Low incidence of acute graft-versus-host disease with short-term tacrolimus in haploidentical hematopoietic stem cell transplantation

Abstract Although tacrolimus (Tac) has immunosuppressive properties and exhibits promising efficacy against graft-versus-host disease (GVHD), little is known about Tac in the prophylaxis of GVHD after HLA-haploidentical hematopoietic stem cell transplantation (haplo-HSCT). In a multicenter randomized controlled trial, 174 patients received haplo-HSCT with GVHD prophylaxis involving short-term Tac (from −8 days to +30 days) or cyclosporine (CsA). The 100 day cumulative incidences of acute GVHD (aGVHD) and grade III–IV aGVHD with the short-term Tac regimen and CsA regimen were 29.1 (19.5–38.7)% vs. 50.0(39.6–60.4)% (p = 0.005) and 3.6(0.0–7.5)% vs. 13.5(6.1–20.9)% (p = 0.027), respectively. There were no significant differences in the incidences of chronic GVHD (cGVHD), relapse and cytomegalovirus infection. Lymphocyte subset analysis showed that T cells decreased to lower levels on the short-term Tac regimen within 3 months of transplantation. The disease-free survival and overall survival on the short-term Tac and CsA regimens were 59.3 (48.9–69.7)% vs. 55.7 (45.3–66.1)% (p = 0.696) and 65.1 (55.1–75.1)% vs. 61.4 (51.2–71.6)% (p = 0.075), respectively. Our findings indicate that the short-term Tac regimen for GVHD prophylaxis in patients undergoing haplo-HSCT is associated with a low incidence and slight severity of aGVHD and did not increase the incidence of relapse and cytomegalovirus infection.