Psychometric evaluation of quality of life measures in a three-arm, randomized, double-blind, placebo-controlled phase III study of everolimus as adjunctive therapy in patients with tuberous sclerosis complex (TSC) who have refractory partial-onset seizures (EXIST-3) (P2.057)

Objective: To examine the psychometric propertiesof three QoL epilepsy measures in TSC. Background: Three age-specific QoL measures were included as secondary endpoints in EXIST-3 (CRAD001M2304), a three-arm, randomized, double-blind, placebo-controlled phase III study to assess theefficacy/safety of two trough ranges of everolimus as adjunctive therapy inpatients with tuberous sclerosis complex (TSC). Design/Methods: Intellectual ability was reportedusing Wechsler Non-Verbal Scale of Ability (WNV). QoL measures included QoL inChildhood Epilepsy (QOLCE) for patients aged ≤10, QoL in Epilepsy Inventory forAdolescents-48 (QOLIE-AD-48) for patients aged 11-≤17, and QoL in EpilepsyInventory-31-Problems (QOLIE-31-P) for patients aged ≥18. A post-hoc analysis examined the psychometric properties of these three QoL instruments (including internal consistency reliability, item-level convergent and discriminant validity and ability to detect change) and generated minimal important difference(MID) values. Results: The majority of patients had intellectual disability (IQ Conclusions: This is the first known evaluation using trial data to determine psychometric properties of these three epilepsy QoL measures for use in TSC. Reliability, validity and ability to detect change were established in this TSC population with refractory seizures, supporting the appropriateness of use of these measures in TSC. Study Supported by: Novartis Pharmaceuticals Corporation Disclosure: Dr. de Vries has received research support from Novartis. Dr. Franz has received personal compensation for activities with Novartis for travel support or honoraria or as a consultant. Dr. Curatolo has received personal compensation for activities with Shire and Novartis as an Advisory Board member. Dr. Nabbout has received personal compensation for activities with Novartis, GW Pharma, Zogenix, Shire, and Eisai. Dr. Berkowitz has received personal compensation for activities with