Impact of posttransplant cyclophosphamide-based GVHD prophylaxis in patients 70 years and older: An update from BMT CTN 1703 Journal Article


Authors: Abedin, S.; Martens, M. J.; Bolaños-Meade, J.; Al Malki, M. M.; Lian, Q.; Runaas, L.; Elmariah, H.; Gooptu, M.; Larkin, K. T.; Shaffer, B. C.; Loren, A. W.; Solh, M.; Alousi, A. M.; Jamy, O. H.; Perales, M. A.; Rezvani, A.; Bhatt, A.; El Jurdi, N.; Yao, J. M.; Applegate, K.; Kean, L. S.; Efebera, Y. A.; Reshef, R.; Clark, W.; Leifer, E.; Saber, W.; Horowitz, M. M.; Jones, R. J.; Holtan, S. G.; Hamadani, M.
Article Title: Impact of posttransplant cyclophosphamide-based GVHD prophylaxis in patients 70 years and older: An update from BMT CTN 1703
Abstract: Allogeneic hematopoietic cell transplant (allo-HCT) is underutilized in adults aged ≥70 years. Morbidity, often driven by graft-versus-host disease (GVHD), is considered a major barrier to its use. The BMT CTN 1703 trial (ClinicalTrials.gov identifier: NCT03959241) randomly assigned adults with hematologic malignancies undergoing allo-HCT after reduced intensity conditioning to receive either posttransplant cyclophosphamide, mycophenolate mofetil, and tacrolimus (PTCy) or tacrolimus and methotrexate (Tac/MTX) for GVHD prophylaxis. Overall study results revealed superior GVHD-free, relapse-free survival (GRFS) with PTCy-based prophylaxis. This analysis explored the impact of PTCy in patients aged ≥70 years enrolled in BMT CTN 1703. We analyzed outcomes for 96 patients aged ≥70 years. PTCy maintained superiority for the primary end point with a GRFS rate of 67.1% compared with 29.5% with Tac/MTX (P = .001). GVHD control and improved immunosuppression-free survival contributed to a lower 1-year nonrelapse mortality (NRM) with PTCy. Furthermore, lower rates of relapse/progression were observed with PTCy, altogether resulting in significantly improved adjusted 1-year survival with PTCy at 94.3% vs 60.2% with Tac/MTX (P = .001). PTCy-based GVHD prophylaxis should be considered standard prophylaxis for older adults. Given low rates of NRM and excellent survival outcomes with this approach, there should be greater consideration for allo-HCT in older patients, particularly patients aged ≥70 years. © 2025 American Society of Hematology. Published by Elsevier Inc. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.
Keywords: adult; controlled study; aged; survival analysis; major clinical study; overall survival; busulfan; fludarabine; cytarabine; methotrexate; outcome assessment; follow up; progression free survival; quality of life; infection; etoposide; bleeding; randomized controlled trial; incidence; relapse; cytogenetics; cyclophosphamide; melphalan; hematopoietic stem cell transplantation; acute lymphoblastic leukemia; acute graft versus host disease; engraftment; myelodysplastic syndrome; whole body radiation; karnofsky performance status; prophylaxis; allogeneic hematopoietic stem cell transplantation; immunosuppressive treatment; tacrolimus; gastrointestinal disease; disease exacerbation; multiple organ failure; platelet count; hematocrit; recurrence free survival; clinical outcome; septic shock; acute myeloid leukemia; mycophenolate mofetil; human; male; female; article; comorbidity assessment; physical functioning; mortality risk; transplant complication
Journal Title: Blood Advances
Volume: 9
Issue: 14
ISSN: 2473-9529
Publisher: American Society of Hematology  
Date Published: 2025-07-22
Start Page: 3495
End Page: 3501
Language: English
DOI: 10.1182/bloodadvances.2025015964
PUBMED: 40305657
PROVIDER: scopus
PMCID: PMC12274666
DOI/URL:
Notes: Article -- Source: Scopus
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  1. Miguel-Angel Perales
    940 Perales
  2. Brian Carl Shaffer
    174 Shaffer