May 2018

WFH: More Factor, More Treated Patients… Now

The World Federation of Hemophilia convened its biannual Congress this week in Glasgow, Scotland, for approximately 4,000 attendees from 120 countries. It is an amazing mix of cultures, languages, disciplines—all for those with bleeding disorders.

WFH President Alain Weill

This was my 12th Congress, and I am always amazed to meet up with people I’ve known for over 20 years now. We are all still fighting the good fight, in trying to bring advanced care and relief to those who suffer with bleeding disorders in other countries.I have so many notes from so many sessions, that I’ll need to read them out over a few blogs. Always fascinating is the opening night, which sets the tone for the Congress. The address is by the current president, in this case, Mr. Alain Weill, father of a son with hemophilia. The audience was packed with people and a hush fell over us as Alain took the stage.

Alain was rightly proud to share the accomplishments of the WFH. From 2004-2016 over 70,000 people with hemophilia were identified, representing 16 new patients a day. He described the State of the World of Hemophilia: out of the 7 billion people in the world, .5 billion are located in high income countries (with .6% growth annually), 1.5 billion are in middle income countries (1.2% growth annually), and 5 billion are in low income countries (with 2.7% growth annually). These are startling statistics: most people live in low income but high growth countries, where of course, we will find the most number of people with hemophilia.  Except that we cannot find them. We lack diagnostic equipment, trained lab staff and funding to fund them. And 42 babies will be born daily with hemophilia… with most in the low-income countries.

So Alain put forward two Calls to Action. The first was a call to develop a diagnostic test for hemophilia. Something like a test used to check glucose for those with diabetes. Imagine just pricking your finger and learning whether you have hemophilia! This may not seem a priority for those of us in developed countries but for those in developing countries, it would be a revolution.

Laurie Kelley with Andrea Trinidad Echavez of the Philippines

The second call to action pertains to commercial clotting factor (CCF). Basically, most poor countries lack access to factor. The WFH measures how much factor a country uses by how many international units (IU) are consumed per capita (per person). Alain mentioned that 55 countries use less than 1 IU per capita, far below the recommended amounts. With 12 billion IU sold in 2016, and assuming that the world population of hemophilia used 1 IU per person, Alain noted that industry would need to increase production of factor by 21 billion to reach all people with hemophilia. Is this realistic? he questioned. Can pharma do this?

It’s Scotland!





This led to his second Call to Action, a series of changes to factor and its production:

  • Increase donations of factor. (Bioverativ/Sobi have pledged 1 billion IU of factor over 10 years. Grifols has committed 200 million per year. CSL Behring has donated substantial amounts. Where are the rest of the pharma players?)
  • Track the vast volumes of unused donated plasma and turn it into factor.
  • Increase existing production capacities
  • Create new manufacturing facilities
  • Make factor accessible and affordable
  • Make gene therapy (when it arrives) accessible to all

And finally he offered a stinging comment about pharma companies that sue one another (this would be pointed directly at Shire): end the legal actions against one another, especially if they block innovative therapies.

Alain urged a task force at once to review these and implement them. I’d like to be on that task force. These are action items I have been working for the past 20 years. Free factor to the poor, more factor and accessible factor for all… not just the lucky ones, like us.

Shetland: Hemophilia at 60° North

Croft House
Two Hemo-Moms!

The Shetland Islands are a wind-swept, treeless, group of islands belonging to Scotland, that are carpeted with lush green, rolling hills, rugged rocks and daring cliffs that overlook the crashing North or Atlantic Sea. They are enchanting and idyllic. Having the best of the modern world—airport, hospital, cable TV and internet—they preserve a way of life not seen often in modern society. Community is paramount; civility the glue that holds communities together. You immediately take a deep breath of fresh sea air, and slow down your pace to enjoy a land where the surrounding sea seems to buffer you from cares and woes elsewhere. 

