will face a mass exodus to the
community for the purpose of million) since she was 11-months- continental U.S. Since Hurricane
awareness and fundraising, and old. She was being infused factor Maria, 40,000 people have moved
will bring to you in a future article. while we listened to her story. from the island
I would like to share 3 such cases Before the hurricane, Joanne lived
we encountered and, with their with her stepmother, aunt, uncle Please donate your time and money.
permission, share their stories and and grandmother in Ponce. Since If asked to help, respond with “I am
pictures in order to raise awareness their house was near the beach, here!”
and funds to help Puerto Ricans in they were evacuated from their
their hour of greatest need. home the day before the hurricane. * https://www.nytimes.com/interactive/2017/12/08/
They stayed in a refuge center for us/puerto-rico-hurricane-maria-death-toll.html
www.hemob.org Factor Nine News 17INDUSTRY NEWS BY DR. DAVID CLARK
Alnylam Receives FDA Approval to Re-Start data showing that treatment with Alprolix results in
Clinical Studies effective bleeding protection with extended dosing
Alnylam Pharmaceuticals has received FDA approval intervals. The new labeling includes additional clinical
to resume its clinical studies of fitusiran for treatment trial experience in 93 subjects treated prophylactically
of hemophilia A and B. The studies were put on hold for more than two years. The new data shows median
earlier after the death of a subject. The death was later spontaneous annualized bleeding rates of zero for
judged to not have been caused by fitusiran but rather children and 1.04 for adults and adolescents when
to a misdiagnosis that lead to dosing the hemophilia A treated weekly.
subject with high doses of factor VIII. That apparently
lead to fatal blood clots in his brain. Alnylam will resume Bioverativ Presents New Clinical Data for
the clinical study including new risk-mitigation measures 14-Day Dosing with Alprolix
to better control the concurrent use of other hemophilia At the recent American Society of Hematology (ASH)
medications. meeting, Bioverativ presented additional clinical data
showing that weekly, 10-day or even 14-day or longer
The fitusiran death and the death of a subject in a trial dosing with Alprolix can deliver optimal protection
of Hemlibra, a hemophilia A drug that was recently against bleeds. Study participants who received Alprolix
licensed, show that the community needs to develop prophylactically every 14 days or longer had a median
a new awareness of the potential dangers when spontaneous annualized bleeding rate of 0.7 over three
manipulating the clotting system. Many of the new years of observation.
hemophilia drugs being developed are not just new
versions of factor VIII or IX. They exploit other parts Bioverativ Presents Data on the Tissue
of the clotting system to try to shift the balance back Distribution of Alprolix
toward being able to efficiently form clots. In doing so, Bioverativ in collaboration with Invicro, LLC, a leading
they are potentially interfering with the regulation and imaging company, performed a study of the distribution
control naturally occurring in the system, which can lead of Alprolix and other factor IX products in the body.
to thrombosis, dangerous unneeded clotting. Products Normally, only about half of the factor IX made in the
like factor VIII and IX concentrates and inhibitor- liver or infused by a patient remains in the blood stream.
bypassing agents that seem very safe in normal use can The other half binds to the endothelial cells that line
have unexpected consequences when other parts of the the blood vessels or distributes into the extravascular
clotting system have been modified. space, the space in the body outside the blood vessels.
One idea is that this provides a reservoir of extra factor
Alnylam Restructures Agreement with IX in case of severe bleeding. One concern is whether
Sanofi Genzyme the longer-acting factor IXs will also perform the same
Alnylam has restructured their development agreement way.
with Sanofi Genzyme for fitusiran. Under the new
agreement, Sanofi Genzyme will take over all Bioverativ and Invicro radio-labeled Alprolix, a
development activities and conduct of the clinical conventional factor IX product and a glycol-pegylated
studies. Sanofi will have global commercialization longer-acting factor IX and infused them into animals.
rights. This is the next step in Sanofi becoming a major (Radio-labeling is attaching a radioactive tag to a
hemophilia treatment organization. They recently also molecule. The radioactivity can then be detected by
obtained FDA approval of Hemlibra, an innovative imaging to determine where the labeled compound is
treatment for hemophilia A. in the body.) The results indicate that Alprolix showed
the greatest distribution to the extravascular spaces,
Bioverativ Receives Updated Labeling including the joints. The conventional factor IX was
Approval for Alprolix next, and the glyco-pegylated product showed the least
Bioverativ continues to obtain clinical data to support the amount. The meaning of these results for hemophilia
use of Alprolix in treatment of hemophilia B. FDA has treatment and joint issues is currently unknown, but
approved updated labeling including addition of pediatric Bioverativ plans to do more work in this area.
