At a glance
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Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
In Brief
A Phase 4 clinical trial evaluating Busulfan, Cyclophosphamide, and 6 other interventions for Thalassemia Major. Completed, enrolled 823 participants across 1 site.
Detailed Summary
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
Study Details
Timeline
Interventions
Busulfan（4 mg/kg/day,4 days）
Cyclophosphamide（50 mg/kg/day,4 days）
Fludarabine（50 mg/m2/day,3 days）
Thymoglobulin（2.5 mg/kg/day,4 days）
cyclosporine A
Mycophenolate mofetil（0.25g/day）
Tacrolimus
Methotrexate