Abstract Details

Title Impact of Real-World, Case-Driven CME Learning on Diagnostic and Therapeutic Practice in Rett Syndrome

Topic Child Neurology and Developmental Neurology

Presentation(s) P7 - Poster Session 7 (8:00 AM-9:00 AM)

Poster/Presentation
Number 8-006

Objective To characterize the impact of a continuing medical education initiative that incorporated real-world patient scenarios in addressing diagnostic and treatment challenges in Rett syndrome (RTT).

Background The approval of trofinetide, the first therapy for RTT, requires greater clinician preparedness in treatment selection, caregiver counseling, and team-based management.

Design/Methods A multidisciplinary expert faculty panel developed a multicomponent CME curriculum (a 60-minute foundational module and a three-part, case-based series) centered on practical strategies to facilitate early diagnosis, therapy selection, AE management through neurology–gastroenterology collaboration, and integration of rehabilitation therapy. Real-world cases were gathered via a survey of clinicians who manage patients with RTT. The impact of the program was assessed through pre- and post-tests and intended practice changes. Chi-square tests compared responses (P<0.05; pre/post), effect size was estimated with Cohen’s d.

Results As of October 9, 2025, the education had reached 6,207 clinicians (343 on-platform, 5,864 via microlearning). At baseline, the most frequently cited diagnostic challenges were: differential diagnosis of RTT (33%), limited exposure to RTT (26%), and difficulty interpreting early signs in the absence of classic regression patterns (25%). Post education, data demonstrated substantial gains in recognition of early RTT signs (pre 48%, post 78%; P < 0.001), trofinetide efficacy and safety (pre 44%, post 81%; P < 0.001), and competence in AE management (pre 37%, post 79%; P < 0.001). Other areas of noticeable improvement included confidence in multidisciplinary coordination (pre 42%, post 76%; P < 0.001) and in caregiver counseling (pre 29%, post 68%; P < 0.001). The overall educational effect size was large (Cohen’s d = 0.82), with 79% of participants self-reporting intended practice changes, most related to AE mitigation and enhanced team-based collaboration.

Conclusions

The initiative produced measurable gains in clinician knowledge, competence, and confidence in RTT care, supporting improved diagnostic and management practices.

Authors/Disclosures
Vandana Gupta, MSc PRESENTER	Ms. Gupta has nothing to disclose.
Carole Drexel (PlatformQ LLC)	Carole Drexel has nothing to disclose.
Henry Hasson, MD	Dr. Hasson has nothing to disclose.
David N. Lieberman, MD, PhD (Boston Children'S Hospital)	Dr. Lieberman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Acadia Pharmaceuticals. Dr. Lieberman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Taysha Gene Therapies. Dr. Lieberman has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurogene.

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