Mycophenolate‐based graft versus host disease prophylaxis is not inferior to methotrexate in myeloablative‐related donor stem cell transplantation

We retrospectively reviewed 242 patients who received related donor myeloablative peripheral blood hematopoietic cell transplantation. We compared patients who received mycophenolate (MMF)/cyclosporine (CSA) ( n  = 71), to historical controls who received methotrexate (MTX)/CSA ( n  = 172). There were no differences in overall survival, nonrelapse mortality, and relapse. The MMF/CSA group had significantly faster neutrophil and platelet engraftment: medians of 13 versus 18 days and 10 versus 14 days, respectively ( P  = 0.001). The cumulative incidence of acute graft versus host disease (GVHD) (Grades, 2–4) was significantly lower in the MMF/CSA group (45.1 vs. 74.4%, P  < 0.001). The MMF/CSA group showed a lower incidence of skin (51.5 vs. 72.1%, P  < 0.001) and liver acute GVHD (11.3 vs. 54.2%, P  < 0.001) and a higher incidence of lung (42.2 vs. 19.0%, P  = 0.045), eye (59.7 vs. 30.1%, P  < 0.001), and mouth (72.8 vs. 56.4%, P  = 0.001) chronic GVHD but only eye chronic GVHD was confirmed in propensity score matching (PSM) analysis. The incidence of cytomegalovirus (CMV) viremia was higher in the MMF/CSA group (55.8 vs. 39.6%, P  < 0.001) but this was not confirmed in PSM analysis. MMF/CSA was identified as an independent favorable factor for acute GVHD ( P  < 0.001, hazard ratio, 0.41) but as a possible adverse risk factor for CMV viremia as this was not found to be statistically significant in PSM analysis. MMF/CSA in myeloablative matched related donor peripheral blood stem cell transplant is not inferior as GVHD prophylaxis in comparison with MTX/CSA and is associated with faster engraftment but a potentially higher risk of CMV viremia.Am. J. Hematol. 90:392–399, 2015. © 2015 Wiley Periodicals, Inc.