Real-World Clinical Burden of Non-seizure Symptoms on Patients with Dravet or Lennox-Gastaut Syndromes: Results from a Global Multinational Survey
Abstract number :
1.307
Submission category :
7. Anti-seizure Medications / 7E. Other
Year :
2023
Submission ID :
462
Source :
www.aesnet.org
Presentation date :
12/2/2023 12:00:00 AM
Published date :
Authors :
Presenting Author: Stevie Olsen, BSc – Adelphi Real World, Bollington, UK
Drishti Shah, PhD – Takeda Pharmaceutical Company, Ltd; Vicente Villanueva, MD – Hospital Universitario y Politécnico La Fe, Valencia, Spain; Dennis Dlugos, MD – The Children's Hospital of Philadelphia and University of Pennsylvania School of Medicine, Philadelphia, PA; Arturo Benitez, MD, MBA – Takeda Pharmaceutical Company, Ltd; Jonathan de Courcy, BSc – Adelphi Real World, Bollington, UK; Sophie Lai, BSc – Adelphi Real World, Bollington, UK; Jeannine Roth, MSc Pharm – Takeda Pharmaceutical Company, Ltd; J Scott Andrews, PharmD – Takeda Pharmaceutical Company, Ltd
Rationale:
Dravet (DS) and Lennox-Gastaut (LGS) syndromes are characterized by treatment-resistant seizures and non-seizure symptoms. The burden of seizure management has been described, but there is a lack of comprehensive data on the impact of non-seizure symptoms and broader seizure impacts. This analysis describes the global clinical burden of DS and LGS beyond seizures.
Methods:
This was a retrospective analysis of real-world data from the Adelphi DS and LGS Disease Specific Programme (DSPTM) collected in France, Germany, Italy, Spain, UK, USA, China and Japan from July 2022. Neurologists/pediatric neurologists completed cross-sectional surveys to provide information on patient demographics, seizure history, non-seizure symptoms, and quality of life (QoL). Caregivers of patients with DS or LGS also completed a survey asking about the patient’s non-seizure symptoms and QoL, and satisfaction with treatment. Interim data (7 April 2023) from France and Japan were used; all other data were final.
Results:
Physicians (n=259) reported data on 617 patients with DS (mean [SD] age 9.7 [8.4] y) and 803 with LGS (14.7 [11.1] y) who received some treatment for DS or LGS. The most common type of seizure was myoclonic (49%) in patients with DS and tonic for those with LGS (63%). In the previous 6 months, patients with DS with ≥ 1 seizure injury (n=317) had a mean (SD) of 4.9 (6.2) seizure injuries (median: 3.0) while patients with LGS (n=407) had a mean of 4.8 (6.3) injuries (median 3.0). In the previous 6 months 54% of patients with DS and 39% with LGS had status epilepticus. Overall, among patients with DS and LGS, the most common physician-reported non-seizure related impairments were in verbal communication (95%; 69% moderate–very severe), learning/intellect (95%; 73% moderate–very severe) and non-verbal communication (93%; 61% moderate–very severe (Tables). Physicians rated daytime seizures and mental impairment as having the highest impact on patient QoL. Caregivers (n=357, mean [SD] age 39.9 [10.5] y) also reported that non-seizure symptoms moderately or significantly impacted QoL for 54% (188/348) of patients (DS: 82/174, 47%; LGS: 106/174, 61%). Caregiver satisfaction with treatment of DS and LGS was lowest (0–2 where 0 is least and 4 highest satisfaction) for control over cognition and memory (64%; 226/355), verbal communication (60%; 211/354) and non-verbal communication impairment (56%; 201/357). Of note, treatment satisfaction rates for control over non-seizure symptoms were lowest for caregivers located in the USA (n=30), especially for sleep disturbances (97% scored 0–2).
Conclusions:
Reported rates of non-seizure impairment were high and non-seizure symptoms affected patient QoL in more than half of the patients according to their caregivers. Based on these data from real-world practice settings, considerable burden in DS and LGS management and care is driven by non-seizure symptoms suggesting a need for treatments that enable better non-seizure symptom management and control.
Funding:
Analysis funded by Takeda Pharmaceutical Company Ltd.
Anti-seizure Medications