Objective: 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).
Methods: 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.
Results: 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 (P>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 (P <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 (P>0.05). CsA exposure time, rather than different therapy regimens, was the only influence factor for either ORR or relapse rate (P <0.05).
Conclusion: 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.
题目: 环孢素单药和环孢素联合糖皮质激素治疗获得性纯红细胞再生障碍的比较研究.
目的: 评估环孢素（CsA）和环孢素联合糖皮质激素（CS）治疗初治获得性纯红细胞再生障碍（aPRCA）的疗效、安全性及复发情况。.
方法: 回顾性分析2015年1月至2020年5月就诊于北京协和医院的初治aPRCA患者的临床资料。所有入选患者经CsA单药或CsA联合CS治疗至少6个月，随访至少12个月，有完整临床资料并签署知情同意书。比较两组患者的临床特征、疗效、复发和治疗转归情况。.
结果: 共纳入96例患者，其中CsA组72例，CsA+CS组24例。CsA组和CsA+CS组患者基线特征匹配。在相似的随访期内，两组3个月、6个月、12个月和随访期末的总反应率（ORR）和完全反应率（CRR）均无显著差异（P>0.05）；最佳ORR、最佳CRR、起效时间和达到完全反应时间均无显著差异（P>0.05）。两组不良反应率相似，但CsA+CS组的CS相关感染率显著增加（P <0.05），其中1例患者死于多重感染。CsA组和CsA+CS组在不同时间点的复发率、总复发率、复发时间以及无复发生存期（RFS）无显著差异（P>0.05）。患者治疗反应及复发率与CsA给药时间显著相关（P <0.05），而与治疗方案无关。.
结论: 对于初治aPRCA患者，CsA单药与CsA+CS的疗效和复发率相似，但可避免CS相关不良反应（如感染）的发生。.
Keywords: acquired pure red cell aplasia; corticosteroid; cyclosporine A; efficacy; relapse rate.