Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis After Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation Journal Article


Authors: Al Malki, M. M.; Bo-Subait, S.; Logan, B.; Olson, J.; Kou, J.; Smith, S.; Leckrone, E.; Wu, J.; Stefanski, H. E.; Auletta, J. J.; Spellman, S. R.; Malmberg, C.; Askar, M.; Cusatis, R.; Shaffer, B. C.; Modi, D.; Khimani, F.; Gooptu, M.; Hamadani, M.; Madbouly, A.; Maiers, M.; Fingerson, S.; Cook, R.; Ballen, K.; Loren, A.; Larkin, K.; Arai, S.; Qayed, M.; Choi, S. W.; Broglie, L.; Shaw, B. E.; Devine, S. M.; Jimenez Jimenez, A. M.
Article Title: Post-Transplant Cyclophosphamide-Based Graft-Versus-Host Disease Prophylaxis After Mismatched Unrelated Donor Peripheral Blood Stem Cell Transplantation
Abstract: PURPOSEAllogeneic hematopoietic stem cell transplantation (HSCT) is a curative treatment for advanced hematologic malignancies. HSCT using human leukocyte antigen (HLA)-mismatched donors is historically associated with inferior survival. Patients from underrepresented racial and ethnic groups more frequently rely on HLA-mismatched donors. We hypothesized that post-transplant cyclophosphamide (PTCy) based graft versus host disease (GVHD) prophylaxis would improve outcomes for HSCT recipients using peripheral blood stem cells (PBSCs) from HLA-mismatched unrelated donors (MMUDs) by reducing the risk of GVHD.METHODSThis phase II, nonrandomized, multicenter trial assessed PBSCs in the setting of a GVHD prophylaxis regimen of cyclophosphamide, tacrolimus, and mycophenolate mofetil in two adult strata: myeloablative conditioning (MAC) and reduced-intensity or nonmyeloablative (RIC/NMA) conditioning before HSCT from a MMUD. The primary objective was to estimate 1 year overall survival (OS) for each stratum. Key secondary end points included incidences of acute and chronic GVHD.RESULTSA total of 145 patients enrolled, with 59% self-identifying within an underrepresented group. The 1 year OS was 83.8% (95% CI, 73.1% to 90.4%) for MAC and 78.6% (95% CI, 67% to 86.5%) for RIC/NMA. Incidences of grades III to IV acute GVHD at 6 months were 8% (95% CI, 3.2 to 15.6) for MAC and 10% (95% CI, 4.4 to 18.4) for RIC/NMA. Moderate/severe chronic GVHD at 1 year was 10.3% (95% CI, 4.4 to 18.9) for MAC and 8.6% (95% CI, 3.5 to 16.6) for RIC/NMA. 32% of patients whose donors matched at fewer than seven of eight HLA alleles had similar OS compared with those with donor matched at seven of eight alleles.CONCLUSIONPTCy-based GVHD prophylaxis after MMUD HSCT with PBSC grafts results in favorable 1 year OS. Using MMUDs expands donor availability to all patients regardless of ancestry (ACCESS; ClinicalTrials.gov identifier: NCT04904588). © 2025 American Society of Clinical Oncology.
Journal Title: Journal of Clinical Oncology
ISSN: 0732-183X
Publisher: American Society of Clinical Oncology  
Date Published: 2025-01-01
Start Page: JCO
End Page: 25
Language: English
DOI: 10.1200/jco-25-00856
PROVIDER: scopus
DOI/URL:
Notes: Article -- Source: Scopus
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