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Observational Study
. 2021;100(2):154-163.
doi: 10.1159/000511962. Epub 2021 Jan 18.

New Insights in Adherence and Survival in Myotonic Dystrophy Patients Using Home Mechanical Ventilation

Charlotte Seijger  1 , Joost Raaphorst  2 , Judith Vonk  3 , Baziel van Engelen  4 , Harry Heijerman  5 , Nadine Stigter  5 , Peter Wijkstra  6
Affiliations
Observational Study

New Insights in Adherence and Survival in Myotonic Dystrophy Patients Using Home Mechanical Ventilation

Charlotte Seijger et al. Respiration. 2021.

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.

Keywords: Adherence; Home mechanical ventilation; Myotonic dystrophy; Respiratory failure; Survival.

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Conflict of interest statement

C.S., J.R., J.V., H.H., and N.S. have no conflicts of interest to declare. BvE reports grants from FP7 European Union grant OPTIMISTIC, grants from Marigold Canada, all outside the submitted work. Dr. Wijkstra reports grants and personal fees from Philips; grants, personal fees, and others from RESMED; grants from VIVISOL, grants from Air Liquide, grants from Goedegebuure, personal fees from Bresotec, and personal fees from Synapse, all outside the submitted work.

Figures

Fig. 1
Fig. 1
Overview of survival time. HMV, home mechanical ventilation.
Fig. 2
Fig. 2
Flow diagram of 224 DM1 patients stratified for pCO2 and the initiation of HMV. DM1, myotonic dystrophy type 1; HMV, home mechanical ventilation.
Fig. 3
Fig. 3
a Kaplan-Meier curve showing HMV indication and its effect on mortality. b Kaplan-Meier curve showing HMV adherence and its effect on mortality. HMV, home mechanical ventilation.
See this image and copyright information in PMC

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