Their history is more about Nordic explorers than Scotland proper. The Viking invasion began around 800 AD, and Shetland over time became a strategic location for explorers, shippers and eventually maritime activities during the World Wars. The Norse dominated Shetland for 200 years until 1468, when James III of Scotland married Margaret, a Danish princess. As the Danish struggled to create a dowry for the princess, Shetland was mortgaged to Scotland and finally annexed in 1471. Despite that, about 60% of Shetland men stem from Norwegian (perhaps Viking?) heritage.


My trip to Shetland actually began many years ago, when a mother from Shetland contacted me about her son with hemophilia. To assist answering her questions, I shipped her a copy of Raising a Child with Hemophilia, and our children’s books, and some things for the children, at that time numbering six—all free of charge. And so began a sweet pen pal relationship. Jan Howard is a thoughtful sender of cards, notes and photos of the children. I also love to send things by mail, so we exchanged birthday cards (home made by the children), Christmas cards and regular greeting cards. Jan always suggested I come visit and I wanted to. I had read about the Shetland Islands since a child, because of the Shetland ponies. And Jan ensured I read more by sending me books, some of which have become my favorites!

The World Federation of Hemophilia Congress is being held in Glasgow, Scotland this week, and this decided it for me. I would take a side trip before the Congress and fly to Shetland to visit Jan and the kids.

To read about a place is one thing; to visit, a lifelong memory, an experience that embeds itself in your heart and soul. What is there not to like about Shetland? With a population of only 30,000, people are polite to one another consistently, the pace of life is moderate and steady. The air is fresh, the countryside, though devoid of anything tall, and barren of trees, is stunningly beautiful. Stone cottages and stone walls landmark the horizon. Blue sky meets green land, which borders the ever-moving ocean. I was warmly greeted at the airport by David and Sandra, Jan’s parents, and Abigail, Jan’s 16-year-old daughter, who has mild factor VIII deficiency. Two other daughters have low levels of VWF.

A short drive over winding roads in the open countryside and we arrived at Jan’s pretty home, perched not far from the ocean. First order of business was to walk the gorgeous border collie Eddie, who enjoyed a brisk romp on the nearby beach! I worried even whether the children would understand me as my accent is different, but the Shetland accent is not like what you think of as a typical Scottish accent. It sounds more neutral, with some Welsh accent mixed in. In short, it’s delightful to hear!

Laurie Kelley and Jan Howard at Sumburgh Lighthouse

I come from a big family myself, so to be surrounded by the loving and delightful chaos of laundry, snacks, board games, homework, happy dog, beady-eyed hamster, and chatty children were a throwback to my childhood. I lost 10 games of UNO with seven-year-old Talia. Abi and I belly-laughed  recalling hw much we enjoyed the movie Deadpool. Hillel and Talia peppered me with questions about what’s on the American dollar bill.


We had a lovely visit to the Sumburgh lighthouse, an incredible feat of engineering. Lighthouses are vital to Shetland and Scotland’s safety and made a great contribution to saving thousands of lives through the years. Jan sent me The Lighthouse Stevensons, about how the lighthouses were designed and built by the family of Robert Louis Stevenson, one of my favorite childhood authors. Excellent book! We visited a croft house, representative of the way inhabitants lived centuries ago. We spotted puffins and guillemots on the cliffs in Sumburgh, with the birds nestled into their small coves like people in a high-rise condo.

Meeting with medical team in Lerwick

I gave a talk on hemophilia and our charity work to the medical staff at the Gilbert Bain hospital in Lerwick, where Jan works as a pediatric nurse.


And we visit Yoel, 19, who has hemophilia, in Scalloway. What is it like having hemophilia and living on an island? There’s a hospital in nearby Lerwick; Yoel does receive excellent factor products, all paid for by the National Health System. One look at this over 6 ft lad is to see that he has received excellent medical care. His joints are in good shape. He is charming and fit. We walked about Scalloway, a quaint seaside community, and scaled the stone steps of Scalloway castle. While Yoel gets good medical treatment, he would still be reliant on an emergency helicopter ride to the mainland if needed. And the ambulance service in Shetland is… well, slow. There is a lack of community in hemophilia; there is only one other known family with hemophilia. I met the mother, Joanne, at the hospital, and gave her my book. It would be hard to start a chapter there, as there are only the two families.

