New challenges in the management of prolonged survivors of pediatric neuromuscular diseases: A pulmonologist's perspective

Many patients with pediatric neuromuscular diseases (NMDs) are now achieving prolonged survival through advances in management of the cardiopulmonary complications of their illnesses. Because respiratory complications are among the main causes of mortality and morbidity in these diseases, pulmonologists are in a unique position to observe and describe the largely unanticipated medical, social, and ethical problems generated when patients with progressive NMDs achieve prolonged survival. For example, prolonged survivors of pediatric NMDs are now experiencing previously rare or unknown medical complications, an unprecedented severity of burden of disease and the potential for prolonged impairment of quality of life. As the patients age, their families must cope with a high level of burden of care. Society's acceptance of the eligibility of these patients to utilize critical care resources, and issues related to the transition of prolonged survivors from pediatric to adult medical providers and venues have resulted in complex practical and ethical issues. In this article, the author, a pediatric pulmonologist closely involved in the care of patients with NMDs, will identify and discuss some of the major medical, social, and ethical implications of prolonged survival among these patients, with an emphasis on Duchenne muscular dystrophy (DMD), the most common of the pediatric NMDs.