New Publication Answers Questions About the SIPPET Study

Inhibitors are the most troubling complication of hemophilia A treatment today. In this week’s blog, I share with you a new publication concerning the SIPPET study, which sheds light on factor VIII products and inhibitors.
New Publication Answers Questions About the SIPPET Study


An article written by noted hematologist Dr. Flora Peyvandi and colleagues was recently published in the medical journal Haemophilia. This article directly addresses and answers several questions that were raised about the 2016 publication of the Survey of Inhibitors in Plasma-Product Exposed Toddlers, or SIPPET study.1

With this new article, SIPPET: methodology, analysis and generalizability, the authors respond to the 17 most common questions associated with the design, methodology, and results of the SIPPET study, including1:
  • Is the inhibitor risk higher in SIPPET than in previous reports?
  • Could differences in treatments between countries have affected the results?
  • Could the results have been affected by the way the study was randomized?
  • Do the SIPPET results also apply to other recombinant factor VIII (rFVIII) products
    beyond the 1st and 2nd generation products used in the study?
  • Is there a difference in inhibitor risk between the different brands within the plasmaderived and recombinant groups?
Original SIPPET Study
The SIPPET study, conducted by Dr. Peyvandi and colleagues, was the first randomized trial to compare the incidence of inhibitors in plasma-derived factor VIII (pdFVIII/VWF) products and rFVIII products in previously untreated patients (PUPs).2

Results from this landmark study showed that there was an 87% higher rate of inhibitor development in patients who received rFVIII compared with patients who received pdFVIII containing von Willebrand factor (VWF).2

Based on the results of the SIPPET study, the National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC) now recommends that pdFVIII/VWF be considered as one of several treatment options in PUPs.3
Visit www.inhibitorinfo.com to Learn More About Inhibitors
Inhibitors are the most serious and challenging hemophilia A treatment complications. All patients with hemophilia A are at risk for developing inhibitors, regardless of age and disease severity.
Inhibitorinfo.com is a comprehensive website that provides important information and resources about inhibitors and the risk of inhibitors. There is a discussion guide patients can download and use to talk with their hematologists about inhibitors. Visitors can also read about the results of the SIPPET study and watch leading hematologists talk about its implications.
When visitors sign up for updates at inhibitorinfo.com, they will receive access to the full SIPPET study, as well as updates about hemophilia, inhibitors, and the latest clinical data.
References: 1. Peyvandi F, Mannucci PM, Palla R, Rosendaal FR. SIPPET: methodology, analysis
and generalizability [published online ahead of print March 17, 2017]. Haemophilia. doi:
10.1111/hae.13203. 2. Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII
and neutralizing antibodies in hemophilia A. N Engl J Med. 2016;374(21):2054-2064. 3. National
Hemophilia Foundation. MASAC Update on SIPPET. National Hemophilia Foundation website.
https://www.hemophilia.org/Newsroom/NHF-Community-News/MASAC-Update-on-SIPPET.
Published March 9, 2016. Accessed April 27, 2017.
This is a paid public announcement from Grifols and does not constitute an endorsement of
products or services. When you click on the links in this blog entry, you will be directed to
the Grifols website. LA Kelley Communications always advises you to be a savvy consumer
when contacting any company; do not reveal identifying information against your will.
BN/A8/0517/0275

“My bones are older than me”

