Fludarabine, Busulfan, Thiotepa, and Melphalan: A Novel Low-Toxicity Myeloablative Conditioning Regimen for Haploidentical HSCT in Children and Adolescents Aged ≥7 Years With Transfusion-Dependent β-Thalassemia.
Pediatr Blood CancerJul 23, 2026 (epub)
Guiping Liao, Dongmei Liu, Beibei Yang et al.
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In 30 seconds
This prospective clinical trial evaluated a novel low-toxicity myeloablative conditioning regimen (F-BMT) in 29 children and adolescents aged ≥7 years with transfusion-dependent β-thalassemia undergoing haploidentical donor hematopoietic stem cell transplantation. The study found a 2-year thalassemia-free survival rate of 93.1%, indicating promising outcomes with manageable toxicity.
Key findings
- The 2-year thalassemia-free survival rate was 93.1%.
- Overall survival rate was also 93.1%.
- Neutrophil and platelet engraftment were achieved in all patients.
- Grade II-IV acute GVHD incidence was 17.2%.
Why it matters
The study highlights a promising conditioning regimen that may enhance transplantation outcomes for children and adolescents with transfusion-dependent β-thalassemia, addressing significant barriers such as graft failure and transplant-related mortality.