Chest
Clinical InvestigationsLong-Term Follow-up of Nocturnal Ventilatory Assistance in Patients with Respiratory Failure Due to Duchenne-Type Muscular Dystrophy
Section snippets
METHODS
Patients were referred for the management of hypercarbia from the Muscular Dystrophy Association Clinic at New England Medical Center. Patients had been diagnosed as having Duchenne or Duchenne-type muscular dystrophy using standard clinical criteria including male sex, elevation of creatine phosphokinase, and a compatible muscle biopsy and clinical picture. All patients were in functional class 93 and had severe quadraparesis.
All patients expressed a desire to try intermittent ventilatory
RESULTS
Table 1 shows age at initiation of assisted ventilation, respiratory parameters at initiation of assisted ventilation and at times of early and extended follow-up and clinical outcome in the eight patients comprising our series. Most patients developed respiratory failure at approximately 20 years of age, as is typical for Duchenne muscular dystrophy. Delayed onset in patients 1 and 6 suggests that they may have had a more slowly progressing variety. All patients had severe restriction of their
DISCUSSION
In our series of eight patients with Duchenne or Duchenne-type muscular dystrophy with respiratory failure, we found, as others have,6, 7, 8, 9, 10 that nocturnal ventilatory assistance using noninvasive ventilators brought about a sustained reversal of hypoventilation and improvement in symptoms of chronic hypercarbia. The clinical course was stabilized in most patients who required infrequent hospitalizations despite minimal pulmonary reserve. Although our patients tried a variety of negative
ADDENDUM
Since acceptance of this manuscript, patient 4 died suddenly 62 months after initiation of ventilation when his ventilator became accidentally detached from his tracheostomy tube. Autopsy revealed a well-healed tracheostomy without stricture, severe dystrophy of skeletal muscle, and moderate fatty infiltration of the myocardium without evidence of cardiac failure.
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Breathing with neuromuscular disease: Does compensatory plasticity in the motor drive to breathe offer a potential therapeutic target in muscular dystrophy?
2019, Respiratory Physiology and NeurobiologyCitation Excerpt :Studies reporting the apnoea-hypopnea index in DMD suggest that patients suffer from moderate sleep apnoea (Smith et al., 1989a; Bersanini et al., 2012). As patients reach the second decade of life, they are at an increased risk of hypoventilation during sleep (Smith et al., 1989b; Suresh et al., 2005), culminating in hypoxaemia during sleep (Smith et al., 1988; Barbé et al., 1994; Khan and Heckmatt, 1994) and nocturnal hypercapnia (Baydur et al., 1990; Mohr and Hill, 1990; Bersanini et al., 2012). Patients are known to develop scoliosis, further impacting on respiratory function.
Current treatment of adult Duchenne muscular dystrophy
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2007, Current Anaesthesia and Critical CareInitiation of home mechanical ventilation in children with neuromuscular diseases
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2008, NeurotherapeuticsCitation Excerpt :This includes weakness of inspiratory muscles resulting in hypoventilation and expiratory muscle weakness resulting in weak cough and inability to effectively clear secretions. The greatest contribution to improvement in respiratory care, and thus likely the greatest contribution to extending longevity in DMD, has been noninvasive ventilation.17–19 Pharmacologically, the near lifelong treatment of DMD with corticosteroids appears to have produced improvements in pulmonary function.15,20
Manuscript received March 7; revision accepted June 23.