Low incidence of severe cGvHD and late NRM in a phase II trial of thymoglobulin, tacrolimus and sirolimus for GvHD prevention

Bone Marrow Transplant. 2017 Sep;52(9):1304-1310. doi: 10.1038/bmt.2017.95. Epub 2017 Jun 5.

Abstract

Chronic GvHD (cGvHD) is the leading cause of late non-relapse mortality (NRM) and morbidity after allogeneic hematopoietic stem cell transplant (AHSCT). We analyzed the late effects of a phase II trial testing the efficacy of intermediate dose rabbit anti-thymocyte globulin (Thymoglobulin Thymo) in combination with tacrolimus and sirolimus (TTS) in 47 patients (pts) for the prevention of acute and chronic GvHD after unrelated AHSCT. The median follow-up was 45.2 months. The cumulative incidence of NIH severe cGvHD at 48 months was 6.4% with no new occurrences past 6 months for the entire follow-up period. The overall cumulative incidence of cGvHD was 44.7%. Out of 20 pts who are alive and disease-free at the last follow-up, only 4 pts continue to need systemic immune suppression. We observed low late NRM with only 3 transplant-related deaths after 6 months post transplant. At 4 years of follow-up, the overall cumulative incidence of NRM and disease relapse was 27.7% and 30.0%, respectively. PFS and overall survival (OS) at 4 years were 42 and 47%. At long term follow-up, TTS was associated with low incidence of severe cGvHD and late NRM.

Publication types

  • Clinical Trial, Phase II

MeSH terms

  • Adult
  • Aged
  • Antilymphocyte Serum / pharmacology
  • Antilymphocyte Serum / therapeutic use*
  • Female
  • Graft vs Host Disease / mortality
  • Graft vs Host Disease / prevention & control*
  • Humans
  • Immunosuppressive Agents / pharmacology
  • Immunosuppressive Agents / therapeutic use*
  • Incidence
  • Male
  • Middle Aged
  • Risk Assessment
  • Sirolimus / pharmacology
  • Sirolimus / therapeutic use*
  • Survival Rate
  • Tacrolimus / pharmacology
  • Tacrolimus / therapeutic use*
  • Young Adult

Substances

  • Antilymphocyte Serum
  • Immunosuppressive Agents
  • thymoglobulin
  • Sirolimus
  • Tacrolimus