But the families search the internet to get info and make contacts, have a great relationship with their hospital team, know the Haemophilia Society in Scotland, and Jan is attending the WFH Congress this week, for the first time. In fact, it’s her first time ever attending anything in hemophilia!

Scalloway Castle

We flew down together yesterday, and she was excited. While the five children (Yoel lives independently; Hannah is away at school) stayed with Sandra and David, the grandparents, Jan took a week off to be with her other “family,” hemophilia.

Laurie Kelley with Yoel Howard

Did I mention she is a single mom? Single, nurse, mother of seven (three of whom have bleeding disorders), living on a remote island. She also, incredibly, sponsors two children in Nepal with Save One Life.  If she doesn’t get Mother of the Year, I don’t know who would. I’m so incredibly happy to have visited her special community and to be in her country. I’m honored to have been with her family, and proud she is part of our bleeding disorder family.

You can read more about Jan next May, in PEN’s special issue on Island Life and Hemophilia.

For Mother’s Day: Some SMART Women!

Women are often the frontline defense forces in hemophilia at home. Not only do they carry the child and give birth to him or her, but they must learn about hemophilia, ensure their child gets good treatment, learn how to infuse, worry endlessly and love eternally… I know, as I have been there too!

In honor of Mother’s Day we’re sharing this article from PEN (February 2018), which highlights some amazing moms! Happy Mother’s Day to all the amazing mothers of children with bleeding disorders, and any chronic conditions! I hope you receive the special treatment this day you so deserve.

by Jess O’Donnell

From personal to professional, the women profiled here turned their connections to hemophilia into real, life-changing achievements. While many of us set out to complete a task, we may not label it as a goal or plot out a deliberate path to achieve it. But each of these women had a vision, implemented a plan, and plotted a path to reach her goal.

Each woman’s strategy was unique, but all the goals had something in common: they were all SMART goals. SMART goals are specific, measurable, accountable, realistic, and time-bound. Learn how Mischante, Angelie, Darlene, and Christy and Elise worked hard to get results.

A Better Future for Her Family
After Mischante Cortez’s son, Adam, was diagnosed with hemophilia in 2006, Mischante decided to set a goal: to become a registered nurse to better advocate for Adam’s healthcare needs. It wasn’t easy. Pregnant at age 16, Mischante had dropped out of high school. So before starting her nursing program, she needed to obtain a GED and enroll in community college. After receiving her GED and being accepted into a nursing program, Mischante divided her goal into smaller, specific steps.But her path wasn’t without obstacles. While enrolled in the nursing program, Mischante was often left with no choice but to bring her three young children to school with her because she lacked childcare.

Despite the setbacks, Mischante persevered. “I continued to strive toward my goal to be a nurse while raising three kids alone and working full-time as a certified nurse’s assistant [CNA]. In May 2010, I graduated from college with my nursing degree. It was the best decision I ever made for my family.” Today, Mischante supports her family as a working nurse and single mother. “I am Adam’s number-one advocate, and I get to heal people for a living. I am so grateful that Adam’s diagnosis of hemophilia was exactly what I needed to get into gear.”

A Son’s Diagnosis Prompts a Family Goal
When Angelie Garcia’s son Zayden was diagnosed with hemophilia, Angelie recognized that he was unhappy while being infused with factor. So she told her husband, “Once we’re taught by home nurses, within two weeks we will start involving Zayden.” Then Angelie set a goal to have Zayden take an active role in the infusion process within one year. Angelie and her husband divided their goal into small, attainable steps: the first was to make sure Zayden wouldn’t be afraid of infusions.

On their path to achieving their goal, the family’s largest setback was their location. “Our hometown hospitals need education on hemophilia,” explains Angelie. “I’m the only one here that can infuse my son, and it terrifies me because they don’t know about his condition and we are three hours away from our HTC [hemophilia treatment center].” Angelie learned very quickly that she had to be Zayden’s primary advocate for his hemophilia care. Teaching him to help in the infusion process empowered Zayden, now four years old, to be more aware of his condition. His proud mom says, “He is learning ab­out his condition and will tell you what he can and can’t do.”

