Background: Increasing numbers of families are requesting active supportive management for their child with spinal muscular atrophy type 1 (SMA1), leading to longer survival and greater prevalence of affected children. Strong opinions exist among physicians for and against the provision of care measures prolonging life.
Objective: To describe current practice in the care of SMA1 in Canada, and explore the factors underlying inter-physician variability.
Methods: A cross-sectional survey of Canadian hospital-based pediatric neurologists and pediatric respirologists was performed in 2015. Odds ratios and 95% confidence intervals were calculated to compare proportions between groups.
Results: There was a 54% completion rate (99 physicians). Over half of participants believed that a disease modifying therapy was likely within 10 years. Quebec respirologists were 50 times less likely to offer long-term non-invasive ventilation (NIV) than respirologists in other provinces (OR 50.6, 95% CI 2.4-1075.3), and 20 times less likely to discuss tracheostomy with families (OR 20.4, 95% CI 2.0-211.8). High raters of perceived happiness of affected children were more likely to find NIV an acceptable measure for acute (OR 6.7, 95% CI 1.7-26.0) and chronic (OR 13.7, 95% CI 4.0-46.4) respiratory failure and prophylactic use (OR 5.8, 95% CI 2.2-15.6).
Conclusion: Physician knowledge, opinions, subjective perception of child happiness, and regional factors, all influence physicians' practices and the shared decision-making process. Parents may not be informed or offered all the services available to their child. Knowledge translation initiatives are needed to enhance SMA1 care. Pediatr Pulmonol. 2017;52:662-668. © 2016 Wiley Periodicals, Inc.
Keywords: neuromuscular diseases; quality of life; respiratory support; spinal muscular atrophy.
© 2016 Wiley Periodicals, Inc.