Authors :
Presenting Author: Wesley Kerr, MD, PhD – University of Pittsburgh Neurology
Jaya Khushalani, MD, PhD – UCB
Heidi Henninger, MD – UCB
Patrik Ohagen, BS – UCB
Renjie Li, MS – Ambit Inc.
Nakul Yadav, PhD – Ambit Inc.
Adam Strzelczyk, MD, MHBA, FEAN – Goethe University Frankfurt, Epilepsy Center Frankfurt Rhine-Main, University Medicine Frankfurt
Rationale:
Real-world evidence of fenfluramine (FFA) use among patients with Lennox-Gastaut syndrome (LGS) is limited. This study assessed FFA persistence in patients with LGS and explored characteristics that may be associated with FFA use and persistence from a large US claims database.Methods:
This was a retrospective study of patients with LGS (ICD-10, G40.81) from 1/1/2021–6/30/2024 using the Komodo US healthcare claims database.
For persistence analysis, patients were required to have ≥1 FFA prescription claim (earliest claim was used as FFA initiation date), ≥2 LGS claims (≥1 month apart), ≥3 months of pre-FFA initiation and ≥6 months of post-FFA initiation claims data. The percentage of patients with FFA persistence (continuous FFA claims with no gaps >90 days) at 6 and 12 months post-FFA initiation were evaluated using Kaplan–Meier analysis.
Demographic and clinical characteristics in patients who remained FFA persistent for ≥12 months were compared with nonpersistent patients (discontinued FFA in < 12 months). A similar analysis was performed comparing patients who received FFA (regardless of persistence) with those who did not. In this analysis, the FFA initiation date was used for persistent and nonpersistent patients; the index date for patients not receiving FFA was 1/1/2023. For both groupwise comparisons, patients were required to have claims data for 12 months pre- and post-index date. Group-level comparisons of demographic and baseline characteristics were evaluated using 2 sample t-tests and chi-square tests.
Results:
Data from 544 patients with LGS were included for assessments of 6- and 12-month FFA persistence. FFA persistence was 73% and 61% at 6 and 12 months, respectively (Figure).
There were no significant differences in demographics or baseline characteristics between patients with 12-month persistence (n=148) and nonpersistent patients (n=216).
Compared with patients who did not receive FFA (n=2361), patients receiving FFA (n=373) had a significantly higher comorbidity burden in the pre-index period, including developmental impairments, behavioral disorders, mobility dysfunction, respiratory/cardiovascular complications, sleep disturbances, and gastrointestinal disorders. Additionally, patients who received FFA had a significantly higher average area deprivation index (Table).
Conclusions:
In this first real-world evaluation of patients with LGS who received FFA, persistence was 73% and 61% at 6 and 12 months, respectively. No significant differences in demographic or clinical characteristics of FFA-persistent and nonpersistent patients were observed. Here, patients who received FFA were younger, resided in more socioeconomically disadvantaged neighborhoods, and had greater disease severity, including a greater number of previously attempted ASMs, compared with patients who did not receive FFA. While claims data limit the direct, formal measurement of seizure and non-seizure outcomes, these data indicate that >60% of patients with LGS who initiated FFA experienced treatment benefits in continuing FFA for ≥12 months.Funding:
UCB.