ArticleTherapeutic advances in hematology2026
Comparing two different doses of anti-thymocyte globulin in allogeneic hematopoietic stem cell transplantation for transfusion-dependent thalassemia: an open-label, randomized clinical trial.
Article in Therapeutic advances in hematology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
Background: Anti-thymocyte globulin (ATG) is used as prophylaxis for graft-versus-host disease (GvHD) and graft failure (GF) in allogeneic hematopoietic stem cell transplantation (allo-HSCT) for patients with transfusion-dependent thalassemia (TDT). However, the optimal dose of ATG remains unknown. Objectives: To compare the 3-year overall survival (OS) of 8 mg/kg and 10 mg/kg ATG in allo-HSCT for patients with TDT. Methods: We compared two different doses of ATG for patients with TDT undergoing allo-HSCT from HLA-matched sibling donors (MSDs). Between 2015 and 2023, 147 patients were randomized to the 8 mg/kg ATG group and 148 patients to the 10 mg/kg ATG group. Primary endpoints were 3-year OS and thalassemia-free survival (TFS). Survival analysis for OS and TFS was performed using the Kaplan-Meier method. Results: The 8 and 10 mg/kg ATG groups had similar 3-year OS and TFS (both 98.6% (95% confidence interval [CI]: 96.8-100) vs 95.3% (95% CI: 91.9-98.7), Conclusion: This study provides evidence that an 8 mg/kg ATG is an effective treatment for TDT transplanted from MSDs. A notable finding was the superior survival associated with the 8 mg/kg ATG in the CB and BM co-transplantation. These results provide critical evidence for guiding ATG dosing strategies in allo-HSCT for TDT, particularly in the context of stem cell source selection. Design: Open-label, randomized clinical trial. Trial registration: ChiCTR-IPR-15005779 (Chinese Clinical Trial Registry; https://www.chictr.org.cn/showproj.html?proj=10208; date of registration: December 29, 2014).
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