Survey Suggests Fenfluramine Has Benefits Beyond Seizure Reduction

A survey study (NCT04437004) of people caring for individuals with Dravet syndrome (DS) receiving fenfluramine (Fintepla; Zogenix, Emeryville, CA) treatment suggests fenfluramine treatment may improve more than seizure frequency. Fenfluramine was approved by the Food and Drug Administration (FDA) in 2020 for the treatment of seizures associated with DS in people age 2 and up. According to caregivers surveyed, individuals treated with fenfluramine also experienced improvements in executive functions like cognition, learning, and problem solving, as well as mood, sleep quality, and motor function.

Of those surveyed, 34% cared for adults with DS and 66% cared for children with DS. The mean age of individuals being treated was 15 years (range 2-33 years) and the mean duration of fenfluramine treatment was 21 months (range 5-59 months).

The most common nonseizure-related improvements were:

• cognition (76%) 
• alertness (68%)
• education (65%) 
• problem solving (56%)
• speech (48%)
• sleep quality (47%)
• motor function (46%) 
• mood (41%)

Family benefits reported were:

• Less family stress (76%)
• Improved relationship between DS child and siblings (56%)
• Improved behavior and mood of siblings (58%)
• Easier time for their family to do things with others (64%)

“The impact of DS on the lives of the diagnosed child and the family members who care and worry about their loved one can be severe and unrelenting,” said Mark Jensen, PhD, vice chair for Research in Rehabilitation Medicine at UW Medicine, professor of Rehabilitation Medicine at the University of Washington, and lead author for the study. “Our study shows that treatment with FINTEPLA not only reduces seizure activity, but also results in substantial benefits for many other aspects of the lives of those impacted by DS. The majority of caregivers reported that after treatment began, they felt less overwhelmed, less anxiety, and less stress. They noted improvements in sleep in both their children and themselves and even noticed improvements in the siblings of the child with DS. As they witnessed these improvements, they experienced, for the first time in many years, hope for the future of their child.”

A total of 59 caregivers with a mean age of 48, who were 85% female persons and 88% of whom were living with a partner or spouse participated in the interviews. Discussions were audiotaped and used to generate summaries that were analyzed to identify themes for responses. The numbers and rates of responses in each category were then computed. Caregivers also provided demographic and clinical information about their child with DS and completed a self-report survey about their own health and quality of life.

These data were presented at the virtual 2021 American Academy of Neurology (AAN) Virtual Annual Meeting April 17-22, 2021.