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

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. 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. 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. 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. The selection and timing of anti-thymocyte globulin (ATG)-based immunosuppressive therapy (IST) or allogeneic hematopoietic stem cell transplantation (allo-HSCT) present notable clinical challenges for individuals with transfusion-dependent non-severe aplastic anaemia (TD-NSAA).In terms of treatment outcomes, allo-HSCT exhibited a higher 12-month overall response rate (ORR) in comparison to ATG-based IST among TD-NSAA patients. Nevertheless, comparable rates of 5-year overall survival (OS) and event-free survival (EFS) were observed between the two therapeutic approaches.Several factors warrant consideration when deliberating between ATG-based IST and allo-HSCT for TD-NSAA. These factors include the patient’s prior treatment history, disease duration, number of packed red cell transfusions received, and serum ferritin levels. The selection and timing of anti-thymocyte globulin (ATG)-based immunosuppressive therapy (IST) or allogeneic hematopoietic stem cell transplantation (allo-HSCT) present notable clinical challenges for individuals with transfusion-dependent non-severe aplastic anaemia (TD-NSAA). In terms of treatment outcomes, allo-HSCT exhibited a higher 12-month overall response rate (ORR) in comparison to ATG-based IST among TD-NSAA patients. Nevertheless, comparable rates of 5-year overall survival (OS) and event-free survival (EFS) were observed between the two therapeutic approaches. Several factors warrant consideration when deliberating between ATG-based IST and allo-HSCT for TD-NSAA. These factors include the patient’s prior treatment history, disease duration, number of packed red cell transfusions received, and serum ferritin levels.

针对输血依赖性非重型再生障碍性贫血（transfusion-dependent non-severe aplastic anemia, TD-NSAA）患者，抗胸腺细胞球蛋白（anti-thymocyte globulin, ATG）方案免疫抑制治疗（immunosuppressive therapy, IST）与异基因造血干细胞移植（allogeneic hematopoietic stem cell transplantation, allo-HSCT）的选择及时机把控，均为临床面临的重大挑战。本研究旨在对比两种治疗方案在TD-NSAA患者中的疗效与长期预后。本研究回顾性分析了2011年7月至2019年12月期间接受ATG方案IST或allo-HSCT的患者，收集其临床资料、治疗应答情况及生存数据，并对相关危险因素进行分析。共计纳入97例TD-NSAA患者，最终筛选出接受ATG方案IST（n=27）或allo-HSCT（n=28）的55例患者入组。研究观察到，两组患者12个月总体应答率（overall response rate, ORR）存在显著差异（IST组48.1%，HSCT组78.6%，p<0.05），但5年总体生存率（overall survival, OS）与无事件生存率（event-free survival, EFS）无统计学差异。多因素Cox回归分析显示，在IST组中，输注红细胞（red blood cells, RBCs）≥78.75单位是OS的唯一独立危险因素（风险比HR=17.04，p=0.039）；在HSCT组中，病程≥20个月与输注RBCs≥78.75单位均为不良EFS的预测因素。与非一线治疗相比，一线IST治疗的12个月ORR更高（68.8% vs 18.2%，p=0.018），且5年EFS更优。在HSCT组中，病程为6~20个月的患者EFS显著优于ATG方案IST组（p=0.016）。临床医师在为TD-NSAA患者选择ATG方案IST或allo-HSCT及确定治疗时机时，需综合权衡患者既往治疗史、病程、红细胞输注量及血清铁蛋白水平等因素。针对输血依赖性非重型再生障碍性贫血（transfusion-dependent non-severe aplastic anaemia, TD-NSAA）患者，抗胸腺细胞球蛋白（anti-thymocyte globulin, ATG）方案免疫抑制治疗（immunosuppressive therapy, IST）与异基因造血干细胞移植（allogeneic hematopoietic stem cell transplantation, allo-HSCT）的选择及时机把控，仍为临床亟待解决的棘手问题。就治疗结局而言，在TD-NSAA患者中，allo-HSCT的12个月总体应答率显著高于ATG方案IST。然而，两种治疗方案的5年总体生存率与无事件生存率并无显著差异。临床医师在为TD-NSAA患者抉择ATG方案IST与allo-HSCT时，需综合考量多项因素，包括患者既往治疗史、病程、输注悬浮红细胞的总量及血清铁蛋白水平。针对输血依赖性非重型再生障碍性贫血（transfusion-dependent non-severe aplastic anaemia, TD-NSAA）患者，抗胸腺细胞球蛋白（anti-thymocyte globulin, ATG）方案免疫抑制治疗（immunosuppressive therapy, IST）与异基因造血干细胞移植（allogeneic hematopoietic stem cell transplantation, allo-HSCT）的选择及时机把控，仍为临床亟待解决的棘手问题。就治疗结局而言，在TD-NSAA患者中，allo-HSCT的12个月总体应答率显著高于ATG方案IST。然而，两种治疗方案的5年总体生存率与无事件生存率并无显著差异。临床医师在为TD-NSAA患者抉择ATG方案IST与allo-HSCT时，需综合考量多项因素，包括患者既往治疗史、病程、输注悬浮红细胞的总量及血清铁蛋白水平。