Authors :
Presenting Author: Nazia Rashid, PharmD, MS – Acadia Pharmaceuticals
Vinod Yakkala, PhD – Anlitiks, Inc
Daksha Gopal, PhD – Anlitiks, Inc
Kitty Rajagopalan, PhD, MS – Anlitiks, Inc
Rationale:
Approximately 90% of individuals with Rett syndrome (RTT) have co-existing neurological (i.e., epilepsy) conditions. Trofinetide (TROF) is the first and only FDA approved treatment for RTT. Real-world data evaluating rates of epilepsy among those treated with TROF (TROF group) vs. not treated with TROF (No-TROF group) are needed. Methods:
A retrospective analysis of RTT individuals [diagnosis: ICD-10-CM: F84.2] from a linked medical and pharmacy claims database from 01/01/2021 to 09/30/2024 was completed. RTT individuals were categorized into two groups based on treatment status: TROF group (index date: first TROF prescription between 04/01/2023 and 09/20/2023) and No-TROF group (index date: assigned date using a risk set sampling method). Individuals with brain trauma or cerebrovascular disease prior to RTT diagnosis were excluded. Those with continuous enrollment (≥12-month pre- and post-index) were included. Furthermore, 1:1 matching was conducted using age, gender, and time from RTT diagnosis to index date among both groups. Rates of epilepsy were compared as the following: 1) among the two groups during 12-month post-index; 2) during pre-index among those identified during 12-months post-index; 3) occurrence of epilepsy among the TROF group (last TROF prescription + days’ supply). Descriptive statistics were used to summarize means for continuous variables; counts and percentages for categorical variables. Results:
A total of 2,950 RTT individuals were identified [TROF=766 (25.9%); No-TROF=2,184 (74.0%)]. After matching for both groups [n=644], mean age was 13.7 (SD) vs. 15.2 (SD) years in TROF vs. No-TROF group; a similar proportion of males in both groups (3.9%). Epilepsy rates during post-index were 333 (51.7%) vs. 348 (54.0%) for TROF vs. NO-TROF group, respectively. Among these groups, the pre-index epilepsy rates were 278/333 (83.5%) vs 297/348 (85.3%), respectively. The post-index epilepsy rate during TROF treatment was 287 (44.6%); among these individuals, 242/287 (84.3%) had pre-index epilepsy. Conclusions:
In this real-world analysis of RTT individuals, the TROF vs. NO-TROF groups exhibited similar rates of epilepsy during the 12-month post-index period. Among individuals with post-index epilepsy, there were similar rates of pre-index epilepsy across both groups. These results suggest that the occurrence of post-index epilepsy is attributable to pre-index epilepsy or to the underlying disease itself.Funding: This study was funded by Acadia Pharmaceuticals, Inc.