18 Winter 2018 Factor Nine NewsCatalyst Presents Data from Phase I/II Trial (range 15 - 78%). The annualized bleeding rate of
of Variant Factor IX the group fell from about 11.0 before treatment to 0.4
Catalyst Biosciences announced promising results afterward. Eight of the patients were able to completely
from their Phase I/II trial of their factor IX variant CB stop prophylaxis with clotting factor. There were no
2679d. CB 2679d is a variant of normal factor IX that severe adverse events, although two patients had
has only three different amino acids in its structure, but temporary increases in liver enzymes that were resolved
demonstrates much higher activity and longer half-life with corticosteroids.
than normal factor IX. The clinical results show that CB
2679d exhibits a half-life almost five times the half-life of Meanwhile, in November, Spark and Pfizer announced
normal factor IX when dosed subcutaneously once-daily. that they had amended their license agreement for
The Phase I/II study is expected to be completed in the SPK-9001. They have modified the manufacturing
first quarter of 2018. process and are transferring it to a Pfizer facility for
future production. They will continue the Phase I/II study
Catalyst Starts Phase II/III Study of Variant with up to five additional patients using product made by
Factor VIIa the new method for comparability. Pfizer will take over
development of the product and perform a Phase III
Catalyst Biosciences is initiating a Phase II/III study study.
of their variant form of factor VIIa for treatment of
hemophilia A and B patients with inhibitors. Factor VIIa Tremeau Developing Pain Treatment for
is commonly used to treat inhibitor patients because it Hemophilic Arthropathy
can bypass the step in the clotting cascade that involves
factors VIII and IX. However, the current product has Tremeau Pharmaceuticals, a new pharmaceutical
a relatively short half-life and therefore must often company focused on developing non-opioid pain
be administered once or more a day. Like Catalyst’s treatments for rare diseases, has been granted orphan
variant factor IX above, the variant FVIIa, called drug designation for their TRM-201 under development
marzeptacog alfa (activated) or MarzAA, has only small for treatment of hemophilic arthropathy (joint disease).
changes to its structure, but those changes have large TRM-201 is rofecoxib, which was previously marketed
effects on potency and half-life. It is also administered by Merck & Co. as Vioxx. Rofecoxib is a COX-2 non-
subcutaneously. In the Phase I study MarzAA showed steroidal anti-inflamatory drug (NSAID), which reportedly
a six- to nine-fold improvement in potency and half-life has no effect on bleeding times and a reduced risk of
compared to the current FVIIa product with excellent gastrointestinal bleeding. Vioxx was voluntarily taken
safety and tolerability. off the market by Merck in 2004 after studies showed
that it increased the risk of cardiovascular events, such
Seattle Researchers Engineer B Cells to as heart attacks and strokes. Tremeau believes that the
Secrete Factor IX for Gene Therapy risks can be adequately managed within small specific
indications like hemophilic arthropathy and other niche
Researchers at the Seattle Children’s Research applications. TRM-201 is currently planning a Phase III
Institute (SCRI) are developing a method to insert clinical study.
factor IX genes into B cells for production of factor
IX. B cells, also known as B lymphocytes, are part of uniQure Presents Gene Therapy Data for
the immune system. They are white blood cells that AMT-061
circulate in the bloodstream and secrete antibodies.
