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Rett syndrome and real-world treatment patterns of trofinetide in the United States
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DOI：10.1080/13696998.2026.2682668.png)
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Abstract
En 中文
Trofinetide (TROF) remains the first and only FDA-approved pharmacologic treatment for Rett syndrome (RTT). There is limited real-world evidence on TROF use, restarts, dosing patterns and predictors of non-persistence. This study evaluated long-term treatment patterns and baseline predictors of non-persistence among RTT individuals initiating TROF.
A retrospective claims analysis of linked IQVIA Anonymized Patient Level Data and TROF pharmacy data (1 January 2021 to 30 September 2024) was performed. RTT individuals initiating 1st TROF prescription (RX) between 1 April 2023 and 31 March 2024 with ≥6 months of pre- and post-index continuous enrollment were classified as persistent (gap ≤90 days) and non-persistent. Median (IQR) time on treatment was analyzed, and Kaplan-Meier assessed time to non-persistence. Restarts among non-persistent were examined. Dosing patterns such as mean dose in milligrams (mg) and percentage target daily dose (%TDD) at each RX were evaluated. Predictors of non-persistence were assessed using logistic regression.
Among 1,175 TROF initiators, 54.9% were persistent and 45.1% were non-persistent; 7.6% of non-persistent individuals restarted. Mean ± SD age was 14.7 ± 10.8 vs. 16.5 ± 11.6 years and median ± IQR time on TROF was 14.3 ± 4.6 vs. 3.0 ± 3.8 months, respectively. Over 75% remained on treatment beyond 3 months, and more than half continued through study follow-up. Mean twice daily (BID) TROF dose was lower among persistent (7422.0 mg) vs. non-persistent (7850.8 mg) at RX1 and increased to 7953.7 mg vs 8423.4 mg, respectively at RX2. Thereafter, the persistent individuals maintained stable dosing, while the non-persistent showed greater fluctuation over time. Similar patterns were observed for %TDD. Older age and history of infectious disorders were significantly associated with non-persistence.
In this real-world analysis, more than half of RTT individuals remained persistent on TROF for ≥14 months. Stable dosing patterns were observed among persistent individuals, while greater dose variability was observed among non-persistent individuals. Thus, these findings may provide guidance to clinicians about long-term TROF use in real-world practice.
Keywords:
Rett syndrome
trofinetide
adherence
treatment persistence
real-world evidence
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