PURPOSE: Survival among patients with Duchenne muscular dystrophy (DMD) has increased due to improved cardiopulmonary management. However, due to prolonged survival, patients are now exposed to the risk of developing major medical complications. In this report, we describe the frequency and nature of major non-cardiopulmonary complications in patients with DMD and prolonged survival due to use of assisted ventilation.
METHODS: Retrospective chart review of all pts with DMD in our clinic who are alive and over 20 years of age. Prolonged survival was attributed to assisted ventilation if the pt lived for > 5 years after vital capacity fell below 1 liter (Phillips et al AJRCCM, 2001).
RESULTS: 27 pts in our clinic are alive and > 20 yrs old. Complications: 15 pts have malnutrition/dysphagia requiring gastrostomy tube placement; 6 pts have renal calculi; 2 pts have diabetes and use insulin; 2 pts have deep venous thrombosis and were anti-coagulated; 1 pt has gallstones; 1 pt has inflammatory bowel disease. Of the 19 pts with one or more of these complications, 16 use noninvasive positive pressure ventilation (NPPV) and 2 are ventilated via tracheostomy. The 18 pts using assisted ventilation have thusfar survived a mean (+/− SD) of 6.5 +/− 4.3 yrs after their vital capacity fell below one liter (with 12 of 18 pts surviving > 5 years past the fall below one liter). Their mean survival thusfar after starting assisted ventilation is 7.6 +/− 3.8 yrs. Twelve of the 18 pts are ventilated 24 hrs/day.
CONCLUSION: Our pts with DMD who achieve prolonged survival through the use of assisted ventilation experience a high incidence of major non-cardiopulmonary medical complications. Therapy for these complications, such as gastrostomy placement, urologic procedures, anti-coagulation and insulin use, imposes additional risks.
CLINICAL IMPLICATIONS: These findings have significant implications regarding potential morbidities, burden of care and medical management in pts with DMD whose survival is prolonged by long-term mechanical ventilation.
DISCLOSURE: Kenny Nguyen, None.