Department of Hematology, The First Affiliated Hospital of Zhejiang Chinese Medical University (Zhejiang Provincial Hospital of Chinese Medicine), Hangzhou, Zhejiang, China.
The First School of Clinical Medicine, Zhejiang Chinese Medical University, Hangzhou, Zhejiang, China.
Ann Med. 2023;55(2):2271475. doi: 10.1080/07853890.2023.2271475. Epub 2023 Oct 23.
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 ( = 27) or allo-HSCT ( = 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, < 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, = 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%, = 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 ( = 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.
在输血依赖性非重型再生障碍性贫血（TD-NSAA）患者中，选择和确定抗胸腺细胞球蛋白（ATG）为基础的免疫抑制治疗（IST）或异基因造血干细胞移植（allo-HSCT）的时机具有重要的临床挑战。本研究旨在比较这两种治疗方法在 TD-NSAA 中的疗效和长期结果。
回顾了 2011 年 7 月至 2019 年 12 月期间接受 ATG 为基础的 IST 或 allo-HSCT 的患者。我们收集了他们的临床信息、治疗反应、生存数据，并随后分析了相关的风险因素。
共回顾了 97 例 TD-NSAA 患者，其中 55 例患者接受了 ATG 为基础的 IST（n=27）或 allo-HSCT（n=28）。我们观察到 12 个月时总体缓解率（ORR）有显著差异（IST 组为 48.1%，HSCT 组为 78.6%，<0.05），但 5 年总生存率（OS）和无事件生存率（EFS）无差异。多变量 Cox 回归分析发现，输注≥78.75 单位的红细胞（RBCs）是 IST 组 OS 的唯一独立危险因素（HR：17.04，=0.039）。对于 HSCT 组，疾病持续时间（DD）≥20 个月和输注≥78.75 单位的 RBCs 预示着 EFS 不良。一线 IST 的 12 个月 ORR（68.8% vs 18.2%，=0.018）和 5 年 EFS 优于非一线 IST。DD 为 6 至 20 个月的患者在 HSCT 组的 EFS 优于 ATG 为基础 IST 组（=0.016）。
在 TD-NSAA 患者中，选择 ATG 为基础的 IST 与 allo-HSCT 时，应仔细权衡既往治疗史、疾病持续时间和血清铁蛋白水平。
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