June 2018

Welcome to Massiechusetts!

Randi Clites of Ohio

The US hemophilia community is renowned for being politically active: we’re educated, smart, proactive and dedicated to protecting our need for access to affordable and safe factor concentrate, and medical help from HTCs. No doubt our political savvy was forged in the hellfire of the hemophilia holocaust of the 1980s and 90s, where we fought from the outside, securing justice from government to ensure this never happens again.

But it’s rare that the hemophilia community makes change from inside the government, as members of government.

Currently we have two people that I know and know of, running for offices. 

One is long-time colleague Randi Clites, mother of a child with hemophilia, who is running to be a representative in the Ohio House of Representatives. Randi is currently associate director at the Northern Ohio Hemophilia Foundation, and someone I’ve met at many hemophilia meetings. She is passionate about health care, dedicated, and a true advocate. On June 15 she posted: “It’s official. I am grateful to have earned the Democratic nomination to run in November to become the next State Representative for the 75th House District.” She has a beautiful website that covers her campaign and asks for donations.

I love this post of hers on Facebook from two days ago: “Yesterday I was out door knocking and I stopped at Kent Dairy Queen for a break while it rained. A young man there asked why he should vote for Randi, because I was wearing my campaign shirt. I introduced myself and he had some of the best questions I’ve been asked while canvassing. I am so inspired by our young people. They are paying attention and engaging. My parents didn’t talk to me about politics. I think we need to. Please take a few moments today and talk with your youth and young adult children about their thoughts on politics.”

Bob Massie, gubernatorial candidate, Massachusetts

Canvassing is hard work, as is campaigning. I’ve been trying to help the second person in hemophilia I know, Bob Massie, in his bid to become Massachusetts’ next governor. You may know Bob; he is quite a legend in our circles, though he no longer has hemophilia. Bob was profiled in the movie “Bad Blood.” Bob was also the subject of the 1973 book Journey, written by his parents, Pulitzer Prize winner Robert K. Massie and then wife Suzanne. The book is a gripping true story of Bobby’s life, which chronicled his immense suffering as a child, and showed the origins of his political activism. His parents were politically oriented, especially his mother, who, with her knowledge of Russia, became President Ronald Reagan’s advisor on Russia! His parents also helped pioneer home treatment of hemophilia. His parents are good friends of Al Gore (and the Clintons), and Bob himself has adopted climate issues as a personal and political cause, as well as health care and racial justice. Bob has had long and rewarding careers in many fields, as an executive leader, Episcopal priest, and author. He knows what it is like to suffer, and to worry about paying for health care costs. He has a vision for how our state, and world, can be better.

Massachusetts (or “Massiechusetts” as Bob’s campaign cheekily calls it) is one of our nation’s smallest states, but a leader among states. It is home to the birthplace of our country. It is home to some of the world’s best universities and colleges. It is a medical Mecca for research and hospitals; center of the biotech universe; and has a rich history and culture. It is a multicultural state as we attract so many immigrants, especially from the Caribbean.

But like all of America, we face tough times ahead and need strong leadership. 

It’s heartening to know that people like Bob and Randi are sacrificing so much personally to try to represent us in government. On Thursday night I held a fundraising get-together at my home, where members of our community and my neighborhood were able to meet Bob personally, and hear his platform on health care, transportation, and economic reform. Bob had just been in Springfield the night before, a two-hour commute, and had actually just met my mom!

I’ve known Bob for 30 years, which is a story for another time. (Meet Bob here) But that night, someone asked him where did he get the energy, at age 60, with all that he has been through, to keep up this pace, to sacrifice so much, to meet and greet the thousands of people has this past year. “I think it’s because I missed so much as a child, and was in bed so much, that I just love people and getting out to meet them!” he replied. I’ve heard other guys his age say much the same thing.

We are all so grateful to people like Randi and Bob, for their dedication and sacrifices, to hopefully bring some compassion and leadership back into government, and to especially keep in mind those with chronic disorders like hemophilia, who need insiders in government, more than ever. Good luck to both!

To donate to Bob, visit:    https://secure.actblue.com/donate/bobmassieforgovernor2018
To donate to Randi, visit: https://secure.actblue.com/donate/randi

Why Plasma Matters

I attended the Plasma Protein Therapeutic Association (PPTA) Forum in Washington DC this past week, which is always a stimulating gathering of bright people in the plasma industry. The theme of the meeting was the launch of the campaign, “How is Your Day?” Every session started with this friendly question, which had the effect of the audience responding back, “Great!” 

