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Effect and impact of mechanical ventilation in myotonic dystrophy type 1: a prospective cohort study
  1. Ghilas Boussaïd1,2,3,
  2. Hélène Prigent3,4,
  3. Pascal Laforet5,
  4. Jean-Claude Raphaël6,
  5. Djillali Annane1,3,6,
  6. David Orlikowski1,3,6,
  7. Frédéric Lofaso2,3,4
  1. 1CIC 1429, INSERM, AP-HP, Hôpital Raymond Poincaré, Garches, France
  2. 2Association Française contre les Myopathies–Téléthon, Évry, France
  3. 3Université de Versailles Saint-Quentin-en-Yvelines, INSERM U1179, Versailles, France
  4. 4Service d’Explorations Fonctionnelles Respiratoires, AP-HP, Hôpital Raymond Poincaré, Garches, France
  5. 5Institut de Myologie, Centre de Référence de Pathologie Neuromusculaire Paris-Est, GH Pitié-Salpêtrière, Paris, France
  6. 6Pôle de ventilation à domicile, AP-HP, Hôpital Raymond Poincaré, Garches, France
  1. Correspondence to Ghilas Boussaïd, Centre Investigation Clinique 1429, CHU–Hôpital Raymond Poincaré, 104 Boulevard Raymond Poincaré, 92380 Garches, France; boussaid.gh{at}gmail.com

Abstract

Few studies have assessed the impact of home ventilation in patients with myotonic dystrophy type 1 (DM1) and no specific recommendations are available. We assessed the survival associated with category of home ventilation adherence of patients with DM1 followed up at a home ventilation unit using a Cox proportional hazards model. 218 patients were included; those who refused or delayed their acceptance of non-invasive ventilation were at higher risk for severe events (invasive ventilation or death) (P=0.03). Risk of death was associated with orthopnoea (HR 2.37; 95% CI 1.17 to 4.80; P<0.02) and adherence category (100 to 90% vs >75%: HR 3.26; 95% CI 1.32 to 8.04; P<0.03). Failure to use home ventilation as prescribed may be associated with increased mortality in patients with DM1.

  • respiratory muscles
  • assisted ventilation
  • non-invasive ventilation
  • clinical epidemiology

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Footnotes

  • Contributors GB, FL and DO designed the study. GB, FL, DO, PL, HP, JCR and DA followed the patients and collected the data. GB and FL analysed the data, wrote the manuscript and are the guarantors of the paper. All authors critically revised the manuscript, read the final version and approved its submission for publication.

  • Funding This research received no specific grant from any funding agency in the public, commercial or not-for-profit sectors.

  • Competing interests None declared.

  • Patient consent Obtained.

  • Ethics approval CNIL CCTIRS (No. yK90609496a).

  • Provenance and peer review Not commissioned; externally peer reviewed.

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