Thursday, April 11, 2024

Dravet syndrome and fenfluramine

Posters at the Child Neurology Society Meeting 2023

Outcomes Among Individuals With Dravet Syndrome Using Fenfluramine: A Retrospective Analysis Using U.S. Claims Data

Dravet syndrome, a rare and severe developmental epileptic encephalopathy, has long presented challenges in terms of treatment and care. However, a new retrospective analysis utilizing U.S. claims data has unveiled promising outcomes for individuals with Dravet syndrome who were treated with fenfluramine, following its approval in June 2020 for the management of seizures associated with Dravet syndrome in the United States.

The objective of this analysis was to quantify the relationship between fenfluramine and seizure-related healthcare events through claims data. The study utilized the Komodo U.S. healthcare claims database to measure healthcare utilization, including rescue anti-seizure medication use and healthcare visits. Individuals with Dravet syndrome were identified by ICD-10 codes. Patients receiving prior/concomitant cannabidiol or stiripentol were excluded.

Among the 108 individuals with Dravet syndrome included in the analysis, 91 individuals (84%) had continuous fenfluramine use for a minimum of 6 months. This subset exhibited a 77% reduction in rescue antiseizure medication use (P<0.001). Moreover, the reductions extended to healthcare visits, with a 50% decrease in ER visits, a 27% reduction in outpatient visits, a 24% reduction in neurology visits, and a 24% reduction in inpatient hospitalizations.

The findings suggest that meaningful reductions in healthcare utilization were observed among individuals with Dravet syndrome treated with fenfluramine for at least 6 months. The 6-month persistency rate of fenfluramine usage indicates not only its efficacy but also its tolerability in this patient population.

Authors: Srihari Jaganathan, Derek Ems, Rob Sederman, Chen Chen, Shuang Wu

Diagnosis and Information Management of Dravet Syndrome in Recently Diagnosed Pediatric Patients: Results From a Dravet Syndrome Foundation Caregiver Insight Survey

The second late-breaking poster explored the challenges surrounding the early diagnosis of Dravet syndrome. Caregivers of pediatric patients aged ≤4 years, diagnosed with Dravet syndrome, were surveyed to gather insights into their experiences.

The survey, distributed via the Dravet Syndrome Foundation Family Network, received responses from 73 caregivers. The results highlighted that the average age at symptom onset was 5 months, while the average age at Dravet syndrome diagnosis was 12 months. Changes in healthcare providers were common, with the most frequent reasons being clinical expertise and trust. The poster further highlights the vital role that patient advocacy groups and online resources play in providing caregivers with valuable information and support during the diagnosis and management process.

Authors: Andrea Wilkinson, BA, Danya Kaye, BA, Veronica Hood, PhD, Mary Anne Meskis, Laurie Bailey, BCPA, Rebecca Burns, PharmD, PhD, Amélie Lothe, PhD

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