Previous attempts to genetically engineer B cells to uniQure has replaced the conventional factor IX gene
secrete proteins failed to produce enough expression in its AMT-060 gene therapy treatment with the highly-
for adequate gene therapy. The SCRI scientists used active FIX-Padua gene, hoping to achieve higher factor
CRISPR/Cas9 methods to edit the B cells’ genomes to levels in patients. The new treatment is designated
introduce the factor IX gene in a specific location and to AMT-061. FIX-Padua is a variant of factor IX that is
cause the B cells to mature into a specific type known approximately eight times more active than conventional
as plasma cells. Plasma cells reside for long periods, factor IX. In studies in non-human primates, treatment
even decades, in the bone marrow where they normally with AMT-061 produced about 6.5 times higher factor IX
secrete antibodies, but could potentially secrete proteins levels than treatment with AMT-060. Safety and toxicity
like factor IX. measures showed no significant difference between the
two. Both FDA and the European Union have tentatively
Spark and Pfizer Announce Results from given uniQure approval to move forward substituting
Gene Therapy Study AMT 061 in the ongoing clinical studies. uniQure is
currently preparing the IND amendment for the study,
Spark Therapeutics announced promising results for which it plans to submit to FDA in the first quarter of
their hemophilia B gene therapy treatment, SPK 9001. 2018. The pivotal Phase III study is expected to start
The ten patients who took part in the Phase I/II study during the third quarter of 2018.
achieved an average factor IX level of 34% of normal
www.hemob.org Factor Nine News 19GETTIN’ IN THE GAME
SWIM STYLE
BY MELISSA FRANZEN
My son, Charlie, who is 12 years disorder, so spending time with
old, was lucky to attend CSL community families is always
Behring’s 16th Annual 2017 Gettin’ special to us. The weekend
in the Game Junior National was packed with fun activities
Championship in Phoenix, Arizona with a focus on swimming, golf,
last November. Charlie loves to basketball; as well as being
swim and was excited that they healthy, and ended with an award
added swimming to their sports ceremony.
activities line up this year.
On the first day, time was spent
Charlie has been playing club going over a variety of stretches
water polo for the last year and and learning about sports safety
a half. To be considered for this as well as going through an
opportunity, he submitted an assortment of drills and swimming
essay he wrote on how much he exercises. The second day
loves swimming and water polo. was a competition day where,
In his essay, he expressed that through a variety of games, the
when he’s swimming, he doesn’t boys competed to see who had
feel as though he has hemophilia. the best swim times, diving and
Being in the water is his form of endurance.
meditation. When he’s having a
bad or stressful day, getting in Charlie is pretty competitive, so
the water to swim, surf or playing he really enjoyed this part of the
water polo helps him refocus. He weekend. He tried his hardest,
comes out of the water feeling competed well and was very
instantly better. I am glad that he’s pleased that he came in third
found something to help him cope. place. He won his first big trophy!
It was too big to pack, so he had
The Getting’ in the Game fun-filled to carry it on the plane home. We
weekend included a stay in a hotel are so proud of Charlie and all his
with other kids from the bleeding accomplishments in finding his
disorder community from all over passion! Thank you, CSL Behring,
the country. We don’t live near for giving my son this opportunity!
other children with a bleeding
22 Winter 2018 Factor Nine NewsUPCOMING EVENTS 2018!
MARCH 1, 2018 MARCH 22, 2018 MARCH 23-25, 2018
ETERNAL SPIRIT AWARD DINNER GOLF FORELIFE OUTING 12TH ANNUAL SYMPOSIUM
New York, New York Ponte Vedra Beach, Florida Ponte Vedra Beach, Florida
APRIL 14, 2018 APRIL 19-22, 2018 MAY 3-6, 2018
MEETINGS ON THE ROAD WOMEN’S RETREAT MEN’S RETREAT
Maine Florida Florida
MAY 19, 2018 SEPTEMBER 13-16, 2018 SEPTEMBER 22, 2018
MEETINGS ON THE ROAD MEN’S RETREAT MEETINGS ON THE ROAD
Alabama Arizona Pennsylvania
SEPTEMBER 27-30, 2018 OCTOBER 6, 2018 NOVEMBER 10, 2018
WOMEN’S RETREAT MEETINGS ON THE ROAD MEETINGS ON THE ROAD
Arizona New Mexico and North Carolina Virginia and Colorado
NOVEMBER 17, 2018
MEETINGS ON THE ROAD
California
www.hemob.org Factor Nine News 23You can also read
