Yusup Muyassar, He GuangSheng, Qin YuTing, Tuerxun Niluopaer, Hao JianPing
Department of Hematology, First Affiliated Hospital of Xinjiang Medical University, XinJiang Institute of Hematology , Urumqi, China.
Jiangsu Province Hospital, Nanjing, Jiangsu, China.
Ann Hematol. 2025 Apr;104(4):2189-2206. doi: 10.1007/s00277-025-06315-z. Epub 2025 Mar 19.
Acquired pure red cell aplasia (aPRCA) is a rare hematological syndrome characterized by anemia and a significant reduction in erythroid progenitor cells. Immunosuppressive therapy (IST), including Corticosteroids (CS), Cyclosporine (CsA), and cyclophosphamide (CYC), is the primary treatment. However, variations in clinical efficacy and limited comparative studies have created uncertainty in therapeutic choices. This study aims to evaluate the efficacy of IST and the factors influencing treatment outcomes. A systematic search was conducted using PubMed, Embase, Cochrane Library, and Web of Science. Two researchers independently screened studies and extracted data. The quality of studies was assessed using the MINORS scale. Meta-analysis was performed using STATA/MP16, and effect size (ES) was calculated using fixed- or random-effects models based on heterogeneity. A total of 33 studies involving 1,193 patients were included. The overall efficacy of IST was significant, with a pooled ES of 0.656 (95% CI: 0.600-0.710). CsA demonstrated the highest efficacy (ES = 0.699; 95% CI: 0.615-0.779), followed by CYC (ES = 0.592; 95% CI: 0.423-0.752) and CS (ES = 0.568; 95% CI: 0.457-0.676). Subgroup analyses revealed that factors such as etiology, combination therapies, first- vs. second-line treatment, and genetic characteristics significantly influenced outcomes. Notably, the response to IST was higher in primary aPRCA (ES = 0.667; 95% CI: 0.598-0.733) compared to LGLL-associated (ES = 0.515; 95% CI: 0.393-0.637) and thymoma-associated (ES = 0.690; 95% CI: 0.492-0.864) aPRCA. The combination of CS and CsA yielded superior efficacy (ES = 0.761; 95% CI: 0.658-0.853) compared to combination of CS and CsA and monotherapy. First-line treatment demonstrated better efficacy than second-line treatment (ES = 0.659; 95% CI: 0.596-0.720) vs. (ES = 0.452; 95% CI: 0.199-0.715). The important finding was that (ES = 0.861; 95% CI: 0.595-1.000) in the STAT3 mutation (+) group and (ES = 0.375; 95% CI: 0.034-0.801) in the STAT3 mutation (-) group. IST demonstrates overall efficacy in aPRCA, with variations influenced by etiology, drug combinations, and genetic mutations such as STAT3. These findings highlight the need for personalized treatment strategies and further research to validate and optimize IST efficacy.
获得性纯红细胞再生障碍性贫血（aPRCA）是一种罕见的血液学综合征，其特征为贫血和红系祖细胞显著减少。免疫抑制治疗（IST），包括皮质类固醇（CS）、环孢素（CsA）和环磷酰胺（CYC），是主要治疗方法。然而，临床疗效的差异和有限的比较研究使得治疗选择存在不确定性。本研究旨在评估IST的疗效以及影响治疗结果的因素。使用PubMed、Embase、Cochrane图书馆和Web of Science进行了系统检索。两名研究人员独立筛选研究并提取数据。使用MINORS量表评估研究质量。使用STATA/MP16进行荟萃分析，并根据异质性使用固定效应或随机效应模型计算效应量（ES）。共纳入33项研究，涉及1193例患者。IST的总体疗效显著，合并ES为0.656（95%CI：0.600 - 0.710）。CsA显示出最高疗效（ES = 0.699；95%CI：0.615 - 0.779），其次是CYC（ES = 0.592；95%CI：0.423 - 0.752）和CS（ES = 0.568；95%CI：0.457 - 0.676）。亚组分析显示，病因、联合治疗、一线与二线治疗以及基因特征等因素显著影响治疗结果。值得注意的是，与大颗粒淋巴细胞白血病（LGLL）相关的aPRCA（ES = 0.515；95%CI：0.393 - 0.637）和胸腺瘤相关的aPRCA（ES = 0.690；95%CI：0.492 - 0.864）相比，原发性aPRCA对IST的反应更高（ES = 0.667；95%CI：0.598 - 0.733）。与CS和CYC联合及单一疗法相比，CS和CsA联合产生了更高的疗效（ES = 0.761；95%CI：0.658 - 0.853）。一线治疗显示出比二线治疗更好的疗效（ES = 0.659；95%CI：0.596 - 0.720）与（ES = 0.452；95%CI：0.199 - 0.715）。重要发现是，信号转导和转录激活因子3（STAT3）突变（+）组的ES为0.861（95%CI：0.595 - 1.000），而STAT3突变（-）组的ES为0.375（95%CI：0.034 - 0.801）。IST在aPRCA中显示出总体疗效，其疗效差异受病因、药物组合和STAT3等基因突变的影响。这些发现凸显了个性化治疗策略的必要性以及进一步研究以验证和优化IST疗效的重要性。