Lobbying for Specialized Emergency Treatment
Darlene Shelton founded Danny’s Dose Alliance after her ten-month-old grandson was diagnosed with hemophilia. Although Danny always had his factor with him wherever he went, Darlene’s family learned that paramedics and ER workers were not allowed to administer the factor due to conflicting treatment protocols and liability fears. So Darlene sprang to action and formed Danny’s Dose with these goals in mind: (1) Raise awareness about the current gap in emergency treatment for people with rare diseases, chronic illnesses, and special medical needs; and see current protocols amended. (2) Assist with specialized education for EMS and ER personnel. (3) Provide education for affected families on how to better advocate for their treatment needs. 

The organization’s first goal was to amend protocols in the Sheltons’ home state of Missouri within two years. Darlene says, “Goals can be lofty, but I believe if you don’t set goals high, it removes some of the urgency.” After their proposed legislation was passed in Missouri, Darlene looked forward to their next goal. “We passed EMS legislation, assisted with the beginning of paramedic education, and are helping that grow in 2018 and beyond. Of course, we can’t be sure we will meet our five-year goal, but it looks promising, and we are determined to push hard.” Darlene hopes to have improved treatment protocols for ER and EMS in place across the entire US within five years of their first state, Missouri.The work of Danny’s Dose doesn’t just benefit people with hemophilia. “It benefits all individuals with special medical needs,” says Darlene, “covering rare disease and chronic illness. This covers those with rare, lifesaving meds, those with particular treatments based on their illness, and those with complex medical devices like trachs and heart-pumps.”

Never Too Early to Start!
It’s never too early to start teaching our children the importance of setting goals and achieving them. Christy VanBibber’s eight-year-old daughter Elise was disappointed by her neighborhood’s lack of knowledge and awareness of hemophilia. When Elise began sharing the news about her baby brother Timothy, who has hemophilia, she realized that many people didn’t know what hemophilia was. “She came home with a real concern,” Christy notes, “and asked, ‘Mom, why does nobody know what hemophilia is?’”

 Elise made it her goal to generate awareness in her community about hemophilia—by making and selling beaded bracelets and then donating all proceeds to the Southwestern Ohio
Hemophilia Foundation.

Christy recounts the initial success: “She made some bracelets and asked me to put them on Facebook. We decided to sell them for $3 each. Immediately she sold ten!” Elise went on to sell her bracelets at two local farmers’ markets and local football games.

Christy was thrilled. “She raised $880! Also, people recognize Elise and Timothy at school and the grocery store, asking questions and praising her for such a great job. We are really proud of her!”
All these women—and one young girl—achieved their goals fueled by close-to-home, real-life concerns, and you can too! If you want to learn how you can reach your goals this year, check out our cover story and find out how to make your goals SMART.


HFA: Together We Rock!

Laurie Kelley and Chris Seistrup
Laurie Kelley with Chris Seistrup, a cyclist who will raise money for Save One Life

The Hemophilia Federation of America sure knows how to throw a party! And a three-day meeting, all aimed at educating the community about bleeding disorders while providing a busy but intimate venue for meeting up with old friends, acquaintances and business colleagues. And held in Cleveland, with a final evening a celebration at the Rock and Roll Hall of Fame, the theme “Together, We Rock” was absolutely true and we did!

I was able to attend some sessions while there, in between visiting our Save One Life booth and greeting sponsors, donors and interested people, and chilling with some very great friends (Debbi Adamkin, Neil Herson, Ray Dattoli, Melissa Compton, to name only a few). First was an invitation-only meeting with Genentech about mlibra, and specifically the recently reported deaths. About 20 consumers, all key opinion leaders associated with hemophilia organizations) attended. I won’t go into that here in much detail, as the meeting completely corroborated with the article Paul Clement wrote in the May issue of PEN, available on our website.


