Evidence map›Paper›PMID 41953763›Full record

ArticleERJ open research2026

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

Flore Sicre de Fontbrune, Stéphane Jouneau, Vincent Cottin, Grégoire Prevot, Hilario Nunes, Louis Terriou, Sylvain Marchand-Adam, Bruno Crestani, Ambroise Marcais, Anne Bergeron and 9 more

Registry-linked trialAbstract read
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In one paragraph

Article in ERJ open research, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03710356 (Essai Bayésien de Phase I/II évaluant l'efficacité et la tolérance du Danazol Chez Les Patients Ayant Une Atteinte hématologique ou Pulmonaire sévère liée à Une téloméropathie - ANDROTELO), which is not on this map. Not yet cited in PubMed.

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0citing papers in PubMed
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1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03710356 phase1 / phase2unknown statusnot on this map

Essai Bayésien de Phase I/II évaluant l'efficacité et la tolérance du Danazol Chez Les Patients Ayant Une Atteinte hématologique ou Pulmonaire sévère liée à Une téloméropathie - ANDROTELO

TypeinterventionalSponsorAssistance Publique - Hôpitaux de ParisRan2018 to 2022Enrolled40ConditionsTelomere Shortening, Telomere Length, Mean LeukocyteArmsDanazol 200 MG
3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

19 authors.

Flore Sicre de FontbruneService d'Hématologie Greffe, Hôpital St Louis, APHP, Centre de Référence des Aplasies Médullaires Acquises et Constitutionnelles (CeRAMIC), Paris, France.
Stéphane JouneauService de Pneumologie, Centre de Référence Constitutif pour les Maladies Rares Pulmonaires, Hôpital Pontchaillou, CHU de Rennes, IRSET UMR 1085, Université de Rennes, Rennes, France.ORCID https://orcid.org/0000-0002-1949-3461
Vincent CottinService de Pneumologie, Centre Coordonnateur National de Référence des Maladies Pulmonaires Rares, Hôpital Louis Pradel, Université Claude Bernard Lyon 1, Université de Lyon, INRAE and ERN-LUNG, Lyon, France.ORCID https://orcid.org/0000-0002-5591-0955
Grégoire PrevotService de Pneumologie, Centre de Compétence des Maladies Pulmonaires Rares, Hôpital Larrey, CHU Toulouse, Toulouse, France.
Hilario NunesService de Pneumologie Centre de Référence des Maladies Pulmonaires Rares (Site Constitutif), Hôpital Avicenne, Bobigny, France.
Louis TerriouService de Médecine Interne, CHRU de Lille, Lille, France.
Sylvain Marchand-AdamService de Pneumologie, Centre de Compétence des Maladies Pulmonaires Rares, CHRU Tours, Tours, France.
Bruno CrestaniService de Pneumologie Allergologie et Transplantation, Université Paris Cité, UMR Inserm 1149, CRI, Paris, France.ORCID https://orcid.org/0000-0002-2961-3455
Ambroise MarcaisService d'Hématologie, Hôpital Necker-Enfants Malades, APHP, Paris, France.
Anne BergeronService de Pneumologie, Hôpitaux Universitaires de Genève, Université de Genève, Geneva, Switzerland.
Marie-Pierre DebrayService de Radiologie, Hôpital Bichat, APHP, Paris, France.ORCID https://orcid.org/0000-0003-2109-5662
Aurélie PlessierAPHP DMU DIGEST, Centre de Référence des Maladies Vasculaires du Foie, Inserm U1149, Centre de Recherche sur l'Inflammation (CRI), Université de Paris, ERN Rare Liver Clichy, Paris, France.
Elodie LaineyLaboratoire d'Hématologie, Hôpital Robert Debré, Paris, France.
Ibrahima BaLaboratoire de Génétique, APHP, Hôpital Bichat, Inserm, PHERE, Université Paris Cité, and Université Paris Cité, Inserm, PHERE, Université Paris Cité, Paris, France.
Caroline KannengiesserLaboratoire de Génétique, APHP, Hôpital Bichat, Inserm, PHERE, Université Paris Cité, and Université Paris Cité, Inserm, PHERE, Université Paris Cité, Paris, France.ORCID https://orcid.org/0000-0003-2155-9383
Kristell DesseauxService de Biostatistiques et Informatique Médicale, Hôpital St Louis, APHP, Université Paris Cité, INSERM IRSL U1342 Equipe ECSTRRA, Paris, France.
Matthieu Resche-RigonService de Biostatistiques et Informatique Médicale, Hôpital St Louis, APHP, Université Paris Cité, INSERM IRSL U1342 Equipe ECSTRRA, Paris, France.
Lucie BiardService de Biostatistiques et Informatique Médicale, Hôpital St Louis, APHP, Université Paris Cité, INSERM IRSL U1342 Equipe ECSTRRA, Paris, France.
Raphael BorieService de Pneumologie Allergologie et Transplantation, Université Paris Cité, UMR Inserm 1149, CRI, Paris, France.ORCID https://orcid.org/0000-0002-9906-0024

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: 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). Methods: Included patients were carriers of a pathogenic or likely pathogenic TRG variant and presented a PF lung involving ≥10% parenchymal involvement on chest computed tomography (PF group) and/or severe BMF (BMF group). Treatment was danazol 400 mg twice a day for 12 months. We included 25 patients with PF (16 males) and five with BMF (two males). One PF patient withdrew consent. Results: At inclusion, the median age in the PF group was 62.5 years, forced vital capacity (FVC) was 69% (interquartile range 40-120) and diffusing capacity of the lung for carbon monoxide ( Conclusion: Danazol was poorly tolerated in patients with TRG-related PF in this study, thus precluding efficacy assessment.

Identifiers

PMID41953763
PMCPMC13054709

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