A New Era in the Management of Rett Syndrome: Are Neurologists Ready for Disease-Modifying Therapies?
Colleen Heffner1, Carole Drexel1, David Lieberman2, Paige Nues3
1PlatformQ Health, 2Boston Children's Hospital, 3International Rett Syndrome Foundation
Objective:
To assess the influence of two years of continuing medical education (CME) on clinicians’ (HCPs) ability to recognize key signs of Rett syndrome (RTT), confirm its diagnosis, and evaluate the place of emerging treatments in the standard of care.
Background:
The development of disease-modifying therapies for RTT has made it imperative for HCPs to diagnose affected children early, so they can benefit from multidisciplinary care and breakthrough treatment.
Design/Methods:
One 60-minute CME activity was launched live-online on 4/1/21 and expired on 4/1/2022 (year 1). The second activity launched on 8/18/22 and will remain on-demand until 8/18/23 (year 2). Test questions were administered pre, immediately post, and 2 months post activity. Responses from polling and surveys were analyzed for engagement, lessons learned, and continuing gaps. Chi-square compared paired responses (pre/post and pre/2 mos, P<0.05).
Results:
As of 9/4/22, 587 HCPs have participated in the activities, with 108 seeing 6 patients with RTT each month. Pre-activity, 50% of HCPs reported not being confident in diagnosing and managing RTT. Participation significantly enhanced awareness of RTT diagnosis (+30%) and multidisciplinary management (+34%, both P<0.05) and knowledge of emerging agents (+31%, P<0.05). Baseline knowledge and competence related to RTT were similar during year 1 and 2. More than 90% of HCPs reported that the content positively impacted their practice and gave examples (e.g., diagnosing a new patient and discussing treatment options).
Conclusions:
Data support the positive impact of live-online CME to prepare HCPs to care for patients with RTT. However, baseline knowledge and competence were similar in year 1 and 2, indicating that there is a need for additional education to induce lasting changes in the care of RTT.