Risk‐adapted GVHD prophylaxis with post‐transplantation cyclophosphamide in adults after related, unrelated, and haploidentical transplantations

• Published in: European journal of haematology Vol. 100; no. 5; pp. 395 - 402
• Main Authors: Moiseev, Ivan S., Pirogova, Olga V., Alyanski, Alexandr L., Babenko, Elena V., Gindina, Tatyana L., Darskaya, Elena I., Slesarchuk, Olga A., Bykova, Tatyana A., Chukhlovin, Alexei B., Pevtcov, Dmitrii E., Bondarenko, Sergey N., Afanasyev, Boris V.
• Format: Journal Article
• Language: English
• Published: England Wiley Subscription Services, Inc 01.05.2018
• Subjects: Bone marrow; bone marrow transplantation; clinical trials; Cyclophosphamide; Graft-versus-host reaction; Mortality; Mycophenolate mofetil; Mycophenolic acid; Prophylaxis; Survival; Tacrolimus; Toxicity; Transplantation; clinical trials; bone marrow transplantation
• ISSN: 0902-4441; 1600-0609
• DOI: 10.1111/ejh.13030

Introduction Although a number of studies were published on the efficacy of post‐transplantation cyclophosphamide (PTCy) for graft‐versus‐host disease (GVHD) prophylaxis, no large studies prospectively evaluated this strategy in related, unrelated, and haploidentical grafts. Methods In this study, GVHD prophylaxis for 57 matched bone marrow (MBM) grafts consisted of single‐agent PTCy, for 88 matched PBSC grafts (MPBSC) consisted of PTCy, tacrolimus, and mycophenolate mofetil (MMF) 30 mg/kg, and for 55 mismatched grafts (MMGs) consisted of PTCy, tacrolimus and MMF 45 mg/kg. Results The study met the primary endpoint to demonstrate equivalent rates of acute GVHD grade II‐IV (11%, 17%,19%, P = .46), III‐IV (7%, 2%, 6%, P = .41), and moderate and severe chronic GVHD (22%, 11%, 15%, P = .23). There was also no differences in non‐relapse mortality (11% vs 15% vs 17%, P = .75), overall survival (63% vs 71% vs 56%, P = .72), event‐free‐survival (51% vs 66% vs 48%, P = .32) for MBM, MPBSC, and MMG groups, respectively. Toxicity was comparable between groups except higher incidence of nephrotoxicity in combination arms (P = .0005) and higher incidence of graft failures in MMG group (P = .004). Conclusion The suggested risk‐adapted PTCy‐based prophylaxis is feasible and is associated with low GVHD incidence and mortality in all types of grafts. The study was registered on clinicaltrials.gov (NCT02294552).