Diagnosis and Treatment of Spinal Muscular Atrophy: Online, Case-based Education Successfully Improved Knowledge and Competence of Neurologists and Pe


Clinical Other

Poster Number: 8


Thomas Finnegan, PhD, Pakinam Aboulsaoud, PharmD, Perry Shieh, MD, PhD


1. Medscape Education, 2. Medscape Education, 3. David Geffen School of Medicine at UCLA

Spinal muscular atrophy (SMA) is one of the most common heritable causes of death in infants. Despite the disorder’s prevalence, the nature of the associated complications and potentially severe sequelae, clinicians have limited knowledge regarding the diagnosis and treatment of SMA. To address this, we investigated whether a case-based educational activity was able to improve the knowledge and competence of neurologists and pediatricians regarding the diagnosis and management of SMA.

An online, text-based educational intervention comprised of 2 patient case scenarios was developed. Using a “test and teach” approach, clinicians were presented with multiple-choice questions to evaluate their application of evidence-based recommendations. Each response was followed by detailed, referenced, feedback to teach. Educational effect was evaluated with a repeated-pairs pre- to post-assessment study design in which each individual learner acts as his/her own control. A chi-square test was utilized to identify whether proportions of correct answers at pre and post were significantly different (P < .05 significance level). Cramer’s V was used to calculate the effect size of the intervention. Data were collected between December 5, 2018 and January 14, 2019.

The education resulted in a considerable educational effect for neurologists (n=100; V =.196) and an extensive educational effect for pediatricians (n=437; V =.271). Significant improvements were observed in several areas for both groups (P <.05 for all comparisons) including: interpretation of genetic testing results in a patient with SMA; personalizing the selection of supportive therapy in SMA; and identification of the appropriate SMA subtype where the currently approved drug-based therapy is most effective. Participation in the education resulted in 35% of neurologists and 49% of pediatricians reporting an increase in confidence regarding managing patients with hereditary motor disorders.

This study demonstrated the success of a targeted, online, interactive, case-based educational intervention on improving the knowledge and competence of both neurologists and pediatricians regarding the care of patients with SMA. The results indicated that both clinician groups would benefit from continued education on the clinical recognition and management of SMA.