Fenfluramine treatment for Dravet syndrome: Caregiver- and clinician-reported benefits on the quality of life of patients, caregivers, and families living in Germany, Spain, Italy, and the United Kingdom

Abstract

Objective: Clinical trial data and prior preliminary research indicate that fenfluramine (FFA) provides meaningful improvements in seizure-related and quality of life (QOL) outcomes for individuals with Dravet syndrome (DS), their caregivers, and their families. This study sought to replicate and extend these preliminary findings in a new sample of individuals with DS and their families who live in European countries.

Methods: Study participants were European clinicians and parents caring for individuals with DS who had participated in an EU FFA Early Access Program. Participants completed one-on-one semi-structured interviews and were asked the extent to which they noticed changes in a number of the child's seizure- and non-seizure-related QOL domains after starting FFA treatment. Participants were also asked about the benefits of FFA treatment to the caregivers' lives and for the family unit.

Results: 25 parent caregivers and 16 clinicians participated. The caregivers and clinicians reported improvements in both seizure-related (i.e., reductions in seizure activity, improvements in the frequency or type of seizure triggers and post-ictal recovery times, and improved post-seizure function) and non-seizure-related (e.g., cognition, focus, alertness, speech, academic performance, behavior, sleep, motor function) QOL domains after FFA treatment in individuals with DS. Caregivers also reported improved mood and more time for things they enjoyed, felt less overwhelmed, reported better sleep quality, and had less personal and family stress; clinicians corroborated most of these reports. All clinicians (100%) and most (96%) caregivers said they would "very likely" or "quite likely" recommend FFA to others with DS.

Conclusions: Real-world experience in Europe with FFA treatment is associated with meaningful improvements in many QOL domains for individuals with DS and their families; replicating findings from a previous study of DS patients and their families from the USA. Caregivers and clinicians provided specific examples of the benefits of FFA for people with DS, caregivers, and their families and are very likely to recommend FFA to others with DS.

Keywords: Caregiver; Dravet syndrome; Fenfluramine; Quality of life; Treatment effect.