Considerations for Endpoints in Lung Transplant Clinical Trials: An ISHLT Consensus Statement.

Clinical trials in lung transplantation have been hindered by a lack of clarity on the formulation and significance of endpoints for evaluating therapeutic efficacy. To address this challenge, a multidisciplinary working group from the International Society for Heart and Lung Transplantation developed consensus recommendations on endpoints beyond mortality. These endpoints include primary graft dysfunction (PGD), chronic lung allograft dysfunction (CLAD), acute cellular rejection (ACR), antibody-mediated rejection (AMR), immunosuppression-related complications, patient-reported outcomes (PROs), and pediatric-specific considerations. For each endpoint, a subgroup reviewed measurement best practices, assessed links to clinical benefit, and evaluated the evidence supporting their utility in clinical trial settings. Consensus was established through a Delphi process involving three rounds of voting. This document provides practical guidance for operationalizing these endpoints and outlines their optimal use in clinical trials. By standardizing trial design, these recommendations aim to accelerate the development of urgently needed therapies to improve lung transplantation outcomes.

Duke Scholars

Author Scott Michael Palmer Jr. Medicine, Pulmonary, Allergy, and Critical Care Medicine

Author Laurie D. Snyder Medicine, Pulmonary, Allergy, and Critical Care Medicine

Author Elizabeth N Pavlisko Pathology

Author Megan Lee Neely Biostatistics & Bioinformatics, Division of Biostatistics

Author Jamie Lynn Todd Medicine, Pulmonary, Allergy, and Critical Care Medicine