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Etanercept, mycophenolate, denileukin, or pentostatin plus corticosteroids for acute graft-versus-host disease: a randomized phase 2 trial from the Blood and Marrow Transplant Clinical Trials Network. Blood 2009 Jul 16;114(3):511-7 PMID: 19443659 PMCID: PMC2713466

Pubmed ID



Acute graft-versus-host disease (aGVHD) is the primary limitation of allogeneic hematopoietic cell transplantation. Corticosteroids remain the standard initial therapy, yet only 25% to 41% of patients completely respond. This randomized, 4-arm, phase 2 trial was designed to identify the most promising agent(s) for initial therapy for aGVHD. Patients were randomized to receive methylprednisolone 2 mg/kg per day plus etanercept, mycophenolate mofetil (MMF), denileukin diftitox (denileukin), or pentostatin. Patients (n = 180) were randomized; their median age was 50 years (range, 7.5-70 years). Myeloablative conditioning represented 66% of transplants. Grafts were peripheral blood (61%), bone marrow (25%), or umbilical cord blood (14%); 53% were from unrelated donors. Patients who received MMF for prophylaxis (24%) were randomized to a non-MMF arm. At randomization, aGVHD was grade I to II (68%), III to IV (32%), and (53%) had visceral organ involvement. Day 28 complete response rates were etanercept 26%, MMF 60%, denileukin 53%, and pentostatin 38%. Corresponding 9-month overall survival was 47%, 64%, 49%, and 47%, respectively. Cumulative incidences of severe infections were as follows: etanercept 48%, MMF 44%, denileukin 62%, and pentostatin 57%. Efficacy and toxicity data suggest the use of MMF plus corticosteroids is the most promising regimen to compare against corticosteroids alone in a definitive phase 3 trial. This study is registered at as NCT00224874.

Author List

Alousi AM, Weisdorf DJ, Logan BR, BolaƱos-Meade J, Carter S, Difronzo N, Pasquini M, Goldstein SC, Ho VT, Hayes-Lattin B, Wingard JR, Horowitz MM, Levine JE, Blood and Marrow Transplant Clinical Trials Network


Mary M. Horowitz MD, MS Center Director, Professor in the Medicine department at Medical College of Wisconsin
Brent R. Logan PhD Director, Professor in the Institute for Health and Equity department at Medical College of Wisconsin
Marcelo C. Pasquini MD, MS Associate Professor in the Medicine department at Medical College of Wisconsin


2-s2.0-70149109879   140 Citations

MESH terms used to index this publication - Major topics in bold

Acute Disease
Adrenal Cortex Hormones
Anti-Inflammatory Agents, Non-Steroidal
Antineoplastic Agents
Diphtheria Toxin
Drug Therapy, Combination
Graft vs Host Disease
Hematopoietic Stem Cell Transplantation
Immunoglobulin G
Middle Aged
Mycophenolic Acid
Receptors, Tumor Necrosis Factor
Recombinant Fusion Proteins
Survival Rate
Treatment Outcome
jenkins-FCD Prod-299 9ef562391eceb2b8f95265c767fbba1ce5a52fd6