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.
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).