摘要
目的:分析芦可替尼治疗异基因造血干细胞移植后合并糖皮质激素难治性移植物抗宿主病(SR-GVHD)的疗效及预后。方法:回顾性分析42例移植后合并SR-GVHD患者的临床资料,其中糖皮质激素难治性急性移植物抗宿主病(SR-aGVHD)20例(均为Ⅲ~Ⅳ度),糖皮质激素难治性慢性移植物抗宿主病(SR-cGVHD)22例(均为中/重度)。确诊SR-GVHD的患者给予芦可替尼挽救性治疗,在初始治疗的第28天进行疗效评估,并对影响疗效的相关因素进行分析。结果:20例SR-aGVHD患者治疗28天整体反应率(ORR)为60.0%(12/20),其中完全缓解(CR)率为20.0%(4/20);中位随访时间37.7(1~107)周,其6个月和1年的总体生存率(OS)分别为55.0%(95%CI 33.24%~76.76%)、45.0%(95%CI 23.24%~66.76%);6个月和1年的无失败生存率(FFS)分别为50.0%(95%CI 28.05%~71.95%)、40.0%(95%CI 18.44%~61.56%)。22例SR-cGVHD患者治疗28天ORR为72.7%(16/22),其中CR率9.1%(2/22);中位随访时间76.9(5~110)周,治疗6个月及1年的OS分别为77.3%(95%CI 59.86%~94.74%)、72.7%(95%CI 54.06%~91.32%),6个月及1年的FFS分别为77.3%(95%CI 59.86%~94.74%)、72.7%(95%CI 54.06%~91.32%)。单因素分析显示,Ⅳ度aGVHD、重度cGVHD可能预示芦可替尼治疗疗效不佳(ORR:Ⅲ度vsⅣ度为88.9%vs 36.4%,P=0.028;中度vs重度为92.3%vs 44.4%,P=0.023)。结论:芦可替尼是挽救性治疗SR-GVHD安全、有效的药物,需要大规模的前瞻性研究证实。
Objective:To evaluate the efficacy and prognosis of ruxolitinib in the treatment of steroid-refractory graft-versus-host disease(SR-GVHD)after allogeneic hematopoietic stem cell transplantation.Method:A total of 42 patients who had SR-GVHD were analyzed retrospectively.The 42 patients were classified to SR-aGVHD(n=20,all gradeⅢorⅣ)or SR-cGVHD(n=22,all moderate or severe)group.All patients enrolled were given ruxolitinib as salvage therapy.The efficacy of ruxolitinib was evaluated at day 28 of the initial treatment.The associated factors on clinical outcome were analyzed.Result:The overall response rate(ORR)was 60.0%[20.0%complete response(CR)]in SR-aGVHD group,while for SR-cGVHD group the ORR was 72.7%(9.1%CR).The 6-month and 1-year overall survival(OS)were 55.0%(95%CI 33.24%-76.76%)and 45.0%(95%CI 23.24%-66.76%)respectively in SR-aGVHD group.The median follow-up time was 37.7(1-107)weeks,the 6-month and 1-year failure-free survival(FFS)were 50.0%(95%CI 28.05%-71.95%)and 40.0%(95%CI 18.44%-61.56%)respectively in SR-aGVHD patients.While for SR-cGVHD patients,the median follow-up time was 76.9(5-110)weeks,the 6-month and 1-year OS were 77.3%(95%CI 59.86%-94.74%)and 72.7%(95%CI 54.06%-91.32%)respectively.The 6-month and 1-year FFS was equal to data of OS.Univariate analysis showed that gradeⅣaGVHD and severe cGVHD may indicate poor efficacy of ruxolitinib(ORR:gradeⅢvsⅣwas88.9%vs 36.4%,P=0.028;moderate vs severe was 92.3%vs 44.4%,P=0.023).Conclusion:Ruxolitinib may constitute a promising new salvage treatment option for SR-GVHD,which should be verified in prospective trials and more clinical practice.
作者
赵菲
王佳丽
施圆圆
杨栋林
魏嘉璘
庞爱明
何祎
姜尔烈
冯四洲
韩明哲
ZHAO Fei;WANG Jiali;SHI Yuanyuan;YANG Donglin;WEI Jialin;PANG Aiming;HE Yi;JIANG Erlie;FENG Sizhou;HAN Mingzhe(Institute of Hematology&Blood Diseases Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Tianjin,300020,China)
出处
《临床血液学杂志》
CAS
2020年第1期18-24,共7页
Journal of Clinical Hematology
基金
国家自然科学基金(No:81670171)
中国医学科学院医学与健康科技创新工程(No:2016-I2M-3-023).
