Respiratory Support in Spinal Muscular Atrophy Type I: A Survey of Physician Practices and Attitudes

Objective. To determine whether there is variability in the attitudes and practices of physicians regarding treatment of respiratory failure in children with spinal muscular atrophy type I (SMA type I) and, if so, whether this variation is associated with professional training. Methods. This was a descriptive, cross-sectional survey mailed to a randomly selected subset of the Child Neurology Society, pediatric members of the Society of Critical Care Medicine and to the membership of the Pediatric Interest Section of the American Academy of Physical Medicine and Rehabilitation. A scenario of a child with SMA type I in respiratory distress was followed by questions that explored practices and attitudes regarding mechanical ventilation. Results. Fifty-seven percent of intensivists (75 of 132), 39% physiatrists (61 of 155), and 34% of neurologists (61 of 155) responded. Specialists differed as to whether they offered and/or recommended respiratory support to patients with SMA type I. Intensivists were less likely to offer and recommend tracheostomy than physiatrists. Intensivists were also significantly less likely than physiatrists to agree with statements supporting the ethical necessity of noninvasive mechanical ventilation (NIMV) and intubation in the setting of an acute respiratory illness, and NIMV and tracheostomy in the setting of chronic respiratory failure. Although parallel differences were found between physiatrists and neurologists regarding their attitudes toward mechanical ventilation, no significant differences were detected between intensivists and neurologists. Finally, physicians who reported that a high percentage of their patients with SMA type I received “comfort care only” also tended to view mechanical ventilation, ie, use of NIMV for chronic respiratory failure, use of intubation for an acute respiratory infection, and use of tracheostomy for chronic respiratory failure as an unreasonable intervention in most circumstances. Conclusions. We found a wide variation in physician practice regarding the mechanical ventilation of patients with SMA type I. This study suggests a wide variation not only in what is recommended but also in what is actually offered to families of these children. Furthermore, the study suggests that physician training and attitudes affect recommendations regarding mechanical ventilation and ultimately family decision making. Pediatrics 2002;110(2). URL: http://www.pediatrics.org/ cgi/content/full/110/2/e24; spinal muscular atrophy, Werdnig-Hoffman disease, mechanical ventilation, physician attitudes, ethics, family decision making, survey. ABBREVIATIONS. SMA, spinal muscular atrophy; NIMV, noninvasive mechanical ventilation. Spinal muscular atrophy (SMA) type I, or Werdnig-Hoffman disease, is a devastating neuromuscular disease of childhood. Children with this disease typically have normal intelligence and remain alert and attentive to their environment, despite muscular weakness, and usually die from respiratory failure by age 2 years.1,2 Despite evidence from a small number of centers reporting success in prolonging life with mechanical ventilation,3–6 respiratory support for children with SMA type I is not widely accepted. Several important textbooks of pediatrics and pediatric neurology, for example, recommend against any mode of artificial ventilation for these patients.7,8 It is not clear whether these recommendations are based on the view that mechanical ventilation for these patients is not effective at sustaining life or that the quality of the life sustained is not of sufficient value. The purpose of this study was to describe the clinical practices of physicians regarding mechanical ventilation of patients with SMA type I. We also sought to explore the rationale underlying physician practices with the goal to understand better the basis for decision making. Our hypothesis was that wide variability exists in the care options presented and recommended to the families of children with SMA type I and that this variability may correlate with physician specialty, training, and attitudes.