Review Article

Post-Transplant Cyclophosphamide for the Prevention of Graft-vs.-Host Disease in Allogeneic Hematopoietic Cell Transplantation: A Guide to Management for the Advanced Practitioner

Ekaterina Kachur, PharmD, BCOP, Jai N. Patel, PharmD, BCOP, CPP, Allison L. Morse, PharmD, BCOP, Donald C. Moore, PharmD, BCPS, BCOP, DPLA, FCCP, and Justin R. Arnall, PharmD, BCOP

From Levine Cancer Institute, Atrium Health, Charlotte, North Carolina

Authors’ disclosures of conflicts of interest are found at the end of this article.

Correspondence to: Donald C. Moore, PharmD, BCPS, BCOP, DPLA, FCCP, 1021 Morehead Medical Drive, Charlotte, NC 28204 E-mail: donald.moore1@atriumhealth.org


J Adv Pract Oncol 2023;14(6):520–532 | https://doi.org/10.6004/jadpro.2023.14.6.5 | © 2023 BroadcastMed LLC


  

ABSTRACT

Cyclophosphamide remains a critical component to haploidentical transplant conditioning regimens. Post-transplant cyclophosphamide (PTCy) emerged as an effective component of graft-vs.-host disease (GVHD) prophylaxis in the nonmyeloablative haploidentical bone marrow transplant setting. The relative ease of administration compared with ex vivo manipulations and efficacy in reducing GVHD has led to increasing PTCy use in transplant centers around the world. The role of PTCy has expanded to haploidentical transplantation with myeloablative conditioning regimens and peripheral blood progenitor cells as the donor source. Moreover, encouraging results in GVHD management have been shown with the use of PTCy alone or in combination with other immunosuppressives in the human leukocyte antigen–matched donor setting. The toxicity profile of cyclophosphamide varies extensively depending on dose, duration, overall drug exposure, and, potentially, pharmacogenetics. This review highlights the pharmacology, pharmacokinetics, and toxic effects of cyclophosphamide and offers practical guidance for clinical application in the post-transplant setting. We summarize data on the management of high-dose cyclophosphamide toxicities and provide insights into the pharmacogenetic implications on drug efficacy and safety data. 




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