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
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Language:English
Published: Elsevier 2025-07-01
Series:Blood Advances
Online Access:http://www.sciencedirect.com/science/article/pii/S2473952925002599
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author 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
author_facet 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
author_sort Sameem Abedin
collection DOAJ
description 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.
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spelling doaj-art-07f570e017314183955a0f68b9f1b84d2025-07-19T04:38:40ZengElsevierBlood Advances2473-95292025-07-0191434953501Impact of posttransplant cyclophosphamide-based GVHD prophylaxis in patients 70 years and older: an update from BMT CTN 1703Sameem Abedin0Michael J. Martens1Javier Bolaños-Meade2Monzr M. Al Malki3Qinghua Lian4Lyndsey Runaas5Hany Elmariah6Mahasweta Gooptu7Karilyn T. Larkin8Brian C. Shaffer9Alison W. Loren10Melhem Solh11Amin M. Alousi12Omer H. Jamy13Miguel-Angel Perales14Andrew Rezvani15Ami Bhatt16Najla El Jurdi17Janny M. Yao18Kristy Applegate19Leslie S. Kean20Yvonne A. Efebera21Ran Reshef22William Clark23Eric Leifer24Wael Saber25Mary M. Horowitz26Richard J. Jones27Shernan G. Holtan28Mehdi Hamadani29Division of Hematology and Oncology, Department of Medicine, Medical College of Wisconsin, Milwaukee, WI; Correspondence: Sameem Abedin, Division of Hematology and Oncology, Department of Medicine, Medical College of Wisconsin, 9200 W Wisconsin Ave, Milwaukee, WI 53226;Center for Blood and Marrow Transplant Research Division of Biostatistics, Data Science Institute, Milwaukee, WIDepartment of Oncology, Johns Hopkins University School of Medicine, Baltimore, MDDepartment of Hematology and Hematopoietic Cell Transplantation, City of Hope, Duarte, CACenter for Blood and Marrow Transplant Research Division of Biostatistics, Data Science Institute, Milwaukee, WIDivision of Hematology and Oncology, Department of Medicine, Medical College of Wisconsin, Milwaukee, WIDepartment of Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer and Research Institute, Tampa, FLDepartment of Hematology and Oncology, Dana-Farber Cancer Institute, Boston, MADivision of Hematology, The Ohio State University Comprehensive Cancer Center, Columbus, OHAdult Bone Marrow Transplantation Service, Memorial Sloan Kettering Cancer Center, New York, NY; Department of Medicine, Weill Cornell Medical College, New York, NYDivision of Hematology and Oncology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PABlood and Marrow Transplant Program at Northside Hospital, Atlanta, GADepartment of Stem Cell Transplantation and Cellular Therapy, the University of Texas MD Anderson Cancer Center, Houston, TXDivision of Hematology and Oncology, Department of Medicine, University of Alabama at Birmingham, Birmingham, ALAdult Bone Marrow Transplantation Service, Memorial Sloan Kettering Cancer Center, New York, NY; Department of Medicine, Weill Cornell Medical College, New York, NYDivision of Blood and Marrow Transplantation and Cellular Therapy, Department of Medicine, Stanford University School of Medicine, Palo Alto, CADivision of Hematology, Departments of Medicine and Genetics, Stanford University, Palo Alto, CADivision of Hematology, Oncology, Transplant, University of Minnesota, Minneapolis, MNDepartment of Pharmacy, City of Hope National Medical Center, Duarte, CAEmmes, Rockville, MDDepartment of Pediatrics, Harvard Medical School and Division of Pediatric Hematology and Oncology, Boston Children’s Hospital, Boston, MAOhioHealth, Columbus, OHDepartment of Medicine, Blood and Marrow Transplantation Program, Columbia University Irving Medical Center, New York, NYDivision of Hematology-Oncology and Palliative Care, Department of Medicine, Virginia Commonwealth University, Richmond, VAOffice of Biostatistics Research, National Heart, Lung, and Blood Institute, Bethesda, MDDepartment of Medicine, Center for Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WIDepartment of Medicine, Center for Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WIDepartment of Oncology, Johns Hopkins University School of Medicine, Baltimore, MDDepartment of Medicine, Blood and Marrow Transplantation and Cellular Therapy, Roswell Park Comprehensive Cancer Center, Buffalo, NYDepartment of Medicine, Center for Blood and Marrow Transplant Research, Medical College of Wisconsin, Milwaukee, WIAbstract: 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.http://www.sciencedirect.com/science/article/pii/S2473952925002599
spellingShingle 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
Impact of posttransplant cyclophosphamide-based GVHD prophylaxis in patients 70 years and older: an update from BMT CTN 1703
Blood Advances
title Impact of posttransplant cyclophosphamide-based GVHD prophylaxis in patients 70 years and older: an update from BMT CTN 1703
title_full Impact of posttransplant cyclophosphamide-based GVHD prophylaxis in patients 70 years and older: an update from BMT CTN 1703
title_fullStr Impact of posttransplant cyclophosphamide-based GVHD prophylaxis in patients 70 years and older: an update from BMT CTN 1703
title_full_unstemmed Impact of posttransplant cyclophosphamide-based GVHD prophylaxis in patients 70 years and older: an update from BMT CTN 1703
title_short Impact of posttransplant cyclophosphamide-based GVHD prophylaxis in patients 70 years and older: an update from BMT CTN 1703
title_sort impact of posttransplant cyclophosphamide based gvhd prophylaxis in patients 70 years and older an update from bmt ctn 1703
url http://www.sciencedirect.com/science/article/pii/S2473952925002599
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