Laurie Kelley and Liliana Gomez, with her award for her work in Puerto Rico

In a nutshell, though, Genentech shared how Hemlibra has been granted Breakthrough Designation Status by the US FDA to fast-track approval of Hemlibra for noninhibitor patients, based on the HAVEN 3 study data. Conversation turned to the five deaths, out of the 600 current users of Hemlibra globally. Genentech walked us through each death, one by one, what was known and what was reported. We now know that two were Americans, one of whom was in a clinical study. While the explanations all made sense, Ray Datolli, a COTT representative, remarked, “It’s always a clinical study, and it’s always 1981.” The other attendees also commented that the delay in learning of the five deaths was a problem; the community wants to know sooner about these “adverse events.” Genentech then reviewed why they cannot comment on the deaths, for reasons we spell out clearly in our article, and in some cases, the families of the deceased themselves did not want the information released.

Debbi Adamkin, executive director of the Florida Hemophilia Association commented too that the community needs to be educated that Hemlibra is not a cure, and many think it is!

Laurie Kelley with Crystal Higgins and Tracy Farmer

To learn more about Hemlibra, please read our article in the May issue of PEN.

Another good session was on inhibitors, by Vanessa Byams of the CDC. We learned that the CDC hosted a second national inhibitor summit in January 2017 and is closely following inhibitor trends in our community. Its goal is to share information from the community, and to discuss how to collect high quality data, to make sure inhibitor tests are accurate. NHF’s MASAC has an Inhibitor Prevention and Eradication Working Group, which has led to a national collaborative effort to increase enrollment of babies with hemophilia and inhibitors in the CDC Surveillance study. The bottom line is, all hemophilia patients are at risk of inhibitors (with certain exceptions) and there is a lot of research happening on inhibitors. Vanessa said that in the last 18 months alone there is “an incredible movement and action” regarding inhibitor research. “The road to inhibitor prevention starts with national surveillance, including regular screening and early detection If you detect it early, the better the chance of eradicating it.”

One clear hole in our weekend was Barry Haarde: we were all missing his presence, as HFA was truly a home away from home for him. His sister Emily came to fill in for him, and we all shared Barry stories, while admiring his contributions to HFA and Save One Life, which helped so many around the world. With sadness and sweetness, we all felt his absence all too well.

Metallica fans rock!

On Saturday morning, Novo Nordisk sponsored a “B Yourself” symposium for families with hemophilia B. It was very lively, as the audience could participate in answers using the buttons at their desks; answers were tallied and displayed instantly on the screens, from tables designated with rock and roll names: The Rolling Stones, the Beatles, Prince, etc. It made it all the more fun. The topic was mostly about pharmacokinetic (PK) testing, with different patients sharing about their different half-lives, and different dosing schedules. It got pretty technical but was fascinating. It really is imperative that every person with hemophilia know their PK levels, because we now know that you cannot dose just by weight and correction percentage—you do need to know your half-life. We will have some great articles about that in the November issue of PEN.

Laurie Kelley and Pat DeRatto, long time
friends and hemo-moms!

I spoke with dozens of people, all involved in the community, from patients, to advocates, to chapter leaders to industry reps. Two main themes really became clear, one scary and the other exciting.

Scary? Funding in the community is being restricted. Every executive director of a chapter I spoke to commented on this. Our pharmaceutical sponsors are pulling back funding, perhaps as the marketplace is glutted with products, and we expect some consolidations and acquisitions (like Sanofi buying Bioverativ, and Takeda making moves on Shire?). No one is sure what will happen, but some programs may need to be cut.

Exciting? Gene therapy. More than one person said to me, “It’s not a matter of if anymore, but when.” Gene therapy is coming. How will it impact our lives, both as patients and as community employees?

We don’t know yet, but as we rocked the night away Saturday night to a great band at the Rock and Roll Hall of Fame, we celebrated the great advocacy and grassroots work of HFA and its team, celebrated being a family of people with bleeding disorders, and excited about what the future holds.

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