Objective: To retrospectively investigate the safety and efficacy of mycofenolate mofetil (MMF) in combination with cyclosporine A (CSA) or tacrolimus (FK506) in the treatment of graft-versus-host disease(GVHD) after allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Methods: 44 patients with various hematological diseases underwent allo-HSCT: 23 received transplant from HLA-matched siblings and 21 from mismatched related donors. The conditioning regimens included modified busulphan plus cyclophosphamide (Cy) regimen for the patients diagnosed as with leukemia and myelodysplastic syndrome (MDS), and Cy plus antihuman thymocyte globulin (ATG) for severe aplastic anemia (sAA). Cyclosporine A (CsA), short term methotrexate (MTX), and MMF were used for the prophylaxis of GVHD for all patients. Acute GVHD (aGVHD) and chronic GVHD (cGVHD) were diagnosed according to the international commonly accepted GVHD diagnostic criteria. MMF was added to the standard C-sA/FK506 with prednisone (PSE) as either the first line therapy, or the second line salvage therapy.
Results: 15 patients were diagnosed as with aGVHD, and 29 with cGVHD. MMF was administrated as a first-line therapy drug in 19 patients and as a second-line therapy drug in 25 patients. For aGVHD the overall response rate was 80% with a complete response (CR) rate of 33.33% and a partial response (PR) rate of 46.66%. The response rates were 70% and 100% respectively for the first line and second line therapy. In 58.3% of the responsive patients the daily dose of PSE could be reduced. For chronic GVHD the overall response rate was 86.2% with a CR rate of 41.37% and a PR rate of 44.83%, and the response rate were 100% and 84.21% for the first and second line therapy respectively. PSE taper was realized in 70.83% of the responsive patients. Side effects occurred in 7 patients, all reversible. The infectious complication rate was 34.09%, only one patient died of interstitial pneumonia. The follow-up of 22 months (10 approximately 65 months) showed that 36 patients (81.82%) survived.
Conclusion: MMF combined with CSA/FK506 is effective and well-tolerated in treatment for aGVHD and cGVHD.