New curative treatment for sickle cell disease shows promising results in international trial

Vanderbilt University Medical CenterJun 25 2024 Vanderbilt University Medical Center researchers are touting data from a multicenter, international phase 2 clinical trial showing a new, curative treatment for sickle cell disease .

DeBaun and Adetola Kassim, MD, professor of Medicine and clinical director of the Adult Stem Cell Transplant Program at VUMC, led the international trial that included 32 children and 38 adults from five countries.

The haploidentical transplant protocol only requires a half match, which can be from the mother, father, siblings, and even children, and is nonmyeloablative, an approach that typically preserves fertility. Other study findings revealed 96% of participants were off immunosuppression one year after transplant. The overall two-year survival rate was approximately 95% in children and adults, similar to the results in most SCD curative trials. All eight graft failures occurred in the children; however, the children's bone marrow recovered completely after the transplant and did not require additional treatment.

Source: Education Headlines (educationheadlines.net)

Sickle Cell Disease Blood Bone Bone Marrow Bone Marrow Transplant Children Clinical Trial Cyclophosphamide Efficacy Gene Gene Therapy Hematology Medicine Research Transplant

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