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Is Allogeneic Transplantation for Sickle Cell Disease Still Relevant in the Era of Gene Therapy?
Blood Adv
November 2024
Vanderbilt UniversityVanderbilt-Meharry Center of Excellence in Sickle Cell Disease, Nashville, Tennessee, United States.
Article Synopsis
- - Sickle cell disease (SCD) is the most prevalent inherited blood disorder, affecting primarily children in the U.S. and Europe, where treatments have improved their survival rates, but adults still face significant health risks and early death.
- - Recent FDA-approved genetic therapies have shown promise in reducing pain events associated with SCD, while allogeneic hematopoietic cell transplantation (alloHCT) offers a curative option, though both face challenges such as donor availability and serious health risks.
- - Advances in treatment methods are addressing these challenges, positioning both genetic therapies and alloHCT as potential cures, though genetic therapies are more expensive and may have serious long-term side effects compared to alloHCT.