Ruxolitinib is an effective salvage treatment for multidrug-resistant graft-versus-host disease after haploidentical allogeneic hematopoietic stem cell transplantation without posttransplant cyclophosphamide

• Published in: Annals of hematology 2021, Vol.100 (1), p.169-180
• Main Authors: Zhao, Jiao-Yu, Liu, Si-Ning, Xu, Lan-Ping, Zhang, Xiao-Hui, Wang, Yu, Chen, Yu-Hong, Liu, Kai-Yan, Huang, Xiao-Jun, Mo, Xiao-Dong
• Format: Article
• Language: English
• Subjects: Adolescent; Adult; Child; Child, Preschool; Cyclophosphamide; Drug resistance; Drug Resistance, Multiple - drug effects; Drug Resistance, Multiple - physiology; Female; Follow-Up Studies; Graft versus host disease; Graft vs Host Disease - diagnosis; Graft vs Host Disease - drug therapy; Hematology; Hematopoietic Stem Cell Transplantation - trends; Humans; Inhibitor drugs; Male; Medicine; Medicine & Public Health; Middle Aged; Multidrug resistant organisms; Oncology; Original Article; Pyrazoles - therapeutic use; Retrospective Studies; Salvage Therapy - trends; Stem cell transplantation; Stem cells; Transplantation, Haploidentical - trends; Transplantation, Homologous - trends; Treatment Outcome; Young Adult
• ISSN: 0939-5555; 1432-0584
• DOI: 10.1007/s00277-020-04273-2

The purpose of our study is to identify the efficacy of ruxolitinib in human leukocyte antigen (HLA) haploidentical hematopoietic stem cell transplantation (haplo-HSCT) recipients with multidrug-resistant (MDR)-graft-versus-host disease (GVHD, n = 34). MDR-GVHD was defined as GVHD showing no improvement after at least 3 types of treatments. The median number of previous GVHD-therapies was 4 for both MDR-acute GVHD (aGVHD) and MDR-chronic GVHD (cGVHD). For MDR-aGVHD ( n = 15), the median time to response was 10 days (range 2 to 65), and the overall response rate (ORR) was 60.0% (9/15), including 40.0% (6/15) complete response (CR) and 20.0% (3/15) partial response (PR). The 1-year probability of overall survival after ruxolitinib was 66.7%. The rates of hematologic and infectious toxicities were 73.3% and 46.7% after ruxolitinib treatment. For MDR-cGVHD ( n = 19), the median time to response was 29 days (range 6 to 175), and the ORR was 89.5% (17/19), including 26.3% (5/19) CR and 63.2% (12/19) PR. All patients remained alive until our last follow-up. The rates of hematologic and infectious toxicities were 36.8% and 47.4% after ruxolitinib treatment. Ruxolitinib is an effective salvage treatment for MDR-GVHD in haplo-HSCT recipients.