Post-Transplant Cyclophosphamide, Tacrolimus, and Mycophenolate Mofetil As the New Standard for Graft-Versus-Host Disease (GVHD) Prophylaxis in Reduced Intensity Conditioning: Results from Phase III BMT CTN 1703

作者：Shernan G. Holtan, Mehdi Hamadani, Juan Wu, Monzr M. Al Malki, Lyndsey Runaas, Hany Elmariah, Andrew R. Rezvani, Mahasweta Gooptu, Karilyn T. M. Larkin, Brian C. Shaffer, Najla H El Jurdi, Alison Wakoff Loren, Melhem Solh, Aric C. Hall, Amin Majid Alousi, OMER HASSAN JAMY, Miguel‐Angel Perales, Janny M. Yao, Kristy Applegate, Ami Siddharth Bhatt, Leslie S. Kean, Yvonne Adeduni Efebera, Carrie Lynn Kitko, Ran Reshef, William Clark, Nancy L. DiFronzo, Lori A. Henderson, Richard John Jones, Eric Liefer, Michael J. Martens, Mary M. Horowitz, Javier Bolaños‐Meade · 发表于：Blood · 年份：2022 · DOI：10.1182/blood-2022-171463 · 被引用次数：54 · 研究领域：Hematopoietic Stem Cell Transplantation、Acute Lymphoblastic Leukemia research、Polyomavirus and related diseases

Introduction: Controlling both GVHD and disease relapse is essential for allogeneic hematopoietic cell transplantation (alloHCT) success. For three decades, the standard GVHD prophylaxis strategy has been a calcineurin inhibitor such as tacrolimus (Tac) plus methotrexate (MTX). Although several approaches have been tried in recent years to improve upon Tac/MTX, intensifying immunosuppression frequently comes with an increased risk of serious infections or relapse and usually no effect on chronic GVHD even if acute GVHD is reduced. The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) previously completed a phase II study comparing three novel GVHD prophylaxis regimens to contemporaneous controls receiving Tac/MTX (BMT CTN 1203) in order to determine the most promising novel GVHD prophylaxis approach with reduced intensity conditioning (RIC). The most promising GVHD prophylaxis regimen in BMT CTN 1203 was a 3-drug combination of post-transplant cyclophosphamide (PTCy), tacrolimus, and mycophenolate mofetil (PTCy/Tac/MMF). Here, we report the results of the randomized phase III study comparing outcomes of alloHCT in those randomized to receive PTCy/Tac/MMF versus standard Tac/MTX. Patients and Methods: Eligible adults (age 18+) with hematologic malignancies undergoing RIC alloHCT with an 6/6 matched related (N=128), 8/8 matched unrelated (N=288), or 7/8 single mismatch (N=15) peripheral blood stem cell donor, satisfactory organ function, and adequate performance sta...