Nonseizure symptoms and broader seizure impacts in patients with Dravet syndrome and Lennox-Gastaut syndrome in clinical practice settings: Results from a multinational survey.

objectiveTo assess the burden of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS), including managing seizure and nonseizure symptoms, on patients and caregivers.

methodsData were drawn from the Adelphi Real World DS and LGS Disease Specific Programme™, a cross-sectional survey in Asia (China, Japan), Europe (France, Germany, Italy, Spain, United Kingdom), and the United States of America between July 2022 and August 2023. Neurologists/pediatric neurologists reported demographics, clinical characteristics, and nonseizure symptoms for up to 10 consecutively consulting patients. Caregivers provided data on patient nonseizure symptoms, quality of life (QoL), satisfaction with treatment, and caregiver burden. Analyses were descriptive.

resultsPhysicians (n = 259) reported data on 547 patients with DS and 811 with LGS. Caregivers (n = 348) provided data on 157 patients with DS and 191 with LGS. In the previous 6 months, 51% of patients with DS experienced ≥1 seizure-related injury and 61% experienced status epilepticus. For LGS, this was 50% and 43% of patients, respectively. Rates of moderate to very severe impairment in nonseizure symptoms were reported by physicians in learning/intellect (DS 69%; LGS 78%), verbal communication (DS 69%; LGS 71%), severity of developmental delay (DS 65%; LGS 68%), overall mental status (DS 57%; LGS 67%), and nonverbal communication (DS 61%; LGS 64%). Caregivers reported moderate to very severe impairment in learning/intellect (DS 53%; LGS 67%), severity of developmental delay (DS 52%; LGS 56%), verbal communication (DS 48%; LGS 48%), and nonverbal communication (DS 42%; LGS 45%). Caregivers reported nonseizure symptoms moderately to significantly impacted QoL for 56% of patients (DS 49%; LGS 61%); satisfaction with treatment rate was low for control over cognition/memory, verbal communication, and nonverbal communication impairment. SIGNIFICANCE: In this study, considerable burden in DS and LGS management and care was driven by nonseizure symptoms, suggesting a need for treatments that manage the broad spectrum of disease symptoms. PLAIN LANGUAGE SUMMARY: We asked doctors and caregivers to tell us about the symptoms that patients with Dravet syndrome and Lennox-Gastaut syndrome have. We asked how the symptoms affect the lives of the patients as well as the caregivers. We found that seizures have a big impact on the health and well-being of patients and caregivers. We also found that other symptoms not caused by seizures have a big impact on patients and their caregivers.