Before you blast off back home, touch down at NHF's BDC 2022 Final Night Event, which promises “out of this world” fun. The Saturday night party, which takes place from 5:30p.m.-7:30 p.m. in the Marriott Marquis Houston Ballroom, Salon I-IV, Level 2, will include a live DJ, fun games for all ages, comfort food, an open bar cocktail hour and galaxy-themed drinks for the kids. Houston, there’s no problem here. ■
Candid Conversations
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Pierce pointed out that a safe and effective gene therapy treatment for hemophilia B might be available at soon as next year. “Factor VIII is a different model. It’s difficult to work with. Gene therapy has had its ups and downs. In the phase 3 trials published in the New England Journal of Medicine, it doesn’t look like it’s going to last.”
The gene therapies for hemophilia A and B have had “quite different” results, Reding said. “We haven’t seen scary safety signals in the trials so far. But we don’t know about long-term yet. We’re just going to have to wait for that.”
Reding steered the conversation into the “psychosocial aspect of gene therapy.” “The long-term thing that we’re starting to see: They have very different takes on whether they would do this again. We know that, if you come into gene therapy, no matter how effective, it’s not going to fix those previously existing issues from hemophilia.”
Pierce was able to offer personal insight, having received a liver transplant a few years back, which eliminated his hemophilia. “Before I had my liver transplant, I was living with hemophilia, like many of you. I thought about hemophilia day in and day out, like many of you. I can’t begin to describe the effect it is to have hemophilia disappear from your consciousness. The pain and damage remain, but it’s not bleeding anymore. That changes your whole perspective on life.”
Gene therapy is not yet available for pediatric patients and Rodriguez doesn’t see it happening soon. “We need years of experience in adults to make sure that the benefits outweigh the risks,” she said. “Children will live longer than adults, so if they experience complications, they will do so for a longer period of time.”
There are steps for young adults and children to take now, both Reding and Rodriguez argued. “One of the things that we talked about was to be good to your liver. Alcohol is not so good for your liver. We think the healthier your liver is, the better off gene therapy will be,” Reding said. Rodriquez pointed to obesity and fatty liver as other detriments.
New therapies have left some communities behind, the panelists said. “With all these new therapies, there’s one particular subset of our community that needs something better, and that’s the factor IX folks,” Reding said.
Another overlooked population: women with bleeding disorders. “We, as providers at the family and practitioner level, have to be open with issues of bleeding with adolescents and girls,” Rodriguez said. “If you ask if they bleed heavily or not, that does not help you as a provider. They go undiagnosed because no one took the time to sit down with them and ask questions about their bleeding.” ■