Real-World Evidence of the Impact of Dravet Syndrome and Lennox-Gastaut Syndrome on Patient and Caregiver Quality of Life
Author(s)
Lin J1, Kothare SV2, Salpekar JA3, Connolly H4, Gillespie A4, Vyas K5, Simontacchi K1, Thomas SM1, Viswanathan HN1, Chin R6
1Jazz Pharmaceuticals, Inc., Palo Alto, CA, USA, 2Cohen Children’s Medical Center, Lake Success, NY, USA, 3Kennedy Krieger Institute, Baltimore, MD, USA, 4Adelphi Real World, Bollington, UK, 5Jazz Pharmaceuticals UK Ltd., London, UK, 6The University of Edinburgh, Edinburgh, UK
Presentation Documents
OBJECTIVES: To describe the disease burden experienced by patients with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS), and the impact on health-related quality of life (HRQoL) of their caregivers.
METHODS: The Adelphi DS & LGS Disease Specific Programme™ was a cross-sectional multi-country physician and caregiver survey, conducted between July 2022–August 2023. This analysis comprises descriptive statistics of caregiver-reported demographics (patients and caregivers) and HRQoL.
RESULTS: Patients’ (DS: 146; LGS: 181) median (IQR) age was 6 (4.0–8.0) years (DS: 5.0 [3.0–8.0]; LGS: 6.0 [4.0–8.0]); 65% were male. Moderate-to-very severe physical and mental impairment were reported in 44% and 41% of patients, respectively. Mean (SD) number of caregivers per patient was 1.7 (0.7). Median (IQR) age of caregivers, where available, was 37.0 (32.0–44.0) years (DS: 35.0 [31.0–40.0], n=144; LGS: 37.0 [33.0–46.0], n=179); 80% were female. The top activity of daily living requiring caregiver assistance was patients taking medications (93%). Most caregivers reported managing seizures as the most difficult aspect of DS and LGS (60%). Owing to patient care, 11% reported unemployment; 62% of employed caregivers reduced their working hours. Within the 4 weeks before survey, 54% reported finding time for their own needs/interests difficult because of patient care; 80% and 71% reported sometimes, very often, or always experiencing impact on other family/household responsibilities and disrupted sleep, respectively. Since caring for the patient, 56% of caregivers reported developing health conditions, including insomnia/sleep disorders (24%) and anxiety (22%).
CONCLUSIONS: A large proportion of patients with DS or LGS have physical and mental impairments. Caring for patients with DS or LGS impacts caregiver work status, family responsibilities, and personal health. Data presented herein demonstrate disease burden and associated negative impact on caregiver HRQoL, highlighting the unmet need for support in the management of patients with DS or LGS.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 12, S2 (December 2024)
Code
PCR38
Topic
Patient-Centered Research, Study Approaches
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes, Surveys & Expert Panels
Disease
Neurological Disorders, Pediatrics, Rare & Orphan Diseases