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Updated: Sep 9, 2025
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019
Continuous Ventilatory Support Without Hospitalization or Tracheotomies: A Retrospective Observational Study of Two
John R Bach1, Yuka Ishikawa2, Maria Victoria Herrero3
1From the Department of Physical Medicine and Rehabilitation, Rutgers University-New Jersey Medical School, Newark, New Jersey.
Introduction:
Duchenne muscular dystrophy management often results in tracheostomies or palliative care deaths.
Methods:
Two centers, A in the US and B in Japan, report 486 with Duchenne muscular dystrophy, including 263 dependent on continuous noninvasive ventilatory support, and mechanical in-exsufflation. When needing intubation, all were extubated to continuous noninvasive ventilatory support and mechanical in-exsufflation whether ventilator weanable or not.
Results:
Of 320 noninvasive ventilatory support users, 263 extended it to continuous noninvasive ventilatory support without hospitalization or acute respiratory failure. Another 57 were extubated to, and 15 decannulated to continuous noninvasive ventilatory support. Of 223 noninvasive ventilatory support users in Center A, 197 eventually required continuous noninvasive ventilatory support over 10.1 ± 6.3 yrs with 91 surviving to at least age 35.4 ± 9.0 (21-58) yrs; 106 died or were lost to follow-up at 34.3 ± 9.0 (18-58) yrs of age. Center B had 97 noninvasive ventilatory support/continuous noninvasive ventilatory support users; 31 died at 36.4 ± 8.1 (17-46) yrs of age. Sixty-six became continuous noninvasive ventilatory support dependent from age 24.3 with 35 now 38.2 ± 6.7 (29-51) yrs of age. The centers had 14 over age 50, 57 over age 40. Three underwent tracheotomies for chronic lung disease.
Conclusions:
The facts that 263 became continuous noninvasive ventilatory support dependent without hospitalization, that 15 using trach ventilations were decannulated to continuous noninvasive ventilatory support, and that only three of 486 apparently required tracheotomies suggest that airway tubes are unnecessary for these patients without severe chronic lung disease.
Insights
Continuous noninvasive ventilatory support (CNVS) and mechanical in-exsufflation (MIE) can help Duchenne muscular dystrophy (DMD) patients avoid tracheostomies, extending lives without hospitalization.
Area of Science:
- Neurology
- Pulmonology
- Biomedical Engineering
Background:
- Duchenne muscular dystrophy (DMD) management traditionally involves high rates of tracheostomy or palliative care.
- Respiratory insufficiency is a primary cause of mortality in DMD patients.
Purpose of the Study:
- To evaluate the efficacy of continuous noninvasive ventilatory support (CNVS) and mechanical in-exsufflation (MIE) in managing respiratory function in DMD patients.
- To determine if CNVS and MIE can reduce the need for tracheostomies in DMD patients.
Main Methods:
- A retrospective analysis of 486 DMD patients across two international centers (US and Japan).
- Patients requiring ventilatory support were extubated to CNVS and MIE.
- Data collected on hospitalization rates, acute respiratory failure, decannulation, and long-term survival.
Main Results:
- 263 out of 320 noninvasive ventilatory support (NVS) users transitioned to CNVS without hospitalization or acute respiratory failure.
- 15 patients using tracheostomy ventilation were successfully decannulated to CNVS.
- Significant survival rates observed, with multiple patients exceeding 40 and 50 years of age on CNVS.
Conclusions:
- CNVS combined with MIE offers a viable alternative to tracheostomies for respiratory management in DMD.
- This approach can prevent hospitalizations and improve long-term outcomes for DMD patients.
- Airway tubes may be unnecessary for many DMD patients utilizing advanced noninvasive ventilatory strategies.
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