admap to precision treatments for familial pulmonary fibrosis.

Hurley K., Ozaki M., Philippot Q., Galvin L., Crosby D., Kirwan M., Gill DR., Alysandratos K-D., Jenkins G., Griese M., Nathan N., Borie R., COST Open-ILD Group Management Committee .

Interstitial lung diseases (ILDs) in adults and children (chILD) are a heterogeneous group of lung disorders leading to inflammation, abnormal tissue repair and scarring of the lung parenchyma often resulting in respiratory failure and death. Inherited factors directly cause, or contribute significantly to the risk of developing ILD, so called familial pulmonary fibrosis (FPF), and monogenic forms may have a poor prognosis and respond poorly to current treatments. Specific, variant-targeted or precision treatments are lacking. Clinical trials of repurposed drugs, anti-fibrotic medications and specific treatments are emerging but for many patients no interventions exist. We convened an expert working group to develop an overarching framework to address the existing research gaps in basic, translational, and clinical research and identified areas for future development of preclinical models, candidate medications and innovative clinical trials. In this Position Paper, we summarise working group discussions, recommendations, and unresolved questions concerning precision treatments for FPF.

DOI

10.1016/j.ebiom.2024.105135

Type

Journal article

Publication Date

2024-06-01T00:00:00+00:00

Volume

104

Keywords

Familial pulmonary fibrosis, Induced pluripotent stem cells, Interstitial lung disease of genetic cause, precision medicine, Preclinical models, Pulmonary fibrosis, Surfactant related gene, Telomere related gene, Telomeropathy, Humans, Precision Medicine, Animals, Disease Management, Pulmonary Fibrosis, Clinical Trials as Topic

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