Colleen Kelly: Bridging the Access Gap in Gene Therapy for Sickle Cell Disease
Colleen Kelly, Attending Physician and Pediatric Hematologist/Oncologist at Dana-Farber Cancer Institute and Boston Children’s Hospital, shared a post on LinkedIn about a recent article she and her colleagues co-authored, published in Pediatric Blood and Cancer, adding:
“Our new paper is out in Pediatric Blood and Cancer.
Gene therapy can be a transformative treatment for sickle cell disease. But its resource- and time-intensive nature may limit who can actually access it.
In our study, 60% of families with a child with sickle cell disease faced material hardship — food, housing, utility, or transportation insecurity — in the last year.
Only 40% of parents felt they could access gene therapy with their current resources. And families facing material hardship were far less likely to report being able to access this treatment, with 95% lower odds after adjustment.
A transformative therapy is only effective if families can reach it. As these life-changing but resource-intensive therapies expand, access must be part of the design.”
Title: Out of Reach: Understanding Resource Barriers to Gene Therapy for Pediatric Patients With Sickle Cell Disease
Authors: Colleen A. Kelly, Morgan A. Paul, McKenzie Griffin, Sunyu Kang, Rahela Aziz-Bose, Leanne Duhaney, Callie Fry, Puja J. Umaretiya, Kristine Karvonen, Erica Esrick, Sharl Azar, Natasha M. Archer, Kira Bona
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