Pharmacokinetics-based Mycophenolate Mofetil Dosing for GVHD Prevention
Status:
Completed
Trial end date:
2016-01-01
Target enrollment:
Participant gender:
Summary
Bone marrow transplantation (BMT) is used to successfully treat high-risk forms of leukemia,
lymphoma, and other childhood cancers that were once considered incurable. A major barrier to
the application of this life-saving treatment is acute graft-versus-host disease (GVHD) which
develops in approximately 30-80% of patients and is a leading cause of death from transplant
complications. Current GVHD prevention methods are not very efficacious and lead to
unacceptable side effects. Mycophenolate mofetil (MMF), an anti-rejection medication used in
solid organ transplants, has shown great promise in BMT recipients. The effectiveness of MMF
depends on blood levels of mycophenolic acid (MPA, the active form of MMF). Different
patients have been found to have different blood levels of MPA when they are given the same
dose of MMF. The purpose of this study is to study a novel method of giving MMF based on its
metabolism (pharmacokinetics) to achieve desired blood levels of MPA for prevention of GVHD.
Non-invasive ways of monitoring the drug exposure will also be studied. The ultimate goal of
this study is to improve approaches to GVHD prevention and improve outcomes of BMT in
children.