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

Abstract
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.
AuthorsJiao-Yu Zhao, Si-Ning Liu, Lan-Ping Xu, Xiao-Hui Zhang, Yu Wang, Yu-Hong Chen, Kai-Yan Liu, Xiao-Jun Huang, Xiao-Dong Mo
JournalAnnals of hematology (Ann Hematol) Vol. 100 Issue 1 Pg. 169-180 (Jan 2021) ISSN: 1432-0584 [Electronic] Germany
PMID33159239 (Publication Type: Journal Article)
Chemical References
  • Nitriles
  • Pyrazoles
  • Pyrimidines
  • ruxolitinib
  • Cyclophosphamide
Topics
  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Cyclophosphamide
  • Drug Resistance, Multiple (drug effects, physiology)
  • Female
  • Follow-Up Studies
  • Graft vs Host Disease (diagnosis, drug therapy)
  • Hematopoietic Stem Cell Transplantation (trends)
  • Humans
  • Male
  • Middle Aged
  • Nitriles
  • Pyrazoles (therapeutic use)
  • Pyrimidines
  • Retrospective Studies
  • Salvage Therapy (trends)
  • Transplantation, Haploidentical (trends)
  • Transplantation, Homologous (trends)
  • Treatment Outcome
  • Young Adult

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