TY - JOUR
T1 - Phase 3 clinical trial of steroids/mycophenolate mofetil vs steroids/placebo as therapy for acute GVHD
T2 - BMT CTN 0802
AU - Bolaños-Meade, Javier
AU - Logan, Brent R.
AU - Alousi, Amin M.
AU - Antin, Joseph H.
AU - Barowski, Kate
AU - Carter, Shelly L.
AU - Goldstein, Steven C.
AU - Hexner, Elizabeth O.
AU - Horowitz, Mary M.
AU - Lee, Stephanie J.
AU - Levine, John E.
AU - MacMillan, Margaret L.
AU - Martin, Paul J.
AU - Mendizabal, Adam M.
AU - Nakamura, Ryotaro
AU - Pasquini, Marcelo C.
AU - Weisdorf, Daniel J.
AU - Westervelt, Peter
AU - Ho, Vincent T.
N1 - Publisher Copyright:
© 2014 by The American Society of Hematology.
PY - 2014/11/20
Y1 - 2014/11/20
N2 - Corticosteroids are the accepted primary therapy for acute graft-versus-host disease (GVHD), but durable responses are seen in only about half of the patients. Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 0802, a phase 3 multicenter randomized double-blinded trial,was designed to test whether mycophenolate mofetil (MMF) plus corticosteroids was superior to corticosteroids alone as initial therapy for acute GVHD. Patients with newly diagnosed acute GVHD were eligible if they required systemic therapy. Patients were randomized to receive prednisone with either MMF or placebo. The primary end point was acute or chronic GVHD-free survival at day 56 after initiation of therapy. A futility rule for GVHD-free survival at day 56 was met at a planned interim analysis after 235 patients (of 372) were enrolled: 116 MMF, 119 placebo. Baseline characteristics were well balanced between treatment groups including grade and organ distribution of GVHD. GVHD-free survival at day 56, cumulative incidence of chronic GVHD at 12 months, overallsurvival, Epstein-Barr virus reactivation, severe, life-threatening infections, relapse at 12 months, and quality of life were similar. The addition of MMF to corticosteroids as initial therapy for acute GVHD does not improve GVHD-free survival compared with corticosteroids alone. This trial was registered at www.clinicaltrials.gov as #NCT01002742.
AB - Corticosteroids are the accepted primary therapy for acute graft-versus-host disease (GVHD), but durable responses are seen in only about half of the patients. Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 0802, a phase 3 multicenter randomized double-blinded trial,was designed to test whether mycophenolate mofetil (MMF) plus corticosteroids was superior to corticosteroids alone as initial therapy for acute GVHD. Patients with newly diagnosed acute GVHD were eligible if they required systemic therapy. Patients were randomized to receive prednisone with either MMF or placebo. The primary end point was acute or chronic GVHD-free survival at day 56 after initiation of therapy. A futility rule for GVHD-free survival at day 56 was met at a planned interim analysis after 235 patients (of 372) were enrolled: 116 MMF, 119 placebo. Baseline characteristics were well balanced between treatment groups including grade and organ distribution of GVHD. GVHD-free survival at day 56, cumulative incidence of chronic GVHD at 12 months, overallsurvival, Epstein-Barr virus reactivation, severe, life-threatening infections, relapse at 12 months, and quality of life were similar. The addition of MMF to corticosteroids as initial therapy for acute GVHD does not improve GVHD-free survival compared with corticosteroids alone. This trial was registered at www.clinicaltrials.gov as #NCT01002742.
UR - http://www.scopus.com/inward/record.url?scp=84911435257&partnerID=8YFLogxK
U2 - 10.1182/blood-2014-06-577023
DO - 10.1182/blood-2014-06-577023
M3 - Article
C2 - 25170121
AN - SCOPUS:84911435257
SN - 0006-4971
VL - 124
SP - 3221
EP - 3227
JO - Blood
JF - Blood
IS - 22
ER -