Prince, age 17
“My bones
are older than me,” lamented Prince, the handsome 17-year-old Rwandan with
hemophilia, who I had met previously three years ago when I first came to this
small, lush country. This night, Wednesday, June 14, I welcomed a few families
with hemophilia, who are the founders of the new Rwanda Federation of
Hemophilia. While all competent professionals, they still need some help in
getting their organization jumpstarted. Imagine living in a country that has no
factor, no hemophilia care, and is poor. Of the estimated 800 people with hemophilia here,
no more than 50 are identified.
But this is all about to change.
We gathered socially in the cool evening on the terrace at the famous Hôtel des Mille
Collines, dubbed “Hotel
Rwanda,” a safe haven during the brutal 1994 genocide, where the manager Paul Rusesabagina saved 1,268 Hutu and Tutsi refugees from the Interahamwe militia. (If you haven’t
seen the movie Hotel Rwanda, I urge you to rent it.) The Mille Collines is
a lovely hotel, and while I write this I hear a spry African ensemble playing traditional
music downstairs with chanting and upbeats that make you want to dance!
Shady Sedhom, NNHF, listens to the patients
Prince is rail
thin, and soft spoken, like all Rwandans. I’m glad he arrived first so I could
get caught up with him personally. When I saw him in 2014, I had arrived for
the first time in Rwanda to assist the new Federation. Prince was a stocky
14-year-old then. Now he was lean and taller, with chiseled features. When I
asked him how he was doing, he replied with a phrase that showed his desperate
plight, and poetic aptitude: “My bones are older than me. I have the bones of a
60-year-old, my doctor told me.” His right knee had given him a lot of trouble
three years ago; now the left one was. When was the last time he went to the clinic,
which was only 1 kilometer from his house? Not in years. Why? “Every time I go
they have no factor.”
I’ve been
trying to keep Rwanda supplied with factor; indeed, we are the only ones who
give them factor. And that’s because they are not yet registered with the World
Federation of Hemophilia. Once they register, they will be eligible for much,
much more factor, perhaps regularly. Getting them registered, both with their
own government and then the WFH was my goal this trip.
With me was
Mr. Shady Sedhom, a registered pharmacist and now program manager with the NovoNordisk Haemophilia Foundation, an incredible organization based in
Switzerland, that provides program expertise, management and funding for
hemophilia organizations globally. This was the second time I would work with
them, but the first time in person. We would give a half day workshop on
Thursday, June 15.
Benis’s knee
But this
evening we were here to meet the board members, and hear their stories. I don’t
want to just give a lecture on how to run an organization: I want to know them,
as people, as families, as families with hemophilia, as blood brothers and
sisters in this amazing global family we have.
Little Benis
I met Vivine,
whose son Ness, now age 8, hemophilia B, had a headache as a toddler, and it
continued on. We listened in somber silence, as she continued. She took him to
the doctor, who tested him for malaria, but this was not it. They gave him a painkiller
and sent him home. The headache continued for two more days, getting worse. She
took him to the ER on a Friday night at the public hospital, but they said they
could not give him a CT scan because it was too late. Come back Monday! In a
culture used to respecting authority and not questioning the medics, Vivine’s
strong maternal instinct won out. She went to the King Faisal Hospital (a
private hospital), which would be prohibitively expensive. She went anyway that
night, and they diagnosed him with a head bleed! He got factor and this saved
his life. Her story highlighted the need for education among the country’s
doctors.
A fun evening with many shared stories
We met
James, age 31, also hemophilia B, who was just diagnosed last year! James is a quiet man, lacking a few front teeth. Indeed, he had persistent dental
problems, with constant bleeding. When the doctors here could not figure out
what was causing this, he finally sent his blood to France to be tested, at
cost of $400! This is a ridiculous amount of money in a country where the average
annual household income is about $700. Especially since it could have been
diagnosed in neighboring Kenya.
Sylvestre
has been my email pal for the past few months as we prepared for this visit. He
serves as Secretary of the RFH. Sylvestre is well known to us in the office
back home as he has requested factor for his son Virgil. Little Virgil,
squirming before me with all the energy of a four-year-old, wears glasses for
his still misaligned eyes. He was blind for 18 months after a coma, due to head
bleed as a two year old, but factor from Project SHARE saved his eyesight. Slowly,
Sylvestre told us, he is getting his eyesight back.
Sylvestre
reminded me of how I got involved with Rwanda in the first place—I had actually
forgotten as we have accumulated so many stories working with so many countries.
A nurse named Tracy Kelly was volunteering in Rwanda about five years ago, and
met Sylvestre as he sought help for little Virgil. She contacted her hospital
back home, which eventually found us. We shipped factor over right away. And
when the crisis passed, I asked Sylvestre to consider founding a national
organization for those with hemophilia. Like many we have met, he agreed. And
here we were.
We finished our
juice drinks and tea and cakes, and then they dispersed into the soft night,
hopeful for the next day’s outcomes.
Vivine adds a needs list
The next day
was our workshop. Shady has a prepared slide deck, exercises and came equipped
with markers, post-its, posters. The attendees arrived early, prepared to work!
Besides the RFH we also had several doctors, which was a high point. Doctors in
developing countries have little free time. Most work at two hospitals and/or
have a private practice. They seem on call 24/7. To have them here was an
absolute honor. The day consisted first of a needs assessment, brainstorming
what Rwanda needs to have good hemophilia care. Each attendee wrote out ideas
on a post-it note then attached it to a poster, under one of four areas of
need. The post-its read: A comprehensive center, training, education of health
care workers and families, diagnosing suspected cases, outreach to find more
patients, public awareness to help find patients, and of course… more factor.
Laurie Kelley with patient at CHUK 
But the
greatest need was to register the RFH with the government. Until it becomes an
official, registered nonprofit, nothing much would happen. The WFH needs it
registered and accountable. Project SHARE will keep sending factor of course,
but we can only do so much. Shady said NHF is ready with funding for a project
to help meet these needs… after they get registered.

