Improved Patient-Reported Outcomes With Post-Transplant Cyclophosphamide: A Quality-of-Life Evaluation and 2-Year Outcomes of BMT CTN 1703

Authors: Shernan G. Holtan, Javier BolaƱos-Meade, Monzr M. Al Malki, Juan Wu, Carrie L. Kitko, Ran Reshef, Andrew R. Rezvani, Brian C. Shaffer, Melhem M. Solh, Janny M. Yao, Lyndsey Runaas, Hany Elmariah, Karilyn T. Larkin, Najla El Jurdi, Mahasweta Gooptu, Alison W. Loren, Aric C. Hall, Amin M. Alousi, Omer Jamy, William Clark, Leslie Kean, Ami S. Bhatt, Miguel-Angel Perales, Kristy Applegate, Yvonne Adeduni Efebera, Eric Leifer, Richard J. Jones, Mary M. Horowitz, Deborah Mattila, Wael Saber, Mehdi Hamadani, Michael J. Martens

Published: 2025-01-03

DOI: 10.1200/jco.24.00921

Source: Full article


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 (