title: Efficacy of mycophenolate mofetil in the treatment of chronic graft-versus-host disease.
abstract: Current treatment of chronic graft-versus-host disease (cGVHD) with prednisone (PSE) alone or with added cyclosporine or tacrolimus still has a very high failure and complication rate, and new treatment approaches are needed for both primary and salvage therapy. Mycophenolate mofetil (MMF) is an immunosuppressive agent currently in use for acute graft-versus-host disease prophylaxis. To determine whether MMF had activity in the treatment of cGVHD, we added MMF to standard cyclosporine, tacrolimus, and/or PSE as salvage/second-line (n = 24) or first-line (n = 10) therapy in 34 patients. Nine (90%) of 10 patients receiving first-line and 18 (75%) of 24 receiving second-line MMF therapy responded. Twelve (35%) patients had a complete remission, 15 (44%) had a partial remission, 5 (15%) had stable disease, and only 2 (6%) had progressive disease. Out of 30 patients receiving PSE, 22 (73%) were able to decrease PSE doses (median decrease of 50%; range, 25%-100%). With a median follow-up of 24 months (range, 6-28 months), 29 (85%) patients are alive. Three patients had to discontinue MMF because of abdominal cramps within 3 months of starting treatment. These data suggest that MMF is an active, well-tolerated agent in the treatment of cGVHD and may have a beneficial effect on the survival of patients with this complication.
authors.affiliation: Division of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, California 91010, USA.
authors.forename: Francisco
authors.initials: F
authors.lastname: Lopez
authors.forename: Pablo
authors.initials: P
authors.lastname: Parker
authors.forename: Auayporn
authors.initials: A
authors.lastname: Nademanee
authors.forename: Roberto
authors.initials: R
authors.lastname: Rodriguez
authors.forename: Zaid
authors.initials: Z
authors.lastname: Al-Kadhimi
authors.forename: Ravi
authors.initials: R
authors.lastname: Bhatia
authors.forename: Sandra
authors.initials: S
authors.lastname: Cohen
authors.forename: Peter
authors.initials: P
authors.lastname: Falk
authors.forename: Henry
authors.initials: H
authors.lastname: Fung
authors.forename: Mark
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authors.lastname: Kirschbaum
authors.forename: Amrita
authors.initials: A
authors.lastname: Krishnan
authors.forename: Neil
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authors.lastname: Kogut
authors.forename: Arturo
authors.initials: A
authors.lastname: Molina
authors.forename: Ryotaro
authors.initials: R
authors.lastname: Nakamura
authors.forename: Margaret
authors.initials: M
authors.lastname: O'Donnell
authors.forename: Leslie
authors.initials: L
authors.lastname: Popplewell
authors.forename: Vinod
authors.initials: V
authors.lastname: Pullarkat
authors.forename: Joseph
authors.initials: J
authors.lastname: Rosenthal
authors.forename: Firoozeh
authors.initials: F
authors.lastname: Sahebi
authors.forename: Eileen
authors.initials: E
authors.lastname: Smith
authors.forename: David
authors.initials: D
authors.lastname: Snyder
authors.forename: George
authors.initials: G
authors.lastname: Somlo
authors.forename: Ricardo
authors.initials: R
authors.lastname: Spielberger
authors.forename: Anthony
authors.initials: A
authors.lastname: Stein
authors.forename: Robert
authors.initials: R
authors.lastname: Sweetman
authors.forename: Jasmine
authors.initials: J
authors.lastname: Zain
authors.forename: Stephen
authors.initials: S
authors.lastname: Forman
mesh_terms: D002908:Chronic Disease; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000188:drug therapy* / Q000503:physiopathology; D006801:Humans; D008297:Male; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000031:analogs & derivatives*; D012189:Retrospective Studies
chemical_list: D009173:Mycophenolic Acid