Improved Patient-Reported Outcomes With Post-Transplant Cyclophosphamide: A Quality-of-Life Evaluation and 2-Year Outcomes of BMT CTN 1703
Abstract
The BMT CTN 1703 phase III trial confirmed that graft-versus-host disease (GVHD) prophylaxis with post-transplantation cyclophosphamide (PTCy), tacrolimus (Tac), and mycophenolate mofetil (MMF) results in superior GVHD-free, relapse-free survival (GRFS) compared with Tac/methotrexate (MTX) prophylaxis. This companion study assesses the effect of these regimens on patient-reported outcomes (PROs). Using the Lee Chronic GVHD Symptom Score and PROMIS subscales (physical function, GI symptoms, social role satisfaction) as primary end points and hemorrhagic cystitis symptoms and Lee subscales as secondary end points, responses from English and Spanish speakers were analyzed at baseline and days 100, 180, and 365 after transplant. PRO scores were compared between the arms using inverse probability weighted-independent estimating equation models. The PTCy arm had significantly lower scores on the Lee Chronic GVHD Symptom Scale ( P = .01), indicating lower GVHD symptom burden. Lee Scale nutrition and mouth subscores were also better in the PTCy arm compared with the Tac/MTX arm ( P < .01 for both). Older participants (age >65 years) reported better Lee Scale psychological subscores than younger participants ( P = .003). No significant differences were identified in hemorrhagic cystitis or in the PROMIS subscales between treatment arms. The updated clinical end points at 2 years for the parent trial confirmed that PTCy/Tac/MMF maintained a significant advantage over Tac/MTX in GRFS (42.4% v 28.8%, P = .001). In addition to improved GRFS, patients randomly assigned to the PTCy arm reported lower symptom burden during the first year after transplant.
Article Details
Journal Info
Journal of Clinical Oncology
Lippincott Williams & Wilkins
Authors (32)
Shernan G. Holtan
Roswell Park Comprehensive Cancer Center, Buffalo, NY
Javier Bolanos-Meade
1Johns Hopkins University School of Medicine, Oncology, Baltimore, United States
Monzr M. Al Malki
1City of Hope, Duarte, United States
Juan Wu
Shanghai Public Health Clinical Center and Shanghai Institute of Infectious Diseases and Biosecurity, Fudan University
Carrie L. Kitko
23Pediatric Stem Cell Transplant Program, Vanderbilt University Medical Center, Nashville, TN
Ran Reshef
13Division of Hematology/Oncology, Blood and Marrow Transplantation and Cell Therapy Program, Columbia University Irving Medical Center, New York, NY
Andrew R. Rezvani
Stanford University Cancer Institute, Palo Alto, CA
Brian C. Shaffer
1Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY
Melhem M. Solh
26The Blood and Marrow Transplant Program at Northside Hospital, Atlanta, GA
Janny M. Yao
Department of Pharmacy, City of Hope National Medical Center, Duarte, CA
Lyndsey Runaas
13Medical College of Wisconsin, Milwaukee, United States
Hany Elmariah
Stanford University, Stanford, California, United States
Karilyn T. Larkin
Division of Hematology, Department of Medicine, The Ohio State University Wexner Medical Center, Columbus, OH
Najla El Jurdi
3CIBMTR® (Center for International Blood and Marrow Transplant Research), Medical College of Wisconsin, Milwaukee, United States
Mahasweta Gooptu
1Dana Farber Cancer Institute, Boston, United States
Alison W. Loren
Perelman School of Medicine of the University of Pennsylvania, Philadelphia, PA
Aric C. Hall
Division of Hematology/Oncology, Department of Medicine, University of Wisconsin School of Medicine and Public Health, Madison, WI
Amin M. Alousi
Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX
Omer Jamy
15Division of Hematology and Oncology, The University of Alabama at Birmingham, Birmingham, AL
William Clark
Leslie Kean
1Boston Children's Hospital/Dana-Farber Cancer Institute, Harvard Medical Schol, Boston, United States
Ami S. Bhatt
Miguel-Angel Perales
1Adult Bone Marrow Transplant Service, Memorial Sloan Kettering Cancer Center, New York, NY
Kristy Applegate
4The Emmes Company, Rockland, United States
Yvonne Adeduni Efebera
Division of Blood and Marrow Transplant and Cellular Therapy, OhioHealth, Columbus, OH
Eric Leifer
Richard J. Jones
Department of Oncology, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University School of Medicine
Mary M. Horowitz
2Division of Hematology and Oncology, Department of Medicine, Center for International Blood and Marrow Transplant Research/Medical College of Wisconsin, Milwaukee, WI
Deborah Mattila
2CIBMTR® (Center for International Blood and Marrow Transplant Research), NMDP, Minneapolis, United States
Wael Saber
3CIBMTR® (Center for International Blood and Marrow Transplant Research), Medical College of Wisconsin, Milwaukee, United States
Mehdi Hamadani
12Blood and Marrow Transplant and Cellular Therapy Program, Medical College of Wisconsin, Milwaukee, WI
Michael J. Martens
Center for International Blood and Marrow Transplant Research, Milwaukee, WI