Abstract
In this retrospective study of a randomised trial of simtuzumab in idiopathic pulmonary fibrosis (IPF), prodromal decline in forced vital capacity (FVC) was significantly associated with increased risk of mortality, respiratory and all-cause hospitalisations, and categorical disease progression. Predictive modelling of progression-free survival event risk was used to assess the effect of population enrichment for patients at risk of rapid progression of IPF; C-index values were 0.64 (death), 0.69 (disease progression), and 0.72 (adjudicated respiratory hospitalisation) and 0.76 (all-cause hospitalisation). Predictive modelling may be a useful tool for improving efficiency of clinical trials with categorical end points.
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Raghu, G., Ley, B., Brown, K. K., Cottin, V., Gibson, K. F., Kaner, R. J., … Martinez, F. J. (2020). Risk factors for disease progression in idiopathic pulmonary fibrosis. Thorax, 75(1), 78–80. https://doi.org/10.1136/thoraxjnl-2019-213620
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