Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Beijing, China.
Department of Hematology, the Second Hospital of Shanxi Medical University, Taiyuan, 030001, China.
Ann Hematol. 2021 Jan;100(1):169-180. doi: 10.1007/s00277-020-04273-2. Epub 2020 Nov 7.
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.
我们的研究目的是确定鲁索利替尼在 HLA 单倍体相合造血干细胞移植（haplo-HSCT）受者中治疗多重耐药（MDR）-移植物抗宿主病（GVHD，n=34）的疗效。MDR-GVHD 定义为至少接受 3 种治疗后仍无改善的 GVHD。MDR-急性 GVHD（aGVHD）和 MDR-慢性 GVHD（cGVHD）患者的既往 GVHD 治疗中位数均为 4 种。对于 MDR-aGVHD（n=15），缓解的中位时间为 10 天（范围 2-65 天），总体缓解率（ORR）为 60.0%（9/15），包括 40.0%（6/15）完全缓解（CR）和 20.0%（3/15）部分缓解（PR）。鲁索利替尼治疗后，1 年总生存率为 66.7%。鲁索利替尼治疗后血液学和感染毒性的发生率分别为 73.3%和 46.7%。对于 MDR-cGVHD（n=19），缓解的中位时间为 29 天（范围 6-175 天），ORR 为 89.5%（17/19），包括 26.3%（5/19）CR 和 63.2%（12/19）PR。所有患者在我们的最后一次随访时均存活。鲁索利替尼治疗后血液学和感染毒性的发生率分别为 36.8%和 47.4%。鲁索利替尼是 haplo-HSCT 受者中治疗 MDR-GVHD 的有效挽救治疗方法。
