环孢素A糖皮质激素治疗成人获得性纯红细胞再生障碍疗效对照研究

Comparison of cyclosporine A with corticosteroids in the treatment of adult patients with acquired pure red cell aplasia

目的评估环孢素A(CsA)、糖皮质激素(CS)治疗成人获得性纯红细胞再生障碍(PRCA)疗效。方法回顾性分析2009年10月至2017年4月南京医科大学第一附属医院收治的42例PRCA接受CsA(16例)、CS(13例)、CsA联合CS(6例)及其他免疫抑制剂(4例)治疗临床资料。结果接受CsA、CS、CsA联合CS及其他免疫抑制剂初始有效率分别为75.0%,46.2%,66.7%,75.0%(P=0.456),累积有效率分别为69.6%(16/23),50.0%(7/14),71.4%(5/7),42.9%(6/14)(P=0.376),累积完全缓解(CR)率分别为26.1%(6/23),28.6%(4/14),57.1%(4/7),14.3%(2/14)(P=0.284)。27例难治复发PRCA,17例改为CsA或CS方案中11例(64.7%)有效,10例改为其他免疫抑制剂3例有效(30%)(P=0.120)。结论 CsA和CS治疗PRCA疗效相似。难治复发PRCA患者,CsA和(或)CS无效后尚无满意的治疗方法。

Objective To assess the effect of cyclosporine A （CsA） and corticosteroids （CS） in the treatment of adult patients with acquired pure red cell aplasia （PRCA）. Methods Clinical data of 42 cases receiving cyclosporine A （CsA）, and/or corticosteroids （CS）, or other immunosuppressive agents were retrospectively analyzed, including CsA （n=16）, CS （n=13）, CsA in combination with CS （n=6）, or other immunosuppressive agents （n=4）. Results The initial response rates were similar among these patients （75.0%, 46.2%, 66.7%, 75.0%, P=0.456）. q-he cumulative response rates in patients receiving CsA, CS, CsA in combination with CS, or other immunosuppressive agents were 69.6% （16/23）, 50.0% （7/14）, 71.4% （5/7）, 42.9% （6/14）, respectively （P=0.376）; the cumulative rates of CRwere 26.1% （6/23）, 28.6% （4/14）, 57.1% （4/7）, 14.3% （2/14）, respectively （P=0.284）. In 27 refractory and relapsed PRCA patients, 11 out of 17 patients （64.7%） achieved remission by CsA and/or CS regimen, while 3 of 10 patients （30.0%） responded to other agents （P=0.120）. Conclusion CsA and/or CS are similarly effective in treating PRCA. For patients with relapse or refractory PRCA, there are no satisfactory treatments ifCsA and/or CS fail.