VP.56 Perception of treatment efficacy among pediatric neurologists caring for patients with spinal muscular atrophy

Abstract

The therapeutic landscape of spinal muscular atrophy (SMA) has changed over the last few years with the appearance of new treatment options. However, limited information is available about how pediatric neurologists make therapeutic decisions. The aim of this study is to assess how pediatric neurologists perceive treatment efficacy and its influence on treatment decisions applying principles of behavioral economics. We conducted a multicenter, non-interventional, cross-sectional, web-based study. Pediatric neurologists involved in the care of patients with SMA were invited to participate in the study by the Spanish society of pediatric neurology. Participants completed a survey on risk preferences and management of eleven simulated case scenarios mimicking the current treatment landscape in types I-III SMA. Participants were also exposed to four scenarios to determine their treatment expectations. Concomitant behavioral characteristics that could be associated with therapeutic decisions were assessed. A total of 35 participants completed the study. Mean age (±SD) was 40.6 (9.6) years and 62.9% were women. Fourteen (40.0%) were pediatric neurologists specialized in SMA with a mean experience of 11.5 (9.11) years. The mean (SD) participants' expectation for having an improvement by starting a therapy for type I SMA I (case#1: 5-month-old) and type II SMA (case#6: 1-year-old) were both 59.6% (22.2 and 21.5, respectively). Contrarily, the expectation for a benefit with treatment declined to 20.2% (12.2) for a case scenario representing a 16-year-old treatment naïve patients with a diagnosis of an advanced type II SMA (case#10). There was a significant difference in treatment expectation between case#1 or case#6 vs. case#10 (p<0.0001). Overall, the combination of treatment expectations for all four simulated scenarios was 43.9% (±14.6). Participants´ older age (p=0.019), lower years of experience (p=0.035), lower aversion to ambiguity (p=0.015) and lower expectation to treatment benefits (p=0.006) were associated with inertia treatment initiation. Figure 1 despite participants' expectation of treatment benefits vs. predicted probability of treatment benefit. A better knowledge of evidence-based guidelines for the management of SMA is needed to reduce bias in the perception of therapeutic efficacy.