Evaluation of mycophenolate mofetil for initial treatment of chronic graft-versus-host disease
Open Access
- 21 May 2009
- journal article
- research article
- Published by American Society of Hematology in Blood
- Vol. 113 (21), 5074-5082
- https://doi.org/10.1182/blood-2009-02-202937
Abstract
We conducted a double-blind, randomized multicenter trial to determine whether the addition of mycophenolate mofetil (MMF) improves the efficacy of initial systemic treatment of chronic graft-versus-host disease (GVHD). The primary endpoint was resolution of chronic GVHD and withdrawal of all systemic treatment within 2 years, without secondary treatment. Enrollment of 230 patients was planned, providing 90% power to observe a 20% difference in success rates between the 2 arms. The study was closed after 4 years because the interim estimated cumulative incidence of success for the primary endpoint was 23% among 74 patients in the MMF arm and 18% among 77 patients in the control arm, indicating a low probability of positive results for the primary endpoint after completing the study as originally planned. Analysis of secondary endpoints showed no evidence of benefit from adding MMF to the systemic regimen first used for treatment of chronic GVHD. The estimated hazard ratio of death was 1.99 (95% confidence interval, 0.9-4.3) among patients in the MMF arm compared with the control arm. MMF should not be added to the initial systemic treatment regimen for chronic GVHD. This trial was registered at www.clinicaltrials.gov as #NCT00089141 on August 4, 2004.Keywords
This publication has 42 references indexed in Scilit:
- Comparison of two mycophenolate mofetil dosing regimens after hematopoietic cell transplantationBone Marrow Transplantation, 2009
- Treatment of High-Risk Chronic GVHDTransplantation and Cellular Therapy, 2008
- Murine Models of Chronic Graft-versus-Host Disease: Insights and Unresolved IssuesTransplantation and Cellular Therapy, 2008
- Relapse risk in patients with malignant diseases given allogeneic hematopoietic cell transplantation after nonmyeloablative conditioningBlood, 2007
- A Limited Sampling Model for Estimation of Total and Unbound Mycophenolic Acid (MPA) Area Under the Curve (AUC) in Hematopoietic Cell Transplantation (HCT)Therapeutic Drug Monitoring, 2006
- Measuring Therapeutic Response in Chronic Graft-versus-Host Disease: National Institutes of Health Consensus Development Project on Criteria for Clinical Trials in Chronic Graft-versus-Host Disease: IV. Response Criteria Working Group ReportTransplantation and Cellular Therapy, 2006
- Efficacy of mycophenolate mofetil in the treatment of chronic graft-versus-host diseaseTransplantation and Cellular Therapy, 2005
- Duration of immunosuppressive treatment for chronic graft-versus-host diseaseBlood, 2004
- Different efficacy of mycophenolate mofetil as salvage treatment for acute and chronic GVHD after allogeneic stem cell transplantEuropean Journal of Haematology, 2004
- Clinical Pharmacokinetics of Mycophenolate MofetilClinical Pharmacokinetics, 1998