#AANAM – Caregivers Tie Fintepla to Quality-of-life Improvements

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by Forest Ray PhD |

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Caregivers of children with Dravet syndrome who are taking Fintepla (fenfluramine) report sustained seizure reductions and quality-of-life improvements, among other benefits, according to a recent study.

These findings were presented as a poster titled “Caregivers’ Perspectives on the Long-term Seizure- and Non-seizure-related Benefits of Fenfluramine on Patients with Dravet Syndrome and Their Families,” at the 2021 American Academy of Neurology virtual meeting, held online April 17–22.

“The impact of Dravet syndrome on the lives of the diagnosed child and the family members who care and worry about their loved one can be severe and unrelenting,” Mark Jensen, PhD, the study’s lead author, said in a related press release.

The study surveyed caregivers of children who received Fintepla in either Phase 3 clinical testing or through the U.S. early access program, as well as clinicians who treated those patients.

Participants described their experiences with Fintepla through semi-structured, one-on-one interviews and a self-reported survey of their health and quality of life. Researchers recorded the interviews and analyzed them for common themes.

An earlier interim analysis of an open-label extension study had suggested that Fintepla reduced convulsive seizure frequency for up to three years.

“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 Dravet syndrome,” Jensen stated.

“The majority of caregivers reported that after treatment began, they felt less overwhelmed, less anxiety, and less stress,” he added. “They noted improvements in sleep in both the child and themselves, and even noticed improvements in the siblings of the child with Dravet syndrome. 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 and three clinicians were interviewed. Caregivers had a mean age of 48, 85% were female, 88% lived with their partner or spouse, and 34% provided for adults with Dravet. Dravet patient ages ranged from 2 to 33 years, with a mean age of 15 years. They had been using Fintepla for an average of 21 months.

Overall, 98% of caregivers said they would be “very” or “quite” likely to recommend Fintepla to other families of children with Dravet.

They reported a range of both seizure- and non-seizure-related improvements.

Seizure-related improvements included less seizure activity, fewer seizure triggers, and shorter recovery periods.

The most common non-seizure-related improvements included cognition (76%), alertness (68%), education (65%), problem-solving (56%), speech (48%), sleep quality (47%), motor function (46%), and mood (41%).

Caregivers reported personal relief in terms of feeling less overwhelmed (71%), less stress (66%), and less anxiety and depression (68%). Most also reported better sleep quality (71%) and having more time for activities they enjoy (56%). Close to half (44%) of respondents said the relationship with their spouse or partner improved, and 62% of employed parents said they missed less work.

“Before,” said one respondent, “I would miss work at least one or two days per week because of her status seizure activity. Now we can go eight to nine weeks without me missing a day.”

Benefits appeared to extend to the whole family. These included less overall family stress (76%), better relationships between children with Dravet syndrome and their siblings (56%), and better behavior and/or mood experienced by these siblings (58%). Most participants (64%) said Fintepla treatment translated into having more time as a family to do things with others.

The clinicians interviewed for this study corroborated the caregivers’ responses.

“This qualitative study provides insight directly from caregivers about the real world benefits they observe in their children and family as a result of their child being treated with Fintepla,” said Bradley Galer, MD, chief medical officer of Zogenix, Fintepla’s developer. “These findings further support the data reported in our Phase 3 studies related to seizure and non-seizure outcomes.”