 
And an
interesting phenomenon: learning how to challenge each other’s ideas. When
someone offered an idea, such as the most important need was to get more
factor, Shady and I challenged that. Spending your time securing factor is
urgent, especially when your child has a bleed. But allowing the registration
issue to languish means you will only get dribs and drabs of donated factor. Focusing
on registering now will open so many doors later. Short term pain for long term
gain. The RFH was learning now to priorities needs.

After this
we did goal setting, based on those needs. And then prioritizing those goals.
This took over an hour. Later, Shady asked the group to plan a birthday party,
as an exercise in planning a hemophilia event later on (like World Hemophilia
Day next April). It was a fun exercise to see who remembered what action item
was needed. And ironic: that very day, June 15, was Shady’s birthday! Unknown
to him I ordered a cake. And right after the birthday party exercise, we took a
break, and out came a cake with candles! It was fun to all join in and sing him
happy birthday. We thanked him for his dedication for spending his birthday working on Rwanda’s hemophilia future. Then we learned that we had another cause to celebrate: James’s wife had just given birth a few hours ago! But he stayed to complete the workshop with us. Such dedication!
After break
came Stakeholder Awareness, an exercise I’ve never done before so this was
educational for me. Even just learning who to identify who is influential and
how much they were influential—Prince offered the media, which was
brilliant—and others offered families, the public, the ministry of health and
more.
By the end
of the day we had the components of a strategic plan, with action items. It
will take many more meetings to hammer out the details but it was a powerful
five hours. Afterward, we dined outside in the night air and had a buffet
dinner together. Shady had to dash off to catch a flight but the rest of us
relaxed and shared our thoughts on this truly historic day.

We planted
the seeds of growth, and now, it’s up to the Rwandans to take next steps on the
road to better hemophilia treatment care.




And they
deserve it and can do it. Rwanda spends more on healthcare per capita than most
African countries. The country is peaceful, functions well and has infrastructure.
It’s a small country, about the size of Massachusetts, my home state. Best of
all, it has interested and dedicated doctors. All ingredients of success.
There will
be challenges. I read a Rwandan proverb that says: If you are building a
house and a nail breaks, do you stop building, or do you change the nail?
We don’t want them to ever stop building.

A reason to celebrate!
My vision?
To see Rwanda join the WFH, and be present next year at the WFH Congress in
Scotland, where they will meet the world community, and their fellow Africans,
to learn, to share and to get the resources that so many others get. When they
join everyone else, they will then be able to determine their treatment and
destiny, and a whole generation of Rwandans will grow up free of the pain and
disabilities they suffer now.

To see photos of the trip, go here. 

“My bones are older than me”

Prince, age 17
“My bones are older than me,” lamented Prince, the handsome 17-year-old Rwandan with hemophilia, who I had met previously three years ago when I first came to this small, lush country. This night, Wednesday, June 14, I welcomed a few families with hemophilia, who are the founders of the new Rwanda Federation of Hemophilia. While all competent professionals, they still need some help in getting their organization jumpstarted. Imagine living in a country that has no factor, no hemophilia care, and is poor. Of the estimated 800 people with hemophilia here, no more than 50 are identified.
 
But this is all about to change.
 
