Cognitive Debriefing of the Quality-of-Life Inventory – Disability with Caregivers of Patients with Dravet and Lennox-Gastaut Syndromes

Abstract number : 1.353
Submission category : 11. Behavior/Neuropsychology/Language / 11B. Pediatrics
Year : 2023
Submission ID : 470
Source : www.aesnet.org
Presentation date : 12/2/2023 12:00:00 AM
Published date :

Authors :
Presenting Author: J Scott Andrews, PharmD – Takeda Pharmaceutical Company, Ltd

Drishti Shah, PhD – Takeda Pharmaceutical Company, Ltd; Alise Nacson, MPH – Clinical Outcomes Solutions, Folkestone, UK; Sophie Hughes, MSc – Clinical Outcomes Solutions, Folkestone, UK; Arturo Benitez, MD, MBA – Takeda Pharmaceutical Company, Ltd; Tara Symonds, PhD – Clinical Outcomes Solutions, Folkestone, UK; Lara Sams, MSc – Clinical Outcomes Solutions, Folkestone, UK

Rationale: Dravet (DS) and Lennox-Gastaut syndromes (LGS) are rare epileptic encephalopathies characterized by treatment-resistant seizures and intellectual or developmental disability. Patients with DS or LGS often cannot self-report; existing quality of life (QoL) measures for children with these seizure disorders are inadequate and insensitive to meaningful change. The Quality-of-Life Inventory – Disability (QI-Disability) is a reliable and valid caregiver completed measure of QoL across the spectrum of intellectual disability. We explored understanding, relevance, and comprehensiveness of the QI-Disability for DS and LGS via caregiver interviews.

Methods: The QI-Disability comprises six domains: physical health, positive emotions, negative emotions, social interactions, leisure and the outdoors, and independence. Qualitative semi-structured interviews included a concept elicitation element and cognitive debriefing of the QI-Disability items for their relevance in patients with DS and LGS. Participants were recruited via DS and LGS Foundations and interviews conducted between January and March 2022. Participants were caregivers to a patient with a clinical diagnosis of DS or LGS aged 2–21 y (DS) or 2–35 y (LGS) at enrollment. Western Independent Review Board approved the study. Transcripts were analyzed via NVivo using an a priori qualitative analysis plan.

Results: Twenty-one caregivers participated in the concept elicitation study; all were female, parents of an individual with DS (n=10) or LGS (n=11), and aged 33–57 y. In terms of the patients, 13 were male and the age range was 3–24 y. Most caregivers understood all or most items in each domain. All caregivers (n=20; 1 did not complete the debrief) sufficiently understood the QI-Disability instructions and the majority provided positive feedback on the response options (14/15) and recall period (1 month; 15/16). Four caregivers suggested modification to a single sentence in the instructions. The QI-Disability was found to be comprehensive, with only two suggestions made for additional items by two caregivers. Within this study, relevance focused on whether patients encountered problems with the concepts to understand if these could be modified by treatment. Due to this, some items, such as in the positive emotions and social interactions domains, were reported to be of lower relevance since impairments were not always observed. In addition, for some items, relevance differed between the DS and LGS groups.

Conclusions: Overall, caregivers could easily understand the QI-Disability instructions, and could understand and relate the items to their child/dependent with DS or LGS. The measure appeared to be comprehensive, and the response options and recall period were appropriate. Some items were reported to be less relevant to patients with LGS and DS because deficits were not seen or there was an observed ceiling effect which may limit the ability to show improvement within a trial setting.

Funding: Takeda Pharmaceutical Company Ltd.