Objective: To investigate the efficacy of anti-CD(25) monoclonal antibody for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in allogeneic hematopoietic stem cell transplantation (allo-HSCT) recipients. Methods: A total of 80 patients with SR-aGVHD from January 1st 2012 to December 31st 20l6 were enrolled in this study. Acute GVHD were classified as classic aGVHD (n=72) and late-onset aGVHD (n=8). Anti-CD(25) monoclonal antibodys (mAb) were administrated on days 1, 4, 8, 15, and 22. The efficacy of anti-CD(25) mAb was evaluated at day 28 after the initial treatment. The associated factors of clinical outcome were analyzed. Results: The overall response (OR) rate of anti-CD(25) mAb was 75% (60/80), with complete response (CR) rate, partial response (PR) rate and no response(NR) rate 52.5% (42/80), 22.5% (18/80), and 25% (20/80), respectively. GVHD-relapse was not observed with a median follow-up time of 394.5 days (range, 12-1 761 days). The 6-month overall survival (OS) rate was 68.4%(95%CI 63.2%-73.6%). The 1-year OS rate was 63.1% (95%CI 57.6%-68.6%), and 2-years OS rate was 50.7% (95%CI 44.3%-57.1%). Non-relapse mortality (NRM) rate of 1 and 3 years was 32.6% (95%CI 27.2%-38%) and 41.7% (95%CI 35.3%-48.1%), respectively. The 1 and 2 years cumulative incidence of chronic graft versus host disease (cGVHD) was 32.9% (95%CI 26.4%-39.4%) and 38.9% (95%CI 31.8%-46.0%). By univariate and multivariate analysis, liver involvement was an independent poor risk factor of SR-aGVHD (OR=4.66, 95%CI 1.145-18.962, P=0.032). Conclusion: Anti-CD(25) mAb serves as an alternative and effective salvage therapy for SR-aGVHD at present. Liver involvement is a predictive factor of poor response in patients with SR-aGVHD.
目的： 分析CD(25)单克隆抗体（简称CD(25)单抗）治疗异基因造血干细胞移植后合并糖皮质激素（简称激素）耐药型急性移植物抗宿主病(aGVHD)的疗效及预后。 方法： 回顾性总结苏州工业园区星海医院血液科2012年1月1日至2016年12月31日移植后合并激素耐药型aGVHD的80例患者的临床资料，其中经典型aGVHD 72例，迟发型aGVHD 8例。患者给予CD(25)单抗治疗（第1、4、8、15、22天），在CD(25)单抗初始治疗后的第28天对疗效进行评估，并对影响疗效的相关因素进行分析。 结果： 总有效率为75.0%（60/80），其中完全缓解率为52.5%（42/80），部分缓解率22.5%（18/80），无效率25.0%（20/80）。从CD(25)单抗治疗开始随访，中位随访时间394.5（12~1 761）d。随访期间未发现aGVHD复发，6个月总生存（OS）率为68.4% (95%CI 63.2%~73.6%)，1年OS率为63.1% (95%CI 57.6%~68.6%)，3年OS率为50.7% (95%CI 44.3%~57.1%)；1年非复发死亡（NRM）率为32.6% (95%CI 27.2%~38.0%)，3年NRM率为41.7%（95%CI 35.3%~48.1%）；1年和2年慢性移植物抗宿主病的累积发生率分别为32.9% (95%CI 26.4%~39.4%)和38.9%（95%CI 31.8%~46.0%）。单因素和多因素分析均显示，肝脏受累是影响CD(25)单抗治疗激素耐药型aGVHD疗效的独立危险因素（OR=4.66，95% CI 1.145~18.962，P=0.032）。 结论： CD(25)单抗仍然是现阶段治疗激素耐药型aGVHD安全、有效的手段之一，本研究显示肝脏受累的激素耐药型aGVHD疗效不佳、预后差。.
Keywords: Acute graft vs host disease; Anti-CD(2)5 monoclonal antibody; Hematopoietic stem cell transplantation.