We gathered socially in the cool evening on the terrace at the famous Hôtel des Mille Collines, dubbed “Hotel Rwanda,” a safe haven during the brutal 1994 genocide, where the manager Paul Rusesabagina saved 1,268 Hutu and Tutsi refugees from the Interahamwe militia. (If you haven’t seen the movie Hotel Rwanda, I urge you to rent it.) The Mille Collines is a lovely hotel, and while I write this I hear a spry African ensemble playing traditional music downstairs with chanting and upbeats that make you want to dance!
Shady Sedhom, NNHF, listens to the patients
Prince is rail thin, and soft spoken, like all Rwandans. I’m glad he arrived first so I could get caught up with him personally. When I saw him in 2014, I had arrived for the first time in Rwanda to assist the new Federation. Prince was a stocky 14-year-old then. Now he was lean and taller, with chiseled features. When I asked him how he was doing, he replied with a phrase that showed his desperate plight, and poetic aptitude: “My bones are older than me. I have the bones of a 60-year-old, my doctor told me.” His right knee had given him a lot of trouble three years ago; now the left one was. When was the last time he went to the clinic, which was only 1 kilometer from his house? Not in years. Why? “Every time I go they have no factor.”
I’ve been trying to keep Rwanda supplied with factor; indeed, we are the only ones who give them factor. And that’s because they are not yet registered with the World Federation of Hemophilia. Once they register, they will be eligible for much, much more factor, perhaps regularly. Getting them registered, both with their own government and then the WFH was my goal this trip.
With me was Mr. Shady Sedhom, a registered pharmacist and now program manager with the NovoNordisk Haemophilia Foundation, an incredible organization based in Switzerland, that provides program expertise, management and funding for hemophilia organizations globally. This was the second time I would work with them, but the first time in person. We would give a half day workshop on Thursday, June 15.
Benis’s knee
But this evening we were here to meet the board members, and hear their stories. I don’t want to just give a lecture on how to run an organization: I want to know them, as people, as families, as families with hemophilia, as blood brothers and sisters in this amazing global family we have.
Little Benis
I met Vivine, whose son Ness, now age 8, hemophilia B, had a headache as a toddler, and it continued on. We listened in somber silence, as she continued. She took him to the doctor, who tested him for malaria, but this was not it. They gave him a painkiller and sent him home. The headache continued for two more days, getting worse. She took him to the ER on a Friday night at the public hospital, but they said they could not give him a CT scan because it was too late. Come back Monday! In a culture used to respecting authority and not questioning the medics, Vivine’s strong maternal instinct won out. She went to the King Faisal Hospital (a private hospital), which would be prohibitively expensive. She went anyway that night, and they diagnosed him with a head bleed! He got factor and this saved his life. Her story highlighted the need for education among the country’s doctors.
A fun evening with many shared stories
We met James, age 31, also hemophilia B, who was just diagnosed last year! James is a quiet man, lacking a few front teeth. Indeed, he had persistent dental problems, with constant bleeding. When the doctors here could not figure out what was causing this, he finally sent his blood to France to be tested, at cost of $400! This is a ridiculous amount of money in a country where the average annual household income is about $700. Especially since it could have been diagnosed in neighboring Kenya.
Sylvestre has been my email pal for the past few months as we prepared for this visit. He serves as Secretary of the RFH. Sylvestre is well known to us in the office back home as he has requested factor for his son Virgil. Little Virgil, squirming before me with all the energy of a four-year-old, wears glasses for his still misaligned eyes. He was blind for 18 months after a coma, due to head bleed as a two year old, but factor from Project SHARE saved his eyesight. Slowly, Sylvestre told us, he is getting his eyesight back.
Sylvestre reminded me of how I got involved with Rwanda in the first place—I had actually forgotten as we have accumulated so many stories working with so many countries. A nurse named Tracy Kelly was volunteering in Rwanda about five years ago, and met Sylvestre as he sought help for little Virgil. She contacted her hospital back home, which eventually found us. We shipped factor over right away. And when the crisis passed, I asked Sylvestre to consider founding a national organization for those with hemophilia. Like many we have met, he agreed. And here we were.
We finished our juice drinks and tea and cakes, and then they dispersed into the soft night, hopeful for the next day’s outcomes.
Vivine adds a needs list
The next day was our workshop. Shady has a prepared slide deck, exercises and came equipped with markers, post-its, posters. The attendees arrived early, prepared to work! Besides the RFH we also had several doctors, which was a high point. Doctors in developing countries have little free time. Most work at two hospitals and/or have a private practice. They seem on call 24/7. To have them here was an absolute honor. The day consisted first of a needs assessment, brainstorming what Rwanda needs to have good hemophilia care. Each attendee wrote out ideas on a post-it note then attached it to a poster, under one of four areas of need. The post-its read: A comprehensive center, training, education of health care workers and families, diagnosing suspected cases, outreach to find more patients, public awareness to help find patients, and of course… more factor.
Laurie Kelley with patient at CHUK
But the greatest need was to register the RFH with the government. Until it becomes an official, registered nonprofit, nothing much would happen. The WFH needs it registered and accountable. Project SHARE will keep sending factor of course, but we can only do so much. Shady said NHF is ready with funding for a project to help meet these needs… after they get registered.

