Post-transplantation cyclophosphamide versus conventional graft-versus-host disease prophylaxis in mismatched unrelated donor haematopoietic cell transplantation

Abstract

Post-transplantation cyclophosphamide (PTCy) is an effective strategy to prevent graft-versus-host disease (GVHD) after haploidentical haematopoietic cell transplantation (HCT). We determined the efficacy of PTCy-based GVHD prophylaxis in human leucocyte antigen (HLA)-mismatched unrelated donor (MMUD) HCT. We analysed 113 adult patients with high-risk haematological malignancies who underwent one-antigen MMUD transplantation between 2009 and 2013. Of these, 41 patients received PTCy, tacrolimus and mycophenolate mofetil (MMF) for GVHD prophylaxis; 72 patients received conventional prophylaxis with anti-thymocyte globulin, tacrolimus and methotrexate. Graft source was primarily bone marrow (83% PTCy vs. 63% conventional group). Incidence of grade II-IV (37% vs. 36%, P = 0·8) and grade III-IV (17% vs. 12%, P = 0·5) acute GVHD was similar at day 100. However, the incidence of grade II-IV acute GVHD by day 30 was significantly lower in the PTCy group (0% vs. 15%, P = 0·01). Median time to neutrophil (18 days vs. 12 days, P < 0·001) and platelet (25·5 days vs. 18 days, P = 0·05) engraftment was prolonged in PTCy group. Rates of graft failure, chronic GVHD, 2-year non-relapse mortality, relapse, progression-free survival or overall survival were similar. Our results demonstrate that PTCy, tacrolimus and MMF for GVHD prophylaxis is safe and produced similar results as conventional prophylaxis in patients with one antigen HLA-MMUD HCT.

Keywords: GVHD; HLA-mismatched transplantation; MMUD; post transplantation cyclophosphamide; unrelated donor.

Conflict of interest statement

Disclosure of conflicts of interest

The authors do not have any conflicts of interest.

© 2016 John Wiley & Sons Ltd.

Figures

Fig 1

Cumulative incidence of grade II–IV and III–IV acute graft-versus-host disease (aGVHD) by day 100 in the post-transplant cyclophosphamide (PTCy) group (solid line) compared with the conventional group (dotted line), all patients.

Fig 2

(A) Cumulative incidence of non-relapse mortality (NRM), (B) progression-free survival and (C) overall survival in the post-transplant cyclophosphamide (PTCy) group compared with the conventional group, all patients.