Chen Wan-Shu, Zhang Meng-Lu, Han Bing
Department of Hematology,PUMC Hospital,CAMS and PUMC,Beijing 100730,China.
Zhongguo Yi Xue Ke Xue Yuan Xue Bao. 2021 Oct;43(5):736-742. doi: 10.3881/j.issn.1000-503X.13201.
Objective To compare the efficacy and safety of cyclosporin A(CsA)and CsA combined with recombined human erythropoietin(rhEPO)in the treatment of patients with chronic aplastic anemia(CAA).Methods Data of 79 patients with CAA treated at Department of Hematology,PUMC Hospital between January 2016 and June 2018 were collected for retrospective analysis.Forty-five patients were treated with CsA+rhEPO,and the other 34 patients with CsA alone.All the enrolled patients were treated for at least 1.5-2.0 years and followed for at least 1.0 year.The efficacy,side effects,long-term outcomes were compared between the two groups,and factors that may influence the efficacy were analyzed.Results The patients treated with CsA+rhEPO included 14 males and 31 females,with a median age of 43(19,73)years old.The median treatment duration of CsA and rhEPO was 26(12,38)and 4(3,6)months,respectively,and the median followed-up time was 24(12,42)months.The patients treated with CsA alone included 16 males and 18 females,with a median age of 36(16,85)years old.The median CsA treatment duration was 24(12,40)months and the median follow-up time was 25(12,40)months.There was no statistical difference in baseline characteristics between the two groups(all >0.05).After 6 months of treatment,CsA+rhEPO group had higher overall response(OR)rate(55.6% 31.3%; =4.456,=0.040)and partial response(PR)rate(53.3% 25.0%;=6.181,=0.019)than CsA group,and the complete response(CR)rate showed no statistical difference between the two groups(2.2% 6.3%;=0.810,=0.567).The CR,PR,and OR rates showed no statistical difference between the two groups after 3 and 12 months of treatment and at the end of follow-up(all >0.05).Similarly,the hemoglobin level at the sixth month of treatment with CsA+rhEPO was higher than that with CsA alone [(102.6±24.0)g/L (90.3±29.1)g/L;=2.017,=0.047].However,it showed no significant difference between the two groups at other time points(all >0.05).The side effects,including an increase in serum creatinine,slight increase in bilirubin,increase in aminotransferase,mild to moderate gingival hyperplasia,gastrointestinal reaction,and muscular fibrillation,had no significant differences between the two groups(all >0.05),except that 11.1%(5/45)patients in the CsA+rhEPO group reported soreness at the injection site.The two groups showed similar rates of clonal revolution during the follow-up period and no death.No clinical factor was found for prediction of the efficacy of CsA+rhEPO. Conclusion CsA+rhEPO has higher OR rate and hemoglobin level than CsA alone at the sixth month of treatment,and it has similar side effects compared with CsA alone.
目的 比较环孢素A（CsA）及CsA联合重组人促红细胞生成素（rhEPO）治疗慢性再生障碍性贫血（CAA）患者的疗效及安全性。方法 收集2016年1月至2018年6月在中国医学科学院北京协和医院血液科治疗的79例CAA患者资料进行回顾性分析。45例患者采用CsA+rhEPO治疗，另外34例患者仅采用CsA治疗。所有入组患者均接受至少1.5 - 2.0年治疗，并随访至少1.0年。比较两组患者的疗效、副作用、长期预后，并分析可能影响疗效的因素。结果 CsA+rhEPO组患者中男性14例，女性31例，中位年龄43（19，73）岁。CsA及rhEPO的中位治疗时间分别为26（12，38）个月和4（3，6）个月，中位随访时间为24（12，42）个月。仅采用CsA治疗的患者中男性１6例，女性18例，中位年龄36（16，85）岁。CsA的中位治疗时间为24（12，40）个月，中位随访时间为25（12，40）个月。两组患者的基线特征无统计学差异（均>0.05）。治疗6个月后，CsA+rhEPO组的总缓解（OR）率（55.6%对31.3%；Z = 4.456，P = 0.040）和部分缓解（PR）率（53.3%对25.0%；Z = 6.181，P = 0.019）高于CsA组，两组的完全缓解（CR）率无统计学差异（2.2%对6.3%；Z = 0.810，P = 0.５67）。治疗3个月、12个月及随访结束时，两组的CR、PR及OR率均无统计学差异（均>0.05）。同样，CsA+rhEPO治疗第6个月时的血红蛋白水平高于仅用CsA治疗者[（102.6±24.0）g/L对（90.3±29.1）g/L；Z = 2.017，P = 0.047]。但在其他时间点两组间无显著差异（均>0.05）。两组的副作用，包括血清肌酐升高、胆红素轻度升高、转氨酶升高、轻至中度牙龈增生、胃肠道反应及肌肉震颤，均无显著差异（均>0.05），除了CsA+rhEPO组有11.1%（5/45）的患者报告注射部位疼痛。两组在随访期间的克隆演变率相似，均无死亡。未发现可预测CsA+rhEPO疗效的临床因素。结论 治疗第6个月时，CsA+rhEPO的OR率及血红蛋白水平高于单用CsA，且与单用CsA的副作用相似。