 
And an interesting phenomenon: learning how to challenge each other’s ideas. When someone offered an idea, such as the most important need was to get more factor, Shady and I challenged that. Spending your time securing factor is urgent, especially when your child has a bleed. But allowing the registration issue to languish means you will only get dribs and drabs of donated factor. Focusing on registering now will open so many doors later. Short term pain for long term gain. The RFH was learning now to priorities needs.
After this we did goal setting, based on those needs. And then prioritizing those goals. This took over an hour.
Later, Shady asked the group to plan a birthday party, as an exercise in planning a hemophilia event later on (like World Hemophilia Day next April). It was a fun exercise to see who remembered what action item was needed. And ironic: that very day, June 15, was Shady’s birthday! Unknown to him I ordered a cake. And right after the birthday party exercise, we took a break, and out came a cake with candles! It was fun to all join in and sing him happy birthday. We thanked him for his dedication for spending his birthday working on Rwanda’s hemophilia future. Then we learned that we had another cause to celebrate: James’s wife had just given birth a few hours ago! But he stayed to complete the workshop with us. Such dedication!
After break came Stakeholder Awareness, an exercise I’ve never done before so this was educational for me. Even just learning who to identify who is influential and how much they were influential—Prince offered the media, which was brilliant—and others offered families, the public, the ministry of health and more.
By the end of the day we had the components of a strategic plan, with action items. It will take many more meetings to hammer out the details but it was a powerful five hours. Afterward, we dined outside in the night air and had a buffet dinner together. Shady had to dash off to catch a flight but the rest of us relaxed and shared our thoughts on this truly historic day.

We planted the seeds of growth, and now, it’s up to the Rwandans to take next steps on the road to better hemophilia treatment care.

And they deserve it and can do it. Rwanda spends more on healthcare per capita than most African countries. The country is peaceful, functions well and has infrastructure. It’s a small country, about the size of Massachusetts, my home state. Best of all, it has interested and dedicated doctors. All ingredients of success.
There will be challenges. I read a Rwandan proverb that says: If you are building a house and a nail breaks, do you stop building, or do you change the nail? We don’t want them to ever stop building.


A reason to celebrate!
My vision? To see Rwanda join the WFH, and be present next year at the WFH Congress in Scotland, where they will meet the world community, and their fellow Africans, to learn, to share and to get the resources that so many others get. When they join everyone else, they will then be able to determine their treatment and destiny, and a whole generation of Rwandans will grow up free of the pain and disabilities they suffer now.

To see photos of the trip, go here. 


Personalizing Prophylaxis

Written by Leonard A. Valentino, MD

Originally published in YOU, August 2015
Steve is a lifelong Chicago White Sox fan and rarely misses a home game. For his birthday last year, a friend gave him a team ball cap to replace the one he’d worn for years. The new black and white cap looked cool and had a great brim for blocking the sun. But it was a non-adjustable, “one size fits all” model, and after just a couple of innings, Steve had to take it off because it was tight and uncomfortable. The cap was too large for his daughter but fit his son perfectly, and he became its new owner.
Standard prophylaxis is a lot like that baseball cap. For years, we’ve relied on a one size fits all approach to treatment. For many, these standard regimens, intended to keep FVIII or FIX trough levels above 1% of normal, prevent or reduce bleeding. Other patients may need higher trough levels to prevent bleeds, while some may be overtreated with a standard approach. So, how can we improve the “fit” of prophylaxis and reduce the risk of bleeding or overtreatment for everyone? Make the regimen adjustable by customizing treatment to the individual. In other words, personalizing prophylaxis.
Step One: Assessing Bleeding Risk
To personalize a prophylaxis regimen, your physician must first assess your risk for bleeding.1 This risk is partly determined by your bleeding phenotype—how often you bleed when not on prophylaxis. While many people with severe hemophilia have frequent, spontaneous joint bleeds, some rarely experience bleeding episodes. Your bleeding phenotype helps determine the intensity of prophylaxis. If you have a severe phenotype, you may require a higher dose of factor concentrate and/or more frequent infusions to prevent bleeding.
The types of activities a person participates in may be another risk for bleeding. Although an active lifestyle is essential for maintaining overall health, vigorous physical activity may somewhat increase the risk for bleeding in persons with hemophilia. Personalizing prophylaxis by raising FVIII or FIX levels during certain physical activities may help prevent or reduce injury-related (traumatic) bleeds.
Age at the start of prophylaxis and the presence of a target joint also contribute to bleeding risk. Hemophilia treaters continue to debate the ideal age for beginning prophylaxis. Yet they all agree that it is not a good idea to delay prophylaxis until someone experiences several bleeds— particularly, multiple bleeds into the same joint (a target joint). Once this situation develops, personalized prophylaxis may be needed to reduce a pattern of frequent bleeding.
Finally, FVIII or FIX pharmacokinetics (PK) has a major impact on bleeding risk. PK is a scientific approach to study and describe what the body does to a drug—in this case, clotting factor concentrate. Because this topic is so important, let’s consider it separately.
Your Pharmacokinetic Profile
One of the best tools we have for personalizing prophylaxis is a person’s unique PK profile. This profile, derived from blood samples taken before and after factor infusion, provides information about how your body uses factor concentrate. The main determinants of PK are how often you infuse and how long FVIII or FIX remains active afterwards.1
Following an infusion, factor level increases, with the increase determined by the amount of factor administered and the patient’s response to the infused dose. This information is used to calculate the incremental recovery. For example, a FVIII injection of 50 IU/kg body weight would be expected to increase the FVIII level to 100% of normal values, indicating an incremental recovery of 2 IU/dL per IU/kg infused. This level quickly begins to fall, however, and after about 12 hours for FVIII and 24 hours for FIX, the level drops to 50%.This decline is called factor half-life, meaning that your body has used up half of the infused factor.
Figure 1. Factor VIII Half-Life Levels

