GvHD prophylaxis after single-unit reduced intensity conditioning cord blood transplantation in adults with acute leukemia

S Terakura, Y Kuwatsuka, S Yamasaki, A Wake, J Kanda, Y Inamoto, S Mizuta, T Yamaguchi, N Uchida, Y Kouzai, N Aotsuka, H Ogawa, H Kanamori, K Nishiwaki, S Miyakoshi, M Onizuka, I Amano, T Fukuda, T Ichinohe, Y Atsuta, M Murata, T Teshima

To investigate better GVHD prophylaxis in reduced intensity conditioning umbilical cord blood transplantation (RIC-UCBT), we compared transplant outcomes after UCBT among GvHD prophylaxes using the registry data. We selected patients transplanted for AML or ALL with a calcineurin inhibitor and methotrexate (MTX)/mycophenolate mofetil (MMF) combination. A total of 748 first RIC-UCBT between 2000 and 2012 (MTX+ group, 446, MMF+ group, 302) were included. The cumulative incidence of neutrophil and platelet counts higher than 50 000/μL was significantly better in the MMF+ group (relative risk (RR), 1.55; P<0.001: RR, 1.34; P=0.003, respectively). In multivariate analyses, the risk of grade II-IV and III-IV acute GvHD was significantly higher in the MMF+ group than in the MTX+ group (RR, 1.75; P<0.001: RR, 1.97; P=0.004, respectively). In disease-specific analyses of AML, the risk of relapse of high-risk disease was significantly lower in the MMF+ group (RR, 0.69; P=0.009), whereas no significant difference was observed in the risk of relapse-free and overall survival in high-risk disease. In patients with standard-risk disease, no significant differences were noted in the risk of relapse or survival between the MTX+ and MMF+ groups. Collectively, these results suggest that MMF-containing prophylaxis may be preferable in RIC-UCBT, particularly for high-risk disease.Bone Marrow Transplantation advance online publication, 12 June 2017; doi:10.1038/bmt.2017.116.