RANDOMIZED TRIAL OF PREVENTIVE NASAL VENTILATION IN DUCHENNE MUSCULAR-DYSTROPHY

Duchenne muscular dystrophy (DMD) is the most common muscular dystroph y in children. Paralysis of respiratory muscles causes a decrease in f orced vital capacity (FVC) from age 12 years, and death occurs between 20 and 25 years old and is usually related to respiratory insufficien cy. Uncontrolled studies suggest that early home use of nasal intermit tent positive-pressure ventilation (NIPPV) in DMD patients free of res piratory failure could limit progression of the restrictive syndrome a nd therefore improve survival Because efficacy of preventive NIPPV has not been demonstrated in a controlled trial, we undertook a randomise d multicentre study in which 70 patients with DMD were included. Patie nts were free of daytime respiratory failure and FVC was between 20 an d 50% of predicted values. At least 6 h of nocturnal NIPPV (n=35) was compared with conventional treatment (n=35). During a mean follow-up o f 52 months, 10 patients died, 8 in the NIPPV group and 2 in the contr ol group (p=0.05, log-rank test). No differences were observed between the two groups for occurrence of hypercapnia, decrease of FVC below 2 0% of initial values, or use of necessary mechanical ventilation. Prev entive NIPPV did not improve respiratory handicap and reduced survival of DMD patients. Use of NIPPV for preventive purposes should be avoid ed in patients with FVC between 20 and 50% of predicted values.