摘要目的:探讨低剂量芦可替尼治疗异基因造血干细胞移植术后出现慢性移植物抗宿主病（cGVHD），经一线和二线药物治疗无效患者的疗效和安全性。方法:回顾性分析2018年7月9日至2019年5月23日期间就诊于安徽省立医院，经一线和二线药物治疗无效的cGVHD患者的临床资料。患者均加用低剂量芦可替尼治疗（剂量：患者体质量≥25 kg，5 mg/次，2次/d；体质量<25 kg，2.5 mg/次, 2次/d）。每周或每两周评估治疗疗效和药物安全性，若病情好转则每周或每两周减量2.5 mg直至停药。结果:共47例患者纳入研究。芦可替尼中位服用时间55（21~154）d，中位起效时间14（7~28）d，总体反应率为87.2%（41/47例），完全反应率为63.8%（30/47例），部分反应率为23.4%（11/47例）。其中轻度13例，总体反应率为100%（13/13）；中度21例，总体反应率为90.5%（19/21）；重度13例，总体反应率为69.2%（9/13）。各器官总体反应率最高的是胃肠道达100%（7/7例），其次为皮肤95.8%（23/24例）、肝83.3%（5/6例）和肺部76.9%（10/13例）等；器官完全反应率最高的是皮肤达95.8%。服药期间出现血细胞减少8例（17%），其中出现3~4度血红蛋白下降2例（4.2%）；出现巨细胞病毒再燃3例（6.4%）。停药后cGVHD复发6例（12.7%），复发中位时间为35.5（7~90）d，再次加用芦可替尼后均好转，再次服用芦可替尼反应中位时间为7（5~14）d。中位随访时间为208（33~412）d，死亡3例（6.4%），均死于重度肺部感染；原发病复发3例（6.4%），患者6个月总生存率达95.7%。结论:低剂量芦可替尼挽救性治疗cGVHD患者具有较好的疗效并且安全性良好。

abstractsObjective:To investigate the efficacy and safety of low-dose Ruxolitinib in the treatment of patients with chronic graft-versus-host disease (cGVHD) and refractory to the first-line and/or second-line drugs after allogeneic hematopoietic stem cell transplantation.Methods:The clinical data was retrospectively analyzed of patients diagnosed with cGVHD in Anhui Provincial Hospital from July 9, 2018 to May 23, 2019. They were refractory to first-line and second-line drugs and were given a low-dose of Ruxolitinib (a dose of 5 mg twice daily if body weight ≥ 25 kg and 2.5 mg twice daily if body weight <25 kg). There was 2.5 mg reduction per week or every two weeks if the condition improved until withdrawal. The efficacy and safety of Ruxolitinib were retrospectively analyzed weekly or biweekly. If the condition improved, the dosage would be reduced by 2.5 mg weekly or biweekly until discontinuance. Results:A total of 47 patients were included in the study,and the median time of taking Ruxolitinib was 55 (21-154) days. The median time of taking effect was 14(7-28) days. The overall response rate was 87.2% (41/47). The complete response rate was 63.8% (30/47) and the partial response rate was 23.4%(11/47). Among them, 13 cases were mild and the overall response rate was 100%(13/13). Twenty one cases were moderate and the overall response rate was 90.5%(19/21). Thirteen cases were severe and the overall response rate was 69.2%(9/13). The highest overall response rate of all organs the was 100% in the gastrointestinal tract (7/7), and it was 95.8%(23/24) for the skin, 83.3%(5/6) for the liver and 76.9%(10/13) for the lung. The highest rate of complete organ response was 95.8% for skin. Eight patients (17%) developed cytopenia, of which 2(4.2%) were with a decrease of 3-4 degree hemoglobin. Recrudescence of cytomegalovirus occurred in 3 patients (6.4%). After withdrawal of Ruxolitinib, 6 patients (12.7%) had recurrence of cGVHD. The median time to relapse was 35.5(7-90) days. All of their conditions were improved after addition of Ruxolitinib. The median time of response was 7(5-14) days. The median follow-up was 208(33-412) days. Three patients(6.4%) died, and all of them died of severe pulmonary infection. Three patients (6.4%) had relapse of primary disease. The 6-month overall survival rate was 95.7%.Conclusion:Low-dose Ruxolitinib has good efficacy and safety in the treatment of cGVHD.