Figure 2. Factor IX Half-Life Levels

Although incremental recovery is fairly consistent among persons with hemophilia, factor half-life varies widely. In fact, a clinical study of prophylaxis found that some people have a half-life that is more than 2 times longer than that of someone with the shortest half-life. Personalizing prophylaxis for someone with a short factor half-life may involve infusing more often. Persons who are physically active may also benefit from personalizing prophylactic infusions. This strategy has worked well for several of my former patients.

On the other hand, persons with longer than average half-lives and those who are inactive because of severe joint disease or a sedentary lifestyle may be able to infuse less frequently. Ed* is a retired office worker with hemophilia A who has arthritis in both knees and uses a walker for mobility. He started prophylaxis at age 57 to reduce frequent knee bleeds. Although this regimen was effective, thrice-weekly infusions were a struggle for him, and he often missed doses. We measured his personal half-life and found he was at the high end of the normal range. We leveraged this long half-life and modified his FVIII prophylaxis regimen to every third day infusions. Since personalizing prophylaxis a year ago, Ed’s annual bleed rate has decreased from 24 to 30 to just 2.
Optimizing Success
An essential component of any personalized prophylaxis regimen is good adherence.1
Prophylaxis is unquestionably time-consuming and often inconvenient. However, bleeding resulting from missed doses is even more burdensome and can have negative consequences.
Here are a few suggestions for optimizing your success:
  • Become an educated healthcare consumer by learning about your bleeding disorder and current treatment regimen. Consulting your local hemophilia chapter may be an important first step.
  • Record all infusions in an electronic or paper bleed log and share this information with your healthcare providers.
    Discuss your personal goals and aspirations with your healthcare team, making sure to emphasize how you define a successful treatment regimen.
  • If you have questions about any aspect of the treatment plan, contact your hemophilia treatment center (HTC) and speak with a nurse or doctor.
  • If you experience breakthrough bleeding, immediately contact your HTC, as this may indicate a need to modify the prophylaxis regimen.
  • When traveling, make certain to bring extra factor concentrate/infusion supplies in case of flight delays, etc.
Summing up, one size fits all doesn’t necessarily work for baseball caps or prophylaxis. Personalizing the prophylaxis regimen by taking into consideration your individual risk for bleeding and PK profile is key to ensuring a “good fit” that reduces bleeding while avoiding overtreatment. Once you and your physician have personalized your treatment plan, always infuse on schedule and immediately report any breakthrough bleeding. A little fine-tuning of the regimen to account for changes in your lifestyle may be all that is needed to prevent bleeds.
Dr. Valentino is a professor of pediatrics at Rush University in Chicago, Illinois, and the former director of the Rush Hemophilia and Thrombophilia Center. He is now the global head of Hemophilia & Blood Disorders, Global Medical Affairs at Shire.

Interested in more content like this? 
Click Here to subscribe to our Parent Empowerment Newsletter. 
Free to families with hemophilia
* Names and some details have been changed to protect patient confidentiality.
1. Valentino, L. A. “Considerations in Individualizing Prophylaxis in Patients with Haemophilia A.” Haemaphilia 20(5) (2014): 607–15.

