Pirfenidone for the treatment of bronchiolitis obliterans syndrome related to chronic graft-versus-host disease.

ABSTRACT:

Abstract

Bronchiolitis obliterans syndrome (BOS) is a severe form of chronic graft-versus-host disease (cGVHD) following allogeneic hematopoietic cell transplant (HCT), with a 5-year survival rate of 40%. Currently, there is no curative therapy for BOS. Preclinical data suggest that pirfenidone, an antifibrotic drug, may benefit small airway fibrosis in HCT-associated BOS. A single-arm, open-label, 56-week phase 1 trial with 56-month extension evaluated pirfenidone's tolerability, safety, and efficacy in patients with BOS. Efficacy was measured using pulmonary function tests (PFTs), quantitative computed tomography (qCT) scans, patient-reported outcomes (PROs), cGVHD indices, and laboratory tests. Lung function trajectory was assessed by change in regression slopes before and durin

SUBMITTER: Sharifi H 

PROVIDER: S-EPMC12516044 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature