Mycophenolate mofetil (MMF) is frequently used in hematopoietic cell transplantation (HCT) for graft-versus-host disease (GVHD) prophylaxis and to facilitate engraftment. We previously reported that a higher level of mycophenolic acid can be achieved with an MMF dose of 3 g/day than with 2 g/day. Here, we retrospectively compared clinical outcomes of reduced-intensity conditioning (RIC) double umbilical cord blood (dUCB) HCT recipients receiving cyclosporine A with MMF 2 g (n= 93) versus 3 g (n= 175) daily. Multiple regression analysis adjusted for antithymocyte globulin in the conditioning revealed that MMF 3 g/day led to a 49% relative risk (RR) reduction in grade II to IV acute GVHD rate (RR, .51; 95% confidence interval, .36 to .72; P < .01). However, the higher MMF dose was not protective for chronic GVHD. Additionally, MMF dose was not an independent predictor of neutrophil engraftment or treatment-related mortality at 6 months or 2-year post-transplantation disease relapse, disease-free survival, or overall survival. Higher MMF dose did not increase risk of infectious complications, and infection-related mortality was similar for both MMF doses. Our data indicate that MMF 3 g/day reduces the risk of acute GVHD without affecting other clinical outcomes and should be used for GVHD prophylaxis after RIC dUCB transplantation. -Mycophenolate mofetil 3 g/day dose is associated with 49% relative risk reduction in grade I to IV acute graft-versus-host disease as compared to 2 g/day dose in reduced-intensity-conditioned double umbilical cord blood transplant recipients-Mycophenolate mofetil 3 g/day dose neither had adverse impact on hematopoietic engraftment nor increased the risk of infectious complications-Mycophenolate mofetil 3 g/day does not impact disease relapse, chronic graft-versus-host disease, or survival after reduced-intensity-conditioned double umbilical cord blood transplantation-This study supports the use of mycophenolate mofetil 3 g/day for graft-versus-host disease prophylaxis in the context of reduced-intensity-conditioned double umbilical cord blood transplantation.
- Graft-versus-host disease
- Mycophenolate mofetil
- Reduced-intensity conditioning
- Umbilical cord blood