ArticleAnnals of hematology2026
Transforming the treatment of Alpha-Thalassemia: a single-center retrospective study on hematopoietic stem cell transplantation in transfusion-dependent pediatric patients.
Article in Annals of hematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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Who cites it
1 citing paper in PubMed.
- Reactivation of the embryonic ζ-globin gene ameliorates severe forms of α-thalassemia.Nature genetics · 2026Article
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24 authors.
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Abstract
Hematopoietic stem cell transplantation (HSCT) is the only definitive cure for transfusion-dependent α-thalassemia, though comprehensive studies on its effectiveness are limited. In this retrospective study, we analyzed the clinical characteristics of 21 pediatric patients with transfusion-dependent α-thalassemia who underwent HSCT, all of whom received a standardized conditioning regimen consisting of busulfan, cyclophosphamide, fludarabine, and anti-thymocyte globulin. After a median follow-up of 25 months (range: 7–92 months), the two-year overall survival (OS) and event-free survival (EFS) rates were both 90.2% (95% CI: 66.2–97.4%), and the two-year graft-versus-host disease-free, relapse-free survival (GRFS) rate was 82.3% (95% CI: 52.6–94.3%). The transplant-related mortality rate at two years was 5.0% (95% CI: 0.7–30.0%), with no cases of graft failure observed. Among the 19 surviving patients, hemoglobin levels significantly increased compared to pre-transplant levels (p < 0.05), and all became transfusion-independent. Hematopoietic stem cell transplantation is a curative treatment for α-thalassemia. For patients with transfusion-dependent α-thalassemia, HSCT should be performed as early as possible at an experienced transplant center when a suitable donor is available.
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