Patient characteristics and treatment patterns in Dravet syndrome and Lennox-Gastaut syndrome: real-world evidence from physician- and caregiver-reported data in Japan

Background Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) are rare, severe childhood-onset developmental and epileptic encephalopathies associated with treatment-resistant seizures and significant cognitive, behavioral, and motor impairments, all of which require extensive support from caregivers. Objectives To describe real-world patient characteristics and treatment patterns in those with DS or LGS, and assess caregiver burden in Japan. Methods Data for patients in Japan were analyzed descriptively from the Adelphi DS and LGS Disease Specific Programme™, a cross-sectional linked survey of physicians, patients, and caregivers (November 2022–August 2023). Results Thirty-four physicians provided data for 81 patients (DS = 31; LGS = 50). Within the 4 weeks prior to data collection, the most common seizure types reported for patients with DS were generalized tonic-clonic (35%) and tonic (32%), and for patients with LGS were tonic (68%) and atypical absence (22%). In both DS and LGS, cognitive and language impairments were reported to be prevalent, and valproate and carbamazepine were reported to be the most common first-line treatments before and after diagnosis. Current treatment satisfaction among responding physicians was reported to be moderate (somewhat satisfied: DS 56%; LGS 45%). Caregivers (n = 25) reported that managing seizures was the most difficult aspect of care for patients with DS or LGS. Moreover, many reported an impact of caregiving on their own health. Conclusions Patients with DS or LGS in Japan are reported to experience substantial seizure and non-seizure burden, and perceived caregiver burden is high. These data underscore the importance of optimized therapies and comprehensive support systems to address both patient and caregiver needs.