Efficacy of mycophenolate mofetil in the treatment of chronic graft-versus-host disease.
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.
Lopez
Francisco
F
Division of Hematology and Hematopoietic Cell Transplantation, City of Hope National Medical Center, Duarte, California 91010, USA.
Parker
Pablo
P
Nademanee
Auayporn
A
Rodriguez
Roberto
R
Al-Kadhimi
Zaid
Z
Bhatia
Ravi
R
Cohen
Sandra
S
Falk
Peter
P
Fung
Henry
H
Kirschbaum
Mark
M
Krishnan
Amrita
A
Kogut
Neil
N
Molina
Arturo
A
Nakamura
Ryotaro
R
O'Donnell
Margaret
M
Popplewell
Leslie
L
Pullarkat
Vinod
V
Rosenthal
Joseph
J
Sahebi
Firoozeh
F
Smith
Eileen
E
Snyder
David
D
Somlo
George
G
Spielberger
Ricardo
R
Stein
Anthony
A
Sweetman
Robert
R
Zain
Jasmine
J
Forman
Stephen
S
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
D009173:Mycophenolic Acid