Different efficacy of mycophenolate mofetil as salvage treatment for acute and chronic GVHD after allogeneic stem cell transplant.
OBJECTIVE
The therapeutic options currently available for treating refractory graft-vs.-host disease (GVHD) are limited. Therefore, the present study evaluated the efficacy of mycophenolate mofetil (MMF) as a salvage treatment for acute and chronic GVHD in allograft patients.


METHODS
Twenty-six consecutive patients with refractory acute (13 patients) or chronic (13 patients) GVHD were enrolled. The first-line treatment for all patients with acute GVHD consisted of a combination of cyclosporine A (CyA) and steroids, while the first-line treatment for chronic GVHD was steroids with or without CyA according to the risk group. MMF was added at a dose of 1.5 or 2 g daily and the steroids were tapered in the refractory cases.


RESULTS
Four (30.8%) of 13 patients with refractory acute GVHD responded to MMF. When analyzing the overall results according to the type of acute GVHD, improvement was observed in four (30.8%) of 13 skin cases, four (44.4%) of nine liver cases, and two (22.9%) of nine gut cases. Ten (76.9%) of 13 patients with refractory chronic GVHD responded to MMF. The common side effects were gastrointestinal disturbance (26.9%) or infectious complications (23.1%). The estimated 2-yr survival rate for patients with acute GVHD and chronic GVHD was 33.3% and 53.9%, respectively.


CONCLUSIONS
MMF would appear to be effective and safe for treating refractory chronic GVHD, yet not as effective for treating refractory acute gut GVHD. Accordingly, a prospective randomized clinical trial is warranted to assess the impact of MMF in the treatment of refractory GVHD.
Kim
Jong Gwang
JG
Department of Hematology/Oncology, Kyungpook National University Hospital, Daegu, Korea.
Sohn
Sang Kyun
SK
Kim
Dong Hwan
DH
Lee
Nan Young
NY
Suh
Jang Soo
JS
Lee
Kun Soo
KS
Lee
Kyu Bo
KB
D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002908:Chronic Disease; D005260:Female; D006086:Graft vs Host Disease / Q000150:complications / Q000188:drug therapy*; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008107:Liver Diseases / Q000209:etiology; D008171:Lung Diseases / Q000209:etiology; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use*; D036102:Peripheral Blood Stem Cell Transplantation / Q000009:adverse effects; D016879:Salvage Therapy; D012871:Skin Diseases / Q000209:etiology; D033581:Stem Cell Transplantation / Q000009:adverse effects*; D013997:Time Factors; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous; D016896:Treatment Outcome
D007166:Immunosuppressive Agents; D009173:Mycophenolic Acid