New Benchmark for Reduced-Intensity Sickle Cell BMT Set
Johns Hopkins University has introduced a new reduced-intensity conditioning regimen for bone marrow transplants in patients with severe sickle cell disease. This approach shows promising outcomes, including high disease-free survival rates and preservation of fertility in some patients. The regimen is seen as a significant advancement in providing curative therapy for those who cannot tolerate traditional methods.
- ▪The new regimen allows bone marrow transplants for patients who cannot tolerate traditional myeloablative conditioning.
- ▪The median age of patients in the study was 23 years, with a disease-free survival probability at 2 years of 94.5%.
- ▪Among female patients, 44% resumed menses and some had successful pregnancies after the transplant.
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Johns Hopkins University investigators have reported a novel, reduced-intensity conditioning regimen for severe sickle cell disease (SCD) that allows bone marrow transplants in patients who cannot tolerate traditional myeloablative conditioning.The regimen also appears to preserve fertility in some women and to improve upon an alternative reduced-intensity conditioning regimen first published in 2024 by limiting graft failure in children and shortening the duration of posttransplant immunosuppression.The Hopkins approach combines antithymocyte globulin, fludarabine, cyclophosphamide, and single-fraction 400 cGy total body irradiation.
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