Efficacy and safety of danazol for pulmonary fibrosis or bone-marrow failure associated with telomere-related gene mutation.

Date publication

mars 2026

Journal

ERJ open research

Auteurs

Membres identifiés du Cancéropôle Est :
Dr MARCAIS Ambroise

Tous les auteurs :
Sicre de Fontbrune F, Jouneau S, Cottin V, Prevot G, Nunes H, Terriou L, Marchand-Adam S, Crestani B, Marcais A, Bergeron A, Debray MP, Plessier A, Lainey E, Ba I, Kannengiesser C, Desseaux K, Resche-Rigon M, Biard L, Borie R

Lien Pubmed

https://www.ncbi.nlm.nih.gov/pubmed/41953763

Résumé

Telomere-related gene (TRG) pathogenic variants are detected in ∼30% of familial pulmonary fibrosis (PF) cases. Danazol, a synthetic sex hormone with androgenic properties, has been found associated with telomere elongation and haematological response in patients with short telomeres. The objective of the ANDROTELO multicentre, prospective, open-label single-arm phase II clinical trial (NCT03710356) was to evaluate the efficacy and safety of danazol in carriers of TRG mutations with PF or bone-marrow failure (BMF).

Référence

ERJ Open Res. 2026 03;12(2):