PURPOSE: Patients with Duchenne Muscular Dystrophy (DMD) require 24 hour/day ventilatory support for prolonged survival. The best method of ventilation is controversial; options include mechanical ventilators with nasal or mouthpiece interfaces or tracheostomy. Tracheostomy is common when patients require ventilation > 18 hours/day. We describe characteristics and outcomes of our patients with DMD ventilated 24 hr/day using noninvasive positive pressure ventilation with a bi-level device and a nasal interface (n-NPPV).
METHODS: The IRB approved our study. We reviewed electronic charts of all patients from DMD clinic and identified the 24 hr/day n-NPPV users during the time period January, 1999 through May, 2010. All patients used mechanically assisted cough and were treated for cardiomyopathy.
RESULTS: All patients achieved prolonged survival (age > 20 years and survival > 5 years after vital capacity fell below 1 Liter). Nine patients are alive and use n-NPPV 24 hrs/day. Their mean age: 28.9 years (range 21-34, SD 3.5). They have used 24 hr/day n-NPPV for mean 4.4 years (range 0.25-9, SD 3.9, median 6). All patients speak, swallow, and are mobile, with n-NPPV on their wheelchairs. Two patients progressed to tracheostomy. The first used 24 hr/day n-NPPV for 3 years then died at age 40 years, one year after tracheostomy. The other used 24 hr/day n-NPPV for 4 years and is alive at age 35 years, 5 years after tracheostomy. Three patients used 24 hr/day n-NPPV for 2 to 8+ years, then died. Mean age at death: 27 years (range 23-33, SD 5.0).
CONCLUSION: Our choice for 24 hr/day noninvasive ventilation, using a bi-level device with a nasal interface, is feasible and allows patients with DMD to achieve prolonged survival while avoiding tracheostomy. Patients retain mobility, speech and some swallowing ability, fostering quality of life.
CLINICAL IMPLICATIONS: There are various options to achieve ventilation of patients with advanced DMD, and the technique should be individualized for each patient. Further study is needed to determine the best method of 24 hr/day ventilation for prolonged survivors of DMD.
DISCLOSURE: Garey Noritz, No Financial Disclosure Information; No Product/Research Disclosure Information