Combination of CsA, MTX and low-dose, short-course mycophenolate mofetil for GVHD prophylaxis.

In an effort to reduce the incidence and severity of acute GVHD (aGVHD), we have developed a new prophylaxis regimen combining cyclosporine and MTX with a short 30-day course of low-dose (500 mg per day) mycophenolate mofetil. This regimen was studied prospectively 100 patients undergoing HLA-matched and 1-antigen-mismatched allogeneic peripheral blood SCT from related donors. The cumulative incidence of aGVHD was 16% (grades II-IV (9.5%) and grades III-IV (1%)). The cumulative incidence of chronic GVHD (cGVHD) was 53% with 28% extensive cGVHD. The cumulative incidence of transplant-related mortality at 100 days and 3 years were 6 and 13%. The estimated probabilities of disease-free survival at 3 years in standard- and high-risk patients were 77 and 30%, respectively (P<0.0001). The estimated probabilities of overall survival at 3 years in standard- and high-risk patients were 77 and 37%, respectively (P<0.0001). These data show a substantial decrease in the risk of developing aGVHD without an increase in relapse or any adverse impact on survival in standard-risk patients.