Clinical trial NCT02501083: Natural History of Cardiac and Respiration Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation
Official title: Natural History of Cardiac and Respiratory Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Heart, Respiratory, Disease, Functional
Keywords: cardiomyopathy
echocardiography
prognosis
respiratory
home mechanical ventilation
vital capacity
maximal inspiratory pressure
maximal expiratory pressure
Lead sponsor: Centre d'Investigation Clinique et Technologique 805
Sponsor class: OTHER
First posted: 2015-07-17
Enrollment: 350
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult patients with muscular dystrophies

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.

Eligibility criteria: 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

Primary outcomes:
measure: predictive factors for long term survival
timeFrame: 10 years
Secondary outcomes:
measure: predictuve factors for cardiac events
timeFrame: 10 years
measure: predictive factors for respiratory events
timeFrame: 10 years
measure: cumulative cardiac events incidence
timeFrame: 10 years
measure: cumulative respiratory events incidence
timeFrame: 10 years
measure: annual rate of left ventricular ejection fraction decline
timeFrame: 10 years
measure: annual rate of respiratory vital capacity decline
timeFrame: 10 years
measure: annual rate of maximal inspiratory pressure decline
timeFrame: 10 years
measure: annual rate of maximal expiratory pressure decline
timeFrame: 10 years
Other outcomes:
Locations:
city: Garches
country: France
facility: Hopital Raymond Poincare
Study officials:
affiliation: HOPITAL RAYMOND POINCARE
name: Abdallah FAYSSOIL, MDPhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Fayssoil A, Lazarus A, Wahbi K, Ogna A, Nardi O, Lofaso F, Clair B, Orlikowski D, Annane D. Cardiac implantable electronic devices in tracheotomized muscular dystrophy patients: Safety and risks. Int J Cardiol. 2016 Nov 1;222:975-977. doi: 10.1016/j.ijcard.2016.08.040. Epub 2016 Aug 6.
citation: Fayssoil A, Ogna A, Chaffaut C, Chevret S, Guimaraes-Costa R, Leturcq F, Wahbi K, Prigent H, Lofaso F, Nardi O, Clair B, Behin A, Stojkovic T, Laforet P, Orlikowski D, Annane D. Natural History of Cardiac and Respiratory Involvement, Prognosis and Predictive Factors for Long-Term Survival in Adult Patients with Limb Girdle Muscular Dystrophies Type 2C and 2D. PLoS One. 2016 Apr 27;11(4):e0153095. doi: 10.1371/journal.pone.0153095. eCollection 2016.