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

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...

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Main Authors: Sameem Abedin, Michael J. Martens, Javier Bolaños-Meade, Monzr M. Al Malki, Qinghua Lian, Lyndsey Runaas, Hany Elmariah, Mahasweta Gooptu, Karilyn T. Larkin, Brian C. Shaffer, Alison W. Loren, Melhem Solh, Amin M. Alousi, Omer H. Jamy, Miguel-Angel Perales, Andrew Rezvani, Ami Bhatt, Najla El Jurdi, Janny M. Yao, Kristy Applegate, Leslie S. Kean, Yvonne A. Efebera, Ran Reshef, William Clark, Eric Leifer, Wael Saber, Mary M. Horowitz, Richard J. Jones, Shernan G. Holtan, Mehdi Hamadani
Format: Article
Language:English
Published: Elsevier 2025-07-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S2473952925002599
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Summary: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. This trial was registered at www.ClinicalTrials.gov as #NCT03959241.
ISSN:2473-9529

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