Tocilizumab for the treatment of severe steroid-refractory acute graft-versus-host disease of the lower gastrointestinal tract

Bone Marrow Transplant. 2019 Feb;54(2):212-217. doi: 10.1038/s41409-018-0236-z. Epub 2018 May 24.


Steroid-refractory (SR) acute gastrointestinal (GI) graft-versus-host disease (GVHD) is associated with significant mortality in allogeneic hematopoietic cell transplantation recipients. We retrospectively evaluated the efficacy of tocilizumab for the treatment of SR biopsy-proven acute lower GI GVHD in 16 consecutive adult transplant recipients between October 2015 and July 2016. Tocilizumab 8 mg/kg was administered every 2 weeks until achievement of complete response, defined as resolution of all manifestations of GI GVHD, or until patients had progression or initiation of other therapy. Ten of 16 patients (62.5%; 95% CI, 0.39-82) achieved a complete response after a median time of 11 days (range, 2-28 days) from tocilizumab initiation. The median time to response onset (improvement in stage by at least 1) was 1 day (range, 1-4 days). Tocilizumab was administered at a median of 9 days (range, 3-75 days) from GVHD diagnosis and 10 days (range, 3-75 days) from initiation of high-dose steroids. At a median follow-up of 7.6 months (range, 0.8-27.7 months) from initiation of tocilizumab, 6/16 (37.5%) patients are alive and free of their underlying hematologic malignancy. Tocilizumab appears to be a highly active agent for the treatment of severe SR lower GI acute GVHD.

MeSH terms

  • Adult
  • Aged
  • Antibodies, Monoclonal, Humanized / pharmacology
  • Antibodies, Monoclonal, Humanized / therapeutic use*
  • Female
  • Gastrointestinal Diseases / drug therapy*
  • Gastrointestinal Diseases / mortality
  • Gastrointestinal Diseases / pathology
  • Graft vs Host Disease / drug therapy*
  • Graft vs Host Disease / mortality
  • Hematologic Neoplasms / complications
  • Hematologic Neoplasms / pathology
  • Hematologic Neoplasms / therapy
  • Hematopoietic Stem Cell Transplantation / adverse effects
  • Humans
  • Lower Gastrointestinal Tract
  • Male
  • Middle Aged
  • Retrospective Studies
  • Steroids / pharmacology*
  • Survival Analysis
  • Time Factors
  • Treatment Outcome
  • Young Adult


  • Antibodies, Monoclonal, Humanized
  • Steroids
  • tocilizumab