A multi-center clinical trial of allogeneic hematopoietic stem cell transplantation in transfusion-dependent thalassemia - PubMed
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- #thalassemia
- #hematopoietic stem cell transplantation
- #clinical trial
- A multi-center clinical trial evaluated allogeneic hematopoietic stem cell transplantation (allo-HSCT) for transfusion-dependent thalassemia (TDT).
- The study included 823 patients, using grafts from HLA-matched sibling donors (MSDs), matched unrelated donors (MUDs), and haploidentical related donors (Haplos).
- Conditioning regimen involved busulfan, cyclophosphamide, fludarabine, and anti-thymocyte globulin.
- Graft-versus-host disease (GvHD) prophylaxis varied based on donor type.
- Primary endpoints were 2-year overall survival (OS) and event-free survival (EFS).
- 2-year OS for MSDs, MUDs, and Haplos was 97.2%, 93.1%, and 95.4% respectively.
- EFS for MSDs, MUDs, and Haplos was 97.2%, 92.9%, and 94.7% respectively.
- GvHD-free, relapse-free survival (GRFS) was higher for MSDs (91.4%) compared to MUDs (77.0%) and Haplos (75.6%).
- Incidence of acute and chronic GvHD was higher with alternative donors than MSDs.
- Transplant-related mortality was 4.4%, and graft failure rate was 0.5%.
- The findings suggest expanding the donor pool for TDT patients lacking MSDs.