The meeting was a call for global access and availability of treatment, and the need for policies to improve patient care. Jan Bult, president and chair of the PPTA, reminded us that “plasma derived products can only be made if we have donors.”

While much of the US uses recombinant factor products, plasma products still play a significant role for many people with hemophilia, particularly those undergoing treatment for inhibitors, older patients who grew up on plasma-products and are reluctant to switch, many patients with von Willebrand disease, and above all, those in developing countries, where recombinant products are too expensive.

Jan reminded us that the goal and need is high quality source plasma from healthy, compensated donors.


David Bell, executive vice-president of Grifols and chair of the PPTA global board of directors. commented in his opening remarks that derogatory comments have been made about the plasma industry. Yet there has been no viral transmission for more than two decades. PPTA seems constructive dialog with patient organization and policy makers.  He praised Jan Bult for his long-term efforts leading the PPTA and always trying to push for improvements in treatment.

Anthony J. Castaldo, president of the US Hereditary Angioedema Association

The keynote speaker was someone who left me amazed and inspired. Anthony J. Castaldo is president of the US Hereditary Angioedema Association. Hereditary angioedema (HAE) causes unexpected horrific swelling and excruciating pain. Tony is a patient himself.  It is more rare than hemophilia, and affects one in 30,000. Untreated, the mortality rate is a stunning 30%! It’s a pediatric disease. Tony made it clear that this is not allergic angioedema—swelling in the throat, for example, due to a food or drug allergy. But HAE is often misdiagnosed in ER, where the patient will be given antihistamines— but these don’t work. 

I had never heard of HAE, and clearly there were many parallels to hemophilia as a chronic disorder and the need for advocacy. It was interesting to see that factor XII is involved in the disorder. Tony showed photos of patients, before and after an attack, which were devastating! Previous to current plasma-derived therapy, patients were given toxic anabolic steroids. Tony showed a video sharing patients’ stories, and I felt the way many people unfamiliar with hemophilia must feel when they see our videos. You want to cry for the people who suffer so much!

One woman in the video commented on the excruciating pain, of almost dying… a laryngeal attack that closed her throat was one of worst. She said, you’ll never know when you’ll have an attack, how far it will go, and if next attack is your last one. She suffered high anxiety in between attacks. In a poll, 100% of patients felt it prevented them from attending school and work. Each year they had between 20-100 days of incapacitation.

Tony’s own 5-year-old daughter started having attacks, including weekly abdominal attacks, which are torture. His daughter grew emaciated, depressed. When she then got the C1-inhibitor plasma-derived medicine, he watched in two weeks as his daughter transformed. Tony spoke with passion and commitment, reminding me of so many in our own community!

He closed by saying, “Plasma products are life-saving and life giving. We appreciate what industry does, what donors do— we get it; we love it! We as a patient community want to protect the gains we’ve gotten.”

So do we in the hemophilia community. Plasma-derived products still have their place in our community. If you can, donate blood. It’s the basis for plasma-derived products… and all of life.

Are you enrolled in the Patient Notification System? If not, you should be. PNS is an alert system to notify you when or if your factor product has been withdrawn or recalled from the market. This is an essential program, developed by manufacturers and operated by the Plasma Protein Therapeutic Association (PPTA).

A Facebook Group that Works!

Milena Pirnat and Laurie Kelley

Milena started her talk by reading a post by one mother, about how devastated she felt at the diagnosis of hemophilia, and how much Hemophilia Mother had helped her–more on that letter in a bit.

Milena started Hemophilia Mother in 2011, when her son was 20. Her son with hemophilia–there’s the connection! Of the 6,328 members, 4,526 are “active,”  members.

Mileana added: 3,158 members are from the US.  531 are from India. 69.5% of members are women, and 30.5% men! These include fathers of children with hemophilia and men with hemophilia.

Top cities where members live are: Dhaka, Bangladesh 62; New York 50; LA 42; London 42; Chicago 39; Karachi 29.

More interesting than all the stats is Milena herself. Obviously not from Canada, with a foreign accent. She is a doctor, from Sarajevo, capital of Bosnia and Herzegovina. She survived the siege in 1992, which she recounts online:
“Some 10,000 civilians, including 1,500 children, were killed in Sarajevo during the 44-month-long siege, mostly by snipers and mortars fired from mountains surrounding the city. UNICEF reported that of the estimated 65,000 to 80,000 children in the city, at least 40 per cent had been directly shot at by snipers; 51 per cent had seen someone killed; 39 per cent had seen one or more family members killed; 19 per cent had witnessed a massacre; 48 per cent had their home occupied by someone else; 73 per cent had their home attacked or shelled; and 89 per cent had lived in underground shelters. We lived in a besieged city for 31 months with a toddler, without food, electricity and under constant shelling! And we were somehow spared? Survivor guilt mixed with hemophilia mom guilt stayed tangled in my brain for many years to haunt me more than I would ever want to admit.”

