Abstract Details

Title Real-world Benefits and Tolerability of Trofinetide for the Treatment of Adults with Rett Syndrome: the LOTUS Study

Topic General Neurology

Presentation(s) P11 - Poster Session 11 (11:45 AM-12:45 PM)

Poster/Presentation
Number 7-005

Objective

To characterize the real-world benefits and tolerability of trofinetide in the treatment of adults with Rett syndrome (RTT) using the results of the LOTUS study.

Background Trofinetide is approved for the treatment of RTT in patients aged ≥2 years in the United States and patients aged ≥2 years weighing ≥9 kg in Canada. The trofinetide clinical trial program enrolled patients with RTT aged 2-20 years. Adult patients constitute most patients with RTT, yet general neurologists often consider it the domain of pediatric neurologists.

Design/Methods LOTUS is an ongoing, prospective study of patients with RTT prescribed trofinetide in real-world clinical practice. Caregivers of patients who are prescribed trofinetide under routine clinical care are eligible to participate. Assessments include the Behavioral Improvement Questionnaire (BIQ), the Quality-of-Life Inventory-Disability (QI-Disability) Questionnaire, and the Gastrointestinal Health Questionnaire. This analysis is focused on adult patients aged 20 years or older. Data are reported to 12 months since the initiation of trofinetide.

Results Overall, 77 adult patients with RTT, aged 21-60 years, were included in this analysis. The median age (interquartile range) at trofinetide initiation was 28 (24-33) years. The median dose reported at week 1 was 50.3% of the target weight-banded label dose; dosing ranged from 50.0-66.9% of the target weight-banded label dose between week 5 and month 12. Behavioral improvements reported with the BIQ were nonverbal communication (48–58%), alertness (39–58%), and social interaction/connectedness (32–38%). Modest improvements in patient quality of life (QoL) were reported. Caregivers reported that adult patients were most likely constipated or voided formed/normal stools. The highest incidence of diarrhea was reported at week 2 of treatment (60.0%); most reports were contained inside the patient’s diaper.

Conclusions

The real-world benefits and tolerability of trofinetide in adults with RTT in LOTUS followed similar trends to the overall LOTUS population.

Authors/Disclosures
Louise Cosand, PhD PRESENTER	Dr. Cosand has received personal compensation for serving as an employee of Acadia Pharmaceuticals Inc. Dr. Cosand has or had stock in Acadia Pharmaceuticals Inc.
Ryan Bucco	Dr. Bucco has received personal compensation for serving as an employee of Acadia Pharmaceuticals.