Add-on Fintepla may improve daily functioning in Dravet syndrome: Study
Caregivers noticed improvements in communication, attention, behavior
Written by |
Beyond reducing seizures, add-on treatment with Fintepla (fenfluramine) may improve daily functioning in areas such as communication, attention, and awareness of surroundings in adults and children with Dravet syndrome, especially younger patients and those who engage more frequently in rehabilitation, an Italian study found.
“Caregivers noticed meaningful improvements in communication, attention, alertness, and behavior. These changes were more evident in younger patients and in those receiving regular rehabilitation. Importantly, improvements were reported even when seizures did not markedly decrease,” researchers wrote.
The study, “Beyond seizure control: Functional and caregiver-reported outcomes of long-term fenfluramine treatment in Dravet syndrome,” was published in Epilepsy & Behavior.
More than half of patients experienced reduction in seizures
Dravet syndrome usually begins in early childhood, with symptoms that include frequent seizures, slower development, and problems with communication and attention. UCB’s Fintepla is approved to reduce seizures in patients ages 2 and older. It works by increasing the release of serotonin, a chemical that helps nerve cells communicate. Beyond that, it also involves modulation of other receptors, namely sigma-1 receptors, which may influence attention, behavior, and executive functioning.
In this study, researchers wanted to see whether the benefits of Fintepla extend beyond reducing seizures, based on caregivers’ observations. The study included 21 patients, ages 5 to 31, who were taking Fintepla for a median of three years in addition to their other medications. On average, they were prescribed 3.2 anti-seizure medications.
Most patients (76.2%) received rehabilitation, averaging 2.9 sessions per week, most often speech therapy (38.1%) and occupational therapy (28.6%). Many also had additional issues, such as aggressive behavior (62%). Overall, tests showed severe difficulties in thinking skills and daily functioning.
Over the previous year, more than half of the patients (52.4%) had their seizures reduced by at least 50%. Four (19%) experienced no seizures at all. Others had smaller or no improvements. Over time, response to treatment became less strong, partly due to fewer patients being followed long-term, although more than half of those who were followed still had at least a 50% reduction in seizures after five years.
Combining medication with early and continuous rehabilitative care may help maximize quality of life for individuals with Dravet syndrome and their families.
To assess whether caregivers perceived any functional changes, a 13-item questionnaire was designed to rate daily functioning in Dravet syndrome, based on clinical experience and existing neurodevelopmental clinical tools. Caregivers rated each item on a scale from one (markedly worsened) to five (markedly improved).
Overall, caregivers reported benefits from long-term treatment with Fintepla. Most ratings were above average, especially in expressive language, social communication, environmental alertness (awareness of surroundings), attention, and behavior. Smaller or no changes were observed in school performance, family dynamics, motor coordination, and sleep. Impulsivity and appetite varied.
Younger patients and those receiving more rehabilitation showed greater improvements. Interestingly, these improvements did not always match the benefits observed in seizure reduction. While improvements often occurred alongside a reduction in seizures, even patients who continued to have seizures showed better daily functioning, suggesting that Fintepla “may have broader effects on brain function,” the researchers wrote.
These findings show that, even when standard clinical tools show poor outcomes, patients may still experience real-life improvements. Getting input from caregivers may provide a more complete picture of response to treatment, while “combining medication with early and continuous rehabilitative care may help maximize quality of life for individuals with Dravet syndrome and their families,” the researchers concluded.
