Our research, investigating the influence of seizures on the quality of life of patients with Dravet syndrome and their carers, has recently been published in Neurology and Therapy. We conducted statistical analyses using data from fenfluramine trials to explore the relationship between seizure-free days and quality of life. The analysis revealed that seizure-free days are significantly correlated with both patients’ and carers’ quality of life. Findings suggest the direct positive impact of effective antiseizure treatment on the well-being of patients and their carers. This research supports and highlights the need of health technology assessments (HTA) of fenfluramine for patients with Dravet Syndrome.
Carers and wider families of children with life limiting conditions are often significantly impacted by the condition as well as the child, but these indirect impacts can be difficult to measure and to capture in standard health economic evaluations. NICE have recently updated their guidelines to facilitate inclusion of carer quality of life in impact assessments of new treatments. NICE recognise the importance of including wider benefits on carers in assessment of the value of treatments which reduce symptoms in patients.
We wanted to know if we could describe the relationship between seizures and patients’ and carers’ quality of life so that this could be to quantify the effect of treatments to reduce seizures in HTA.
Patients with Dravet Syndrome often suffer from frequent prolonged seizures with cognitive, behavioural, and motor impairment. This substantially impacts the quality of life of patients and their carers. Fenfluramine has recently been approved as an additional treatment option for patients diagnosed with Dravet Syndrome. We used statistical analyses of data collected from trial data of fenfluramine. We focused on seizure free days as being most clearly associated with quality of life. Each additional day without convulsive seizures results in an additional 0.005 improvement in the quality of life for patients and a 0.014 improvement for carers. This highlights the significance of effective antiseizure treatment in improving the well-being of patients and their caregivers.
This work has supported the HTA assessment evidence of fenfluramine which is now recommended by NICE as an additional treatment option in patients who are not well controlled with other medications.
The full paper is now available open access on the Neurology and Therapy website.
Pinsent A, Weston G, Adams EJ, et al. Determining the Relationship Between Seizure-Free Days and Other Predictors of Quality of Life in Patients with Dravet Syndrome and Their Carers from FFA Registration Studies. Neurol Ther. 2023 Jun 16; doi: 10.1007/s40120-023-00510-8.