Natural History of Cardiac and Respiration Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation

• Conditions: Heart, Respiratory, Disease, Functional

• Registration Number: NCT02501083
• Lead Sponsor: Centre d'Investigation Clinique et Technologique 805

Brief Summary

Muscular dystrophies are inherited disorders that affect skeletal muscle. Cardiac and respiratory function may be affected in this group of diseases. The investigators sought to analyze the long term cardiac and respiratory function in patients with muscular dystrophies recquiring home mechnaical ventilation .

Detailed Description

Muscular dystrophies are neuromuscular disorders with disability and can affect respiratory muscle function and left ventricular function. Medical management of patients with cardiomyopathy relies mainly on angiotensin-converting-enzyme inhibitors and beta blockers. Respiratory management relies on home mechanical ventilation and couph assist.

The investigators aim

* to analyze cardiac and respiratory function over time in patients with muscular dystrophies followed at the home mechanical ventilation unit.

* to determine the long term impact of the home mechanical ventilation on cardiac and respiratory events and to define the predictive factors associated.

Patients will be included retrospectively. Tissue Doppler imaging and Tricuspid annular plane systolic excursion were used for the assessment of the right ventricle. Left ventricular ejection fraction was used for the analysis of the left ventricle. Respiratory function was assessed using the vital capacity, the maximal inspiratory pressure, the maximal expiratory pressure, the peak couph flow rate.

Study Timeline

• Study First Submitted: 2015-06-26
• Study First Submitted QC: 2015-07-15
• Study First Posted: 2015-07-17
• Study Start: 2016-07
• Status Verified: 2018-07
• Last Update Submitted: 2018-08-16
• Last Update Posted: 2018-08-17
• Primary Completion: 2018-11
• Study Completion: 2018-11

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 350

Inclusion Criteria

• Age higher or equal to 18 years
• Duchenne muscular dystrophy
• Becker muscular dystrophy
• Sarcoglycanopathy
• limb girdle muscular dystrophy
• Pompe disease
• Steinert disease
• FSH disease
• Others myopathies

Exclusion Criteria

• Minor patient
• Sepsis condition

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
predictive factors for long term survival	10 years

Secondary Outcome Measures

Name	Time	Method
predictuve factors for cardiac events	10 years
predictive factors for respiratory events	10 years
cumulative cardiac events incidence	10 years
cumulative respiratory events incidence	10 years
annual rate of maximal inspiratory pressure decline	10 years
annual rate of maximal expiratory pressure decline	10 years
annual rate of left ventricular ejection fraction decline	10 years
annual rate of respiratory vital capacity decline	10 years

Trial Locations

Locations (1)

Hopital Raymond Poincare; 🇫🇷 Garches, France