Zhang Ruo-Xi, Huang Yu-Zhou, Han Bing
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100730, China.
Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Beijing 100730, China.Department of Gastroenterology, Sun Yat-sen Memorial Hospital, Sun Yat-sen University, Guangzhou 510120, Guangdong Province, China.
Zhongguo Shi Yan Xue Ye Xue Za Zhi. 2023 Aug;31(4):1138-1142. doi: 10.19746/j.cnki.issn.1009-2137.2023.04.032.
To evaluate the efficacy, safety and relapse of cyclosporine A (CsA) and CsA combined with corticosteroid (CS) as the frontline therapy for patients with newly diagnosed acquired pure red cell aplasia (aPRCA).
The clinical features, treatment responses, relapses and clinical outcomes of patients with newly diagnosed aPRCA in Peking Union Medical College Hospital (PUMCH) from January 2015 to May 2020 were analyzed retrospectively. All the enrolled patients had been treated with either CsA or CsA+CS for at least 6 months and had been followed up for at least 12 months, with complete clinical data and consent forms.
96 patients including 72 treated with CsA and 24 treated with CsA+CS were enrolled. With comparable baseline characteristics and follow-up periods, patients treated with CsA or with CsA+CS had similar overall response rates (ORRs) and complete response rates (CRRs) at the 3rd, 6th and 12th month and at the end of follow-up (>0.05). Meanwhile, no significant difference was found between the two groups in the optimal ORR, optimal CRR, time to response or time to complete response. CsA+CS and CsA groups had similar adverse event (AE) rates, but CsA+CS group had higher CS-related infection rate ( <0.05). One patient in CsA+CS group died of multiple infections. As for the relapse, the two groups had compatible relapse rates at different time points, time to relapse, overall relapse rate and relapse-free survival (>0.05). CsA exposure time, rather than different therapy regimens, was the only influence factor for either ORR or relapse rate ( <0.05).
CsA monotherapy has similar efficacy, AE rate and relapse rate as compared with CsA+CS for patients with newly diagnosed aPRCA, and shows less CS-related AEs such as infection.
评估环孢素A（CsA）及CsA联合皮质类固醇（CS）作为新诊断获得性纯红细胞再生障碍性贫血（aPRCA）患者一线治疗方案的疗效、安全性及复发情况。
回顾性分析2015年1月至2020年5月在北京协和医院（PUMCH）新诊断的aPRCA患者的临床特征、治疗反应、复发情况及临床结局。所有纳入患者均接受CsA或CsA + CS治疗至少6个月，并随访至少12个月，具备完整临床资料及知情同意书。
共纳入96例患者，其中72例接受CsA治疗，24例接受CsA + CS治疗。在基线特征和随访时间可比的情况下，接受CsA或CsA + CS治疗的患者在第3、6、12个月及随访结束时的总缓解率（ORR）和完全缓解率（CRR）相似（>0.05）。同时，两组在最佳ORR、最佳CRR、缓解时间或完全缓解时间方面无显著差异。CsA + CS组和CsA组的不良事件（AE）发生率相似，但CsA + CS组的CS相关感染率更高（<0.05）。CsA + CS组有1例患者死于多重感染。至于复发情况，两组在不同时间点的复发率、复发时间、总复发率及无复发生存率均相近（>0.05）。CsA暴露时间而非不同治疗方案是影响ORR或复发率的唯一因素（<0.05）。
对于新诊断的aPRCA患者，CsA单药治疗与CsA + CS治疗的疗效、AE发生率及复发率相似，且CS相关不良事件（如感染）较少。