Incredible movie about the war in Sarajevo that Milena fled from

Milena emigrated to Canada and settled in Manitoba. Her son was diagnosed with hemophilia at age 6. She was very much alone, with trauma embedded in her memories. It was a difficult time. 

“All our struggles to survive the Bosnian war during the siege in Sarajevo, immigration and my recent diagnosis of a chronic condition were nothing compared to the feeling of loss after hearing our son’s diagnosis. Was it because we were both doctors unaware that he had an underlying condition, or the fact that I didn’t know I was a carrier, or that we were alone in that suburban apartment far away from our family and friends that day? I don’t know and I will never know but it hit us harder and deeper than my understanding goes. I went to my son’s room, I hugged him, I squished him tighter and longer than he wanted. He looked at me with his innocent smiling eyes. He had just turned six.”

Hemophilia Mother was born. And here she was today, sharing her story and her Group with a rapt audience. She closed her talk by finishing the letter from the mom; the mom at one point was seriously considering to take her own life, so severe was her depression. But the support she received from others in the group made her want to live again; the community members gave her hope.

Coming home, reading my notes from that day, I had jotted down what the CEO of Sobi, Guido Gelkers, said when speaking about the 1 billion IU factor donation made by Biovertiv/Sobi to the WFH, referring to those who live in developing countries: “When you sit in your office, you forget what others’ lives are like.”

Hemophilia Mother knows what the lives are like of anyone in any country struggling with hemophilia. Members live it every day. Join it: give advice, get advice.     And be supported and inspired. Thank you, Milena, for your service and your inspiration!

South Africa: Self-Advocacy at Work!

Reeshen Pilay of South Africa

I returned from a three-week visit to the United Kingdom just Saturday night. A visit to Yeovil, England first, to visit my dear friend Bill Boughton, who just turned 95! I try to visit him every year or two. How do I know him? You can read about him here!

Then off to Shetland, Scotland, to visit another dear friend, Jan Howard. I wrote about my visit there on May 20.

Then off to the World Federation of Hemophilia Congress in Glasgow! I attended quite a few sessions and had many meetings. All were interesting and worthwhile. Last week I wrote about the State of the World address by WFH president Alain Weill.

But one of the best sessions was by a young man with hemophilia from South Africa. Reeshen Pilay has severe hemophilia A, and for 11 years used cryo, which carries the risk of blood borne diseases. Now 25, he has been using plasma concentrates. His remarkable presentation was on self-advocacy, and how he was able to get the government of South Africa to approve his special request for recombinant products. Imagine having to self-advocate for that, something that is the accepted standard in the US.

A driving force behind this was simple: his veins could not withstand the huge diluent volume for each infusion. He wanted a product with smaller diluent size, for a faster infusion and less wear and tear on his veins. He got to work.

He learned that in South Africa, a staggering 97% use plasma-derived factor concentrate. It would be difficult to get the country’s health care system to accept his proposal to use a recombinant.

He knew he needed to present facts, not just rhetoric to the socialized health care system of this African nation. So he used the WFH eLearning platform. I was intrigued to know what he did and how he did it. First, he connected to the WFH Online Registry of Products. He learned that the way the WFH calculates how much factor is needed in any country is to set a minimum standard of 1 IU per capita, that is, 1 IU per person. According to the WFH’s Annual Global Survey 2016, a survey of 91 countries showed an average factor VIII usage of .82 IU per capita.  

From the WFH Annual Global Survey 2016

He did incredible research, leaving the audience fairly stunned! He found out which product used the smallest diluent. Reeshan had difficulty with the large volumes used by the plasma-derived product provided by the government. After finding the product with the smallest diluent, he then found where his country stood via others by IU/capita. It uses slightly more than 1 IU factor VIII per capita. (In the US, we use a whopping 9.5 IU per capita)  Then, he calculated how he could actually save money by using the smaller diluent size (which also happened to be an extended half-life product), improve his lifestyle and save his veins.

He presented his findings in a PowerPoint presentation to the health care system managers, won their attention and admiration and secured their approval.

He surely won our admiration for his persistence, and for his rational, economic approach to a huge obstacle!

Be sure to check out the WFH’s eLearning platform, which is filled with excellent webinars, booklets to download, slide shows and more.

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