Goal 2: Reduce Human Disease

What is the place of curative therapies in the management of Sickle Cell Disease

Advances in the care of pediatric patients with sickle cell disease ( SCD) have resulted in improved survival to adulthood.However, adulthood is marked by rapid disease progression, impaired quality of life and premature mortality. Hematopoietic cell transplantation(HCT) from matched sibling donor has curative potential, but has been offered mainly to children. Refinements in the conditioning regimen, supportive care, use of alternate donors and potentially, gene therapy enhance applicability of curative therapies (CT). Absence of a mechanism for conduct of large comparative clinical trials of CT and standard care, and study of impact on organ function, functional outcomes, and fertility is a serious impediment to progress in the field.

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Idea No. 482