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<p>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 (<i>p</i> &lt; 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.</p>

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Transforming the treatment of Alpha-Thalassemia: a single-center retrospective study on hematopoietic stem cell transplantation in transfusion-dependent pediatric patients

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年3月4日

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  • Lihua Huang,
  • Zhenbin Wei,
  • Gaohui Yang,
  • Lianjin Liu,
  • Zhongming Zhang,
  • Hongwen Xiao,
  • Zeyan Shi,
  • Yibin Yao,
  • Meiqing Wu,
  • Lingyuan Pan,
  • Wenqiang Xie,
  • Zhaoping Gan,
  • Beicai Liu,
  • Zhongqing Li,
  • Rui Huang,
  • Xuemei Zhou,
  • Yinghua Chen,
  • Yanye Liu,
  • Juan Bin,
  • Zhaofang Pan,
  • Huicheng Huang,
  • Yongrong Lai,
  • Rongrong Liu,
  • Lingling Shi

摘要

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.