Effect and impact of mechanical ventilation in myotonic dystrophy type 1: a prospective cohort study

• Published in: Thorax Vol. 73; no. 11; pp. 1075 - 1078
• Main Authors: Boussaïd, Ghilas, Prigent, Hélène, Laforet, Pascal, Raphaël, Jean-Claude, Annane, Djillali, Orlikowski, David, Lofaso, Frédéric
• Format: Journal Article
• Language: English
• Published: England BMJ Publishing Group LTD 01.11.2018; BMJ Publishing Group
• Subjects: Adult; Cohort analysis; Ecology, environment; Female; Follow-Up Studies; France - epidemiology; Health; Humans; Life Sciences; Lung diseases; Male; Mortality; Myotonic Dystrophy - complications; Myotonic Dystrophy - mortality; Myotonic Dystrophy - therapy; Neuromuscular diseases; Ostomy; Oxygen therapy; Patient Compliance; Patients; Prognosis; Prospective Studies; Respiration, Artificial - methods; Respiratory Function Tests; Respiratory Insufficiency - etiology; Respiratory Insufficiency - physiopathology; Respiratory Insufficiency - therapy; Studies; Survival analysis; Survival Rate - trends; France; non-invasive ventilation; respiratory muscles; assisted ventilation; clinical epidemiology
• ISSN: 0040-6376; 1468-3296
• DOI: 10.1136/thoraxjnl-2017-210610

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.