Adoptions: Bringing Hudson Home

This is a beautiful story of an amazing adoption by the son of my dear friend John Parler of Florida. Adoption has always been a cause near and dear to my heart, but one I could never pursue. To me, adoptive parents of children with hemophilia can be our everyday heroes.
John “Poppy” Parler holding little girl
at orphanage

On Sunday,
May 7, after a 24-hour delay and a 14-hour flight from Beijing to Washington,
D.C., I stepped off United flight 808 at 7:15 PM with my third son, Dallas, and
my 14th grandchild – a six-year-old boy with severe hemophilia A –
who his entire life had been known as Chang Wang Le. Knowing we would pass
through immigration and, with one more official stamp on his paperwork, his
name would legally change to Hudson John Le Parler, we would then experience
the long-anticipated meeting with his forever mother, April, and his forever
brother and two sisters. While that moment would mark the end of a trip that
had begun for me over two weeks earlier, it was the culmination of a journey
which had begun, quite unexpectedly, over a year ago.

In April
2016, at the Michigan Hemophilia Foundation’s annual Springfest Family
Education Weekend I met Shari Luckey, the Advocacy and Outreach Coordinator for
the Michigan chapter, and mother of a boy with hemophilia she adopted from
China in 2010.  She shared her story and I
asked her about the need, process and challenges of that type of adoption. Her
answers were filled with passion for helping children with hemophilia overseas get
adopted.

As she
shared her passion with me, I kept thinking of my son, Dallas, and his wife,
April. Dallas is the third of my five kids; he does not have hemophilia, unlike
his two brothers with severe hemophilia A. So he’s no stranger to the
challenges of living with hemophilia. Additionally, he and April always had a family
plan to have four biological children and then adopt one more child, or possibly
two more children. Since they already had three kids at the time, I thought it
might be interesting to pass the information Shari was providing along to them
in case they might have an interest four or five years down the road.

Upon
returning home, I did just that, and promptly forgot all about it. A month
later, much to my shock, in a casual conversation, Dallas mentioned to me that
they had applied to an adoption agency with the intent of pursuing adopting one
of the numerous boys available in China’s orphanage system. And so began their
journey to “Bring Hudson Home.”

Initially
there were reams of paperwork, financial disclosures, personal interviews, home
studies, and fees, followed by more reams of paperwork. It was sluggish and
tedious and I kept waiting for the word that they had decided to put the entire
process on hold. But that word never came. They persevered and plowed through
all of the bureaucratic red tape that inevitably accompanies an international
adoption, and – as they did – they began to accumulate a growing following on
the Facebook page they had established to document their journey. Those
followers came from every conceivable interlocking network of relationships;
their family and friends, friends of family, co-workers, their family’s
co-workers, other adoptive parents, the hemophilia community – the circle of
relationships expanded until nearly 800
people
were following along every step of the way!

In September
2016, the kids received some very
unexpected news—they were expecting their fourth biological child! I was
certain that this would put their journey on hold. I was wrong. By that time,
their adoptive son had already been identified, they knew his story, they were
aware that, in addition to his hemophilia, he had a congenital heart defect
from birth, and any thought of suspending the process would, in their minds, be
abandoning this little boy. So they persisted.

Two
significant challenges faced Dallas and April. One – which had been there from
the start – was the cost of the adoption; approximately $35,000. The other
–  April would be due with their next
child less than two months before the anticipated travel to China to finalize
the process, and unable to make the trip. That’s where I was summoned off the
sidelines and thrust into the middle of the process. The kids asked me to
accompany Dallas to China to provide logistical travel assistance and to help
during the two weeks he would have his newly adopted son with him, traveling
domestically, attending hearings and meetings and living in hotel rooms – with
a six year old who spoke no English. I could not refuse.

Praying for a good infusion!

As our
departure date in mid-April, the financial challenge remained unresolved. While
the kids, through their own efforts and the generosity of others, had already
spent $25,000 on the process, one significant financial obligation remained: a
mandatory $6,000 contribution to the orphanage required by the Chinese
government. I posted about the orphanage donation on the Facebook page
dedicated to Hudson. Within 5 days, that network of interested people generously,
graciously provided every last dime needed to provide that donation!

So on April
21, Dallas and I embarked on our trip to Bring Hudson Home. Words are
inadequate to convey all we experienced over the ensuing two weeks, first in
Nanjing and then in Guangzhou.

On our
second full day in China, we were driven to a building where domestic marriages
and international adoptions are officially transacted. For Dallas, it is known
by adoptive parents as “Gotcha Day”; the day the parents are given custody of
their adoptive child. I had the incredible privilege, not only of meeting my
newest grandchild for the first time, but also of watching my own son’s face as
he met their son for the first time after such a grueling process! It was an
experience filled with absolute, unbridled joy!

Infusion time!
Later that
week, we toured the orphanage where Hudson had lived, and where he received his
bi-weekly factor infusions. We had been pleasantly surprised to learn just
weeks before our departure that he had been  receiving these infusions for the past year.

Dallas Parler and Hudson

But nothing prepared
us for what we witnessed that day. Hudson was receiving bi-weekly infusions
with a standard factor product, about one
half
of the manufacturer’s recommended dosing for a boy his size. It was a
stark reminder that Hudson – and every other boy with hemophilia in the
orphanage system – was woefully under-treated.

In order to
understand fully what we witnessed on that tour, it’s important to understand
the context in which China’s orphanages now operate. Twenty years ago, this
same orphanage would have been filled with healthy little girls, primarily
because of the cultural preference for boys to carry on the family name coupled
with, at the time, China’s one-child policy. Today, however, thanks to the
repeal of that policy combined with a variety of other factors, in the same
orphanage (and in orphanages all across China) 98% of all orphans are children suffering from a wide variety of mental or
physical disabilities, or very expensive medical conditions for which their
birth family has no means of providing.

Proud Poppy! John and newest grandson Hudson
In an
attempt to deal with this changing demographic of abandoned children, China has
created “baby hatches” – tiny buildings at the entrance of every orphanage
where parents can safely abandon children they either do not want, or for whom
they simply cannot care. In 2016, one baby hatch in the Shandong province
received 106 children in the first eleven days it was open – all with disabilities or medical
conditions! Among those being turned over to the orphanages are a growing
number of boys who have hemophilia.

As we walked
through Hudson’s orphanage, we entered a room with about 50 children under age
2. It was a large room with a soft mat floor, across which were scattered
little clusters of nannies surrounded by children. The nannies were doing their
best to hold children in turn and provide some semblance of attention and
stimulation. In spite of their best efforts, there were little ones left to
themselves, some entertaining themselves with well-used toys, others crying
inconsolably, and some just lying staring into whatever abyss their little
minds had created as a refuge for themselves. It was one little girl in that
state who caught my attention, and whom I picked up and held for nearly 20
minutes while she simply stared up into my face. That image – compounded by the
nearly 1 million abandoned children that live in China’s orphanage
system today – is one that will last a lifetime. While Dallas and I were
ecstatic at the thought of his and April’s ability to provide a family, a home,
security, love, hope and a future for Hudson, the reality that surrounded us in
that moment was crushing. It was a heart-breaking, gut-wrenching experience!

The
remainder of our trip was filled with much waiting, vitally important
processing of paperwork and great anticipation for heading home. When we
finally left Guangzhou for Beijing on our return, we were full of adrenalin and
excitement. Then the one flight we were to connect with to return from Beijing
to DC was – after 7 hours of waiting at the gate – cancelled. But 24 hours
later, we boarded that final, seemingly endless flight and we – finally – Brought
Hudson Home!

Parler Family with newest son

The
beautiful, exciting sight of Hudson meeting the rest of his Forever Family is one
I will never forget. It marked the end of one journey and one trip, but it
marked the beginning of a life-long journey for Hudson. I have no idea what
that journey will hold for him, but I do know that because of the compassion
and sacrifice of my kids and their family, and the generosity of so many
strangers who were moved to help, he will be able to face a life where neither his
limitations nor opportunities will be defined by being born with severe hemophilia.

Hudson is
the 21st little boy from China with hemophilia in the past seven
years – that we know of – to be given the opportunity to become part of a
family here in America. Here, they will have access to the best treatment for
hemophilia in the world. And yet, there are more remaining in China – and other
countries around the world – desperately waiting for that same hope to become
their reality.

To learn
about international adoptions and hemophilia, download our PEN article:
 Overseas Adoption: A Child with Hemophilia is Waiting”

To get involved in adoptions, or to consider
one, connect with the hemophilia adoption community on Facebook here:

Hemophilia Adoptions

Coalition for Hemophilia Adoption and Aid 

Hemophilia Advocacy-Hand in Hand International Adoptions

ADVERTISEMENT