Background-Respiratory failure is the commonest cause of death in patients with Duchenne muscular dystrophy (DMD). Life expectancy is less than one year once diurnal hypercapnia develops. This study examines the eVects of nasal intermittent positive pressure ventilation (NIPPV) on survival in symptomatic Duchenne patients with established ventilatory failure. Methods-Nocturnal NIPPV was applied in 23 consecutive patients with DMD of mean (SD) age 20.3 (3.4) years who presented with diurnal and nocturnal hypercapnia. Results-One year and five year survival rates were 85% (95% CI 69 to 100) and 73% (95% CI 53 to 94), respectively. Early changes in arterial blood gas tensions following NIPPV occurred with mean (SD) PO 2 increasing from 7.6 (2.1) kPa to 10.8 (1.3) kPa and mean (SD) PCO 2 falling from 10.3 (4.5) kPa to 6.1 (1.0) kPa. Improvements in arterial blood gas tensions were maintained over five years. Health perception and social aspects of SF-36 health related quality of life index were reported as equivalent to other groups with nonprogressive disorders using NIPPV. Conclusion-Nasal ventilation is likely to increase survival in hypercapnic patients with Duchenne muscular dystrophy and should be considered as a treatment option when ventilatory failure develops. (Thorax 1998;53:949-952)

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Noninvasive positive pressure ventilation delivered by nasal mask or facemask has been used widely in the last decade to manage chronic ventilatory failure in adults with neuromuscular and chest wall disease. However, it has been thought that paediatric patients would not be able to tolerate masks, and previous anecdotal reports on the paediatric application of mask ventilation have not assessed the effects on nocturnal and arterial blood gas control. Domiciliary mask ventilation has been used in 40 children with ventilatory insufficiency due to congenital neuromuscular and skeletal disease aged 9 months–16 yrs. Eighteen patients had symptomatic nocturnal hypoventilation, 17 had diurnal ventilatory failure, three were referred for weaning and two had frequent chest infections associated with sleep‐disordered breathing. Thirty eight of the 40 patients tolerated mask ventilatory support long‐term. Diurnal mean±sd oxygen tension in arterial blood (Pa,O2) increased from 8.5±1.8–10.9±1.7 kPa (p<0.001) and meansd carbon dioxide tension in arterial blood (Pa,CO2) fell from 7.0±1.6–5.9±0.8 kPa (p=0.01) following initiation of ventilatory support. Mean and minimum nocturnal Pa,O2 and peak transcutaneous carbon dioxide tension (Ptc,CO2) (n=21) improved significantly. Mask ventilation can be used successfully in young children and reverses ventilatory insufficiency due to congenital neuromuscular and skeletal disease.

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