A phase III study of infliximab and corticosteroids for the initial treatment of acute graft-versus-host disease

Biol Blood Marrow Transplant. 2009 Dec;15(12):1555-62. doi: 10.1016/j.bbmt.2009.08.003. Epub 2009 Sep 25.


Anti-Tumor Necrosis Factor Alpha (TNF-alpha) therapy with infliximab has shown to be effective for patients with steroid-refractory acute graft-versus-host disease (aGVHD). An open-labeled, phase III trial was conducted to determine if the addition of infliximab to steroids could improve results for patients with newly diagnosed grade II-IV aGVHD. A total of 63 patients were randomized either to 2 mg/kg/day methylprednisolone (MP) or infliximab+ MP. Average age was 47 years (range: 20-70 years); 64% were male. Fifty-three percent and 51% of patients received a matched-sibling and/or bone marrow (BM) graft. Sixty-seven percent had grade II, 33% grade III-IV aGVHD; 62% had skin, 53% gastrointestinal (GI), and 7% had liver involvement. At days 7 and 28, the response rate for infliximab+ MP versus MP was 52% versus 78%, P=.03 and 62% versus 58%, P=.7, respectively. Cumulative incidences of GVHD-related mortality, nonrelapse mortality (NRM), and overall survival (OS) were not significantly different between the 2 groups (GVHD-related mortality: 38% versus 32%, P=.6; NRM: 52% versus 36%, P=.3; OS: 17% and 28%, P=.4 for infliximab+ MP versus MP, respectively). Patients with newly diagnosed aGVHD derive no benefit from the addition of anti-TNF-alpha therapy with infliximab when compared to corticosteroids alone.

Publication types

  • Clinical Trial, Phase III
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acute Disease
  • Adult
  • Aged
  • Antibodies, Monoclonal / therapeutic use*
  • Bone Marrow Transplantation / immunology
  • Female
  • Graft vs Host Disease / drug therapy*
  • Graft vs Host Disease / immunology
  • Humans
  • Infliximab
  • Male
  • Methylprednisolone / therapeutic use*
  • Middle Aged
  • Peripheral Blood Stem Cell Transplantation
  • Prospective Studies
  • Treatment Outcome
  • Young Adult


  • Antibodies, Monoclonal
  • Infliximab
  • Methylprednisolone