Hematology · Pediatrics · 2 h ago
Simplified Matched-Sibling Transplant Protocol Shows Promising Results in Children With Thalassemia
A multicenter cohort study evaluated a simplified, low-cost bone marrow transplant protocol in 176 children with transfusion-dependent thalassemia in Asia and the Middle East. At a median follow-up of 11.3 months, thalassemia-free survival was 93.4% and overall survival was 96%.
- 176 children received matched-sibling bone marrow transplants.
- Thalassemia-free survival was 93.4% at median 11.3 months.
- Moderate-to-severe acute GVHD occurred in 3.9%.
- Short follow-up and no comparator limit interpretation.
Matched-sibling bone marrow transplantation can offer a curative treatment for transfusion-dependent thalassemia, but cost and complexity limit access in many low- and middle-income countries. This multicenter study included 176 children treated between February 2022 and November 2025 at centers in Asia and the Middle East. The simplified protocol used fludarabine, dexamethasone, busulfan, and cyclophosphamide, with granulocyte colony-stimulating factor–primed bone marrow and cyclosporine plus methotrexate for graft-versus-host disease (GVHD) prophylaxis.
Engraftment was achieved in 97.3% of patients. At a median follow-up of 11.3 months, thalassemia-free survival was 93.4% and overall survival was 96%. Moderate-to-severe acute GVHD occurred in 3.9%; the reported actuarial composite outcome of GVHD-free and thalassemia-free survival was 93.4%. Cytomegalovirus reactivation occurred in 19.2%, with one patient progressing to CMV disease. Outcomes were reported as similar at experienced and newly established transplant centers.
The findings suggest that a protocol using widely available medicines may be feasible in resource-limited settings. Follow-up was relatively short, and the abstract provides no comparator or detailed center-level results; the reported outcomes therefore do not establish comparative effectiveness or long-term safety.
In practice
For children with transfusion-dependent thalassemia who have a matched sibling donor, these results suggest that bone marrow transplantation using this simplified regimen can achieve high short-term engraftment and survival in centers across resource-limited regions. The cohort reported 93.4% thalassemia-free survival at a median follow-up of 11.3 months. This is encouraging feasibility evidence, not proof that the protocol is equivalent or superior to other regimens: the abstract reports no comparator, and longer-term outcomes and detailed center-level data are not provided. It does not establish a change in transplant practice.
AI-generated clinical interpretation · Not editor-reviewed
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Blood Advances: Optimizing outcomes and accessibility of matched sibling donor transplant for transfusion-dependent thalassemia in LMICs ↗This is an automated AI-condensed summary that has not yet been reviewed by an editor. Always consult the full item at the original source.