Trends in survival from muscular dystrophy in England and Wales and impact on respiratory services

Abstract

Respiratory failure is an important terminal event in muscular dystrophy, but increasingly is effectively treated by non-invasive ventilation. This study was designed to assess mortality statistics in this patient group in order to get an indication of future demand. Mortality data for all deaths from muscular dystrophy registered by death certification in England and Wales between 1993 and 1999 were analysed. In total, 817 deaths from muscular dystrophy were registered between 1993 and 1999. Annual number of deaths was unchanged over this period. Median age at death (interquartile range) for all cause muscular dystrophy increased from 20 (17-42.5) years in 1993, to 26 (17.5-63) years in 1999. Respiratory failure was the primary or contributory cause of death in 82% of cases. Two thirds of these deaths were during acute infection. We can expect 100 patients with muscular dystrophy to develop respiratory failure in England and Wales each year, so non-invasive ventilation services probably need to be able to provide for 0.2 new patients per 100,000 population annually. Respiratory services also need to provide adequate monitoring and early treatment of infection in these patients.

Clinical profiles and prognosis of acute heart failure in adult patients with dystrophinopathies on home mechanical ventilation

“…Left ventricular EF has been reported to be a predictive factor for mortality in DMD . Also, LBBB is associated with heart failure and poor prognosis . The mortality we observed in our population lies well higher than that of the general cardiologic population.…”

“…17 Also, LBBB is associated with heart failure and poor prognosis. 18 The mortality we observed in our population lies well higher than that of the general cardiologic population. This could be due to some particularities of the DMD-associated end stage cardiomyopathy (mean left ventricular EF at 25% in our study).…”

“Hospital at Home” for Neuromuscular Disease Patients With Respiratory Tract Infection: A Pilot Study

“…4,5 Respiratory tract infection is the most common cause of hospitalization for neuromuscular disease (NMD) patients, punctuating the clinical course of these individuals and triggering over 90% of episodes of acute respiratory failure. 6 Acute respiratory failure may be caused by airway mucus accumulation, atelectasis, and, ultimately, blood gas derangement, and can require intensive respiratory assistance and close monitoring. 7 Uncontrolled clinical trials have shown that respiratory tract infections in NMD patients can be effectively managed by a home-care protocol based on the combination of continuous noninvasive ventilation (NIV), intensive manually and/or mechanically assisted cough, and arterial oxygen saturation (S pO 2 ) monitoring, preventing or reversing oxyhemoglobin desaturation, and reducing the need for hospitalization, intubation, and tracheotomy.…”

Adherent Primary Cultures of Mouse Intercostal Muscle Fibers for Isolated Fiber Studies

“…Our culturing of intercostal muscles aims to do this not only by providing a particular model system specifically relevant to the pathology of COPD and the respiratory complications of the muscular dystrophies [16, 17], but also by improving the general techniques of adherent isolated muscle fiber culture. If these techniques are optimized for poorly adhering muscle types, generating cultures from any muscle in the body may become substantially easier, and using the most relevant tissue in a given disease model may become routine.…”

“…Indeed, in cases where diagnosis and treatment for muscular dystrophy is available, respiratory arrest has become the critical factor in the majority of fatalities arising from muscular dystrophy [16, 17]. While the vast majority of studies evaluating the function of the respiratory muscles have used the diaphragm muscle as model of study, information on the intercostal muscles, in particular at cellular level, is more fragmented [8].…”