Post-Transplant Cyclophosphamide–Based Graft-Versus-Host Disease Prophylaxis Attenuates Disparity in Outcomes Between Use of Matched or Mismatched Unrelated Donors

Author:

Shaffer Brian C.1ORCID,Gooptu Mahasweta2,DeFor Todd E.3,Maiers Martin3ORCID,Bolaños-Meade Javier4,Abboud Ramzi5ORCID,Briggs Adrienne D.6,Khimani Farhad7ORCID,Modi Dipenkumar8ORCID,Newcomb Richard9ORCID,Shpall Elizabeth J.10,Bupp Caitrin3ORCID,Spellman Stephen R.3,Stefanski Heather E.3,Shaw Bronwen E.11ORCID,Auletta Jeffery J.312ORCID,Devine Steven M.3ORCID,Jimenez Jimenez Antonio M.13ORCID,Al Malki Monzr M.14ORCID

Affiliation:

1. Adult Bone Marrow Transplantation Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, NY

2. Department of Hematology/Oncology, Dana-Farber Cancer Institute, Boston, MA

3. CIBMTR (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, MN

4. The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, MD

5. Washington University School of Medicine, St Louis, MO

6. Cancer Transplant Institute at HonorHealth, Scottsdale, AZ

7. Department of Blood and Marrow Transplantation and Cellular Immunotherapy, Moffitt Cancer Center, Tampa, FL

8. Barbara Ann Karmanos Cancer Institute, Wayne State University, Detroit, MI

9. Massachusetts General Hospital, Boston, MA

10. Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX

11. CIBMTR (Center for International Blood and Marrow Transplant Research), Department of Medicine, Medical College of Wisconsin, Milwaukee, WI

12. Hematology/Oncology/BMT and Infectious Diseases, Nationwide Children's Hospital, Columbus, OH

13. Sylvester Comprehensive Cancer Center, Miller School of Medicine, Miami, FL

14. City of Hope National Medical Center, Duarte, CA

Abstract

PURPOSE Access to allogeneic hematopoietic cell transplantation (HCT) remains limited among persons of non-European ancestry if human leukocyte antigen (HLA) matching is required. We evaluated whether post-transplant cyclophosphamide (PTCy)–based graft-versus-host disease (GVHD) prophylaxis improved HCT outcomes with HLA-matched unrelated donor (MUD) and mismatched unrelated donor (MMUD) HCT when compared with calcineurin inhibitor (CNI)–based prophylaxis. METHODS Three-year overall survival (OS) and GVHD-free, relapse-free survival (GRFS) were compared between adult recipients undergoing initial MUD or single HLA locus MMUD HCT with either PTCy- or CNI-based prophylaxis who were reported to the Center for International Blood and Marrow Transplant Research between 2017 and 2021. RESULTS Included were 10,025 HCT recipients (7,272 recipients of MUD with CNI, 1,681 MUD with PTCy, 613 MMUD with CNI, and 459 MMUD with PTCy) who underwent HCT for acute leukemia (70.9%) or myelodysplastic syndromes (29.2%). Median patient age was 60.7 years (range, 18.0-82.7) and median follow-up was 36.6 (range, 3.0-77.8) months. When compared with MUD HCT with PTCy, MMUD HCT with PTCy had similar OS (hazard ratio [HR], 0.96 [95% CI, 0.823 to 1.11]; P = .60) and GRFS (HR, 0.90 [0.79 to 1.02]; P = .1). When compared with MUD HCT with CNI, OS was improved after MUD HCT with PTCy (HR, 0.88 [0.80 to 0.96]; P = .004) and GRFS was improved with PTCy after either MUD (HR, 0.61 [0.57 to 0.66]; P < .0001) or MMUD (HR, 0.68 [0.60 to 0.76]; P < .0001) HCT. Benefit from PTCy was independent of patient ancestry. Global registry level analysis demonstrated that inclusion of MMUD increased donor availability regardless of recipient ancestry. CONCLUSION Use of PTCy results in comparable OS and GRFS using either MUD or MMUD HCT, expanding access to HCT for patients from all racial and ethnic ancestry groups.

Publisher

American Society of Clinical Oncology (ASCO)

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