Continuous Ventilatory Support Without Hospitalization or Tracheotomies: A Retrospective Observational Study of Two Centers’ Outcomes for Duchenne Muscular Dystrophy

Introduction Duchenne muscular dystrophy (DMD) management often results in tracheostomies or palliative care deaths. Methods Two centers, A in the U.S., and B in Japan report 486 with DMD, including 263 dependent on continuous noninvasive ventilatory support (CNVS), and mechanical in-exsufflation (MIE). When needing intubation, all were extubated to CNVS and MIE whether ventilator weanable or not. Results Of 320 NVS users, 263 extended it to CNVS without hospitalization or acute respiratory failure. Another 57 were extubated to, and 15 decannulated to CNVS. Of 223 NVS users in Center A, 197 eventually required CNVS over 10.1 ± 6.3 years with 91 surviving to at least age 35.4 ± 9.0 (21-58) years; 106 died or were lost to follow-up at 34.3 ± 9.0 (18-58) years of age. Center B had 97 NVS/CNVS users; 31 died at 36.4 ± 8.1 (17-46) years of age. Sixty-six became CNVS dependent from age 24.3 with 35 now 38.2 ± 6.7 (29-51) years of age. The centers had 14 over age 50, 57 over age 40. Three underwent tracheotomies for chronic lung disease. Conclusion The facts that 263 became CNVS dependent without hospitalization, that 15 using trach ventilation were decannulated to CNVS, and none required tracheotomies suggest that airway tubes are unnecessary for these patients.