A composite endpoint for systemic sclerosis-associated interstitial lung disease: association with mortality in two clinical trial cohorts.

ABSTRACT:

Background

The forced vital capacity (FVC) is the most commonly used endpoint in registrational trials for systemic sclerosis-associated interstitial lung disease (SSc-ILD). However, the FVC has known methodological pitfalls, is affected by extra-pulmonary SSc manifestations and may not be clinically meaningful to patients. Combining individual outcomes into a composite endpoint is an attractive alternative for measuring treatment response and augmenting statistical efficiency in SSc-ILD trials.

Methods

We previously developed a composite endpoint for SSc-ILD using data from the Scleroderma Lung Study (SLS) I (comparing cyclophosphamide versus placebo for SSc-ILD), which included physiological (FVC), radiological (quantitative extent of fibrosis in the zone of maximum involv

SUBMITTER: Volkmann ER 

PROVIDER: S-EPMC12661867 | biostudies-literature | 2025 Nov

REPOSITORIES: biostudies-literature