Fenfluramine treatment for dravet syndrome: Real-world benefits on quality of life from the caregiver perspective

Abstract

Objective: Prior research has demonstrated durable and profound reductions in seizure frequency and improvements in executive functions in individuals with Dravet syndrome (DS) who are treated with fenfluramine (FFA). This study aimed to understand the benefits of FFA from the perspective of the patients' caregivers.

Methods: Caregivers for a child with DS participated in semi-structured interviews to discuss the benefits of FFA treatment on the child with DS, the caregiver, and the family.

Results: 65 caregivers participated. Patients were between 2 and 33 years old and had been treated with FFA for an average of 22.7 months. The most commonly reported seizure-related benefits (> 50 % of participants) of FFA treatment included a reduction in seizure activity, fewer seizure triggers, and shorter post-ictal recovery. The most common quality of life (QOL) benefits in patients included improvements in cognitive function, alertness, and academic performance. In addition, the caregivers reported improvements in their sleep quality (74 %) and that they felt less overwhelmed (72 %) and stressed (69 %) after their children began FFA treatment. Many caregivers also reported improved relationships between the child with DS and their siblings (52 %).

Conclusions: The study found that FFA treatment is associated with meaningful improvement in a large number of QOL domains both for the people with DS who received FFA and their families.

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