Annals of Medicine (Dec 2023)

Comparison of anti-thymocyte globulin-based immunosuppressive therapy and allogeneic hematopoietic stem cell transplantation in patients with transfusion-dependent non-severe aplastic anaemia: a retrospective study from a single centre

  • Yingying Shen,
  • Yuzhu Li,
  • Qi Liu,
  • Wenbin Liu,
  • Qinghong Yu,
  • Huijin Hu,
  • Shan Liu,
  • Jingjie Dong,
  • Min Xu,
  • Yaonan Hong,
  • Ying Chen,
  • Shu Deng,
  • Haifeng Zhuang,
  • Zhiping Hu,
  • Shenyun Lin,
  • Yiping Shen,
  • Jianping Shen,
  • Yuhong Zhou,
  • Baodong Ye,
  • Dijiong Wu

DOI
https://doi.org/10.1080/07853890.2023.2271475
Journal volume & issue
Vol. 55, no. 2

Abstract

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AbstractObjectives The selection and timing of anti-thymocyte globulin (ATG)-based immunosuppressive therapy (IST) or allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with transfusion-dependent non-severe aplastic anemia (TD-NSAA) pose significant clinical challenges. This study aims to compare the efficacy and long-term outcomes of the two treatments in TD-NSAA.Methods Patients who underwent ATG-based IST or allo-HSCT between July 2011 and December 2019 were reviewed. We gathered their clinical information, treatment response, survival data, and subsequently analysed the associated risk factors.Results A total of 97 TD-NSAA patients were reviewed, and 55 patients who underwent either ATG-based IST (n = 27) or allo-HSCT (n = 28) were enrolled. We observed a significant disparity in the 12-month overall response rate (ORR) (48.1% in IST vs 78.6% in HSCT, p < 0.05), but not in five-year overall survival (OS) and event-free survival (EFS). Multivariate Cox regression analysis identified the transfusion of ≥78.75 units of red blood cells (RBCs) as the sole independent risk factor for OS (HR: 17.04, p = 0.039) in the IST group. For the HSCT group, disease duration (DD) ≥20 months and transfusion of ≥78.75 units of RBCs predicted an adverse EFS. Frontline IST exhibited superior 12-month ORR (68.8% vs 18.2%, p = 0.018) and five-year EFS when compared to non-frontline. Patients with a DD ranging from 6 to 20 months displayed a better EFS (p = 0.016) in HSCT group than those in the ATG-based IST group.Conclusions Prior treatment history, disease duration, and serum ferritin levels should be carefully weighed when making the choice between ATG-based IST and allo-HSCT for TD-NSAA.

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