Charlotte Seijger1, Joost Raaphorst2, Judith Vonk3, Baziel van Engelen4, Harry Heijerman5, Nadine Stigter5, Peter Wijkstra6. 1. Department of Pulmonary Diseases and Home Mechanical Ventilation, University of Groningen, University Medical Centre Groningen, Groningen, The Netherlands, c.g.w.seijger@umcg.nl. 2. Department of Neurology, Amsterdam Neuroscience, University of Amsterdam, Amsterdam UMC, Amsterdam, The Netherlands. 3. Department of Epidemiology, University of Groningen, University Medical Centre Groningen, Groningen, The Netherlands. 4. Department of Neurology, Donders Institute for Brain, Cognition and Behaviour, Radboud University Medical Centre, Nijmegen, The Netherlands. 5. Department of Pulmonary Diseases, Utrecht University, University Medical Centre Utrecht, Utrecht, The Netherlands. 6. Department of Pulmonary Diseases and Home Mechanical Ventilation, University of Groningen, University Medical Centre Groningen, Groningen, The Netherlands.
Abstract
BACKGROUND: Non-invasive home mechanical ventilation (HMV) is a complex treatment in myotonic dystrophy type 1 (DM1) patients, due to a presumed poor adherence, variable symptom improvement, and uncertainty regarding survival benefits. OBJECTIVES: We aimed to investigate indications, adherence to HMV and its effects on mortality in a large cohort of DM1 patients. METHODS: In this retrospective cohort study, we evaluated 224 DM1 patients. Different groups based on hypercapnia and HMV treatment were compared. Cox regression analyses were performed to compare mortality between different defined groups. RESULTS: 224 patients were analysed of whom 111 started non-invasive HMV. Indications were daytime hypercapnia (n = 75), only nocturnal hypercapnia (n = 33), or other reasons (n = 3). Adequate adherence (≥4 h/night) was found in 84.9% of patients. Adequate ventilation was reached in 86.5% of patients. In 33 patients (29.7%), HMV was stopped prematurely due to not reaching patients' expectations on symptom relief or treatment burden (n = 22), or intolerance (n = 8), or other reasons (n = 3). HMV did not improve survival in daytime hypercapnic patients (p = 0.61) nor in nocturnal hypercapnia patients compared to daytime hypercapnia (p = 0.21). Significant survival benefits after starting HMV were found for patients with HMV adherence ≥5 h/24 h compared to patients who used HMV less. CONCLUSION: In this large cohort, daytime hypercapnia is the main reason for starting HMV, which is well tolerated and used. Mortality is not associated with the reason why HMV was started, but once started, patients with ≥5 h/24 h adherence have significantly better survival compared to patients who use it less.
BACKGROUND: Non-invasive home mechanical ventilation (HMV) is a complex treatment in myotonic dystrophy type 1 (DM1) patients, due to a presumed poor adherence, variable symptom improvement, and uncertainty regarding survival benefits. OBJECTIVES: We aimed to investigate indications, adherence to HMV and its effects on mortality in a large cohort of DM1 patients. METHODS: In this retrospective cohort study, we evaluated 224 DM1 patients. Different groups based on hypercapnia and HMV treatment were compared. Cox regression analyses were performed to compare mortality between different defined groups. RESULTS: 224 patients were analysed of whom 111 started non-invasive HMV. Indications were daytime hypercapnia (n = 75), only nocturnal hypercapnia (n = 33), or other reasons (n = 3). Adequate adherence (≥4 h/night) was found in 84.9% of patients. Adequate ventilation was reached in 86.5% of patients. In 33 patients (29.7%), HMV was stopped prematurely due to not reaching patients' expectations on symptom relief or treatment burden (n = 22), or intolerance (n = 8), or other reasons (n = 3). HMV did not improve survival in daytime hypercapnic patients (p = 0.61) nor in nocturnal hypercapniapatients compared to daytime hypercapnia (p = 0.21). Significant survival benefits after starting HMV were found for patients with HMV adherence ≥5 h/24 h compared to patients who used HMV less. CONCLUSION: In this large cohort, daytime hypercapnia is the main reason for starting HMV, which is well tolerated and used. Mortality is not associated with the reason why HMV was started, but once started, patients with ≥5 h/24 h adherence have significantly better survival compared to patients who use it less.
Authors: William J Groh; Miriam R Groh; Changyu Shen; Darren G Monckton; Cynthia L Bodkin; Robert M Pascuzzi Journal: Muscle Nerve Date: 2011-05 Impact factor: 3.217
Authors: Kees Okkersen; Cecilia Jimenez-Moreno; Stephan Wenninger; Ferroudja Daidj; Jeffrey Glennon; Sarah Cumming; Roberta Littleford; Darren G Monckton; Hanns Lochmüller; Michael Catt; Catharina G Faber; Adrian Hapca; Peter T Donnan; Gráinne Gorman; Guillaume Bassez; Benedikt Schoser; Hans Knoop; Shaun Treweek; Baziel G M van Engelen Journal: Lancet Neurol Date: 2018-06-19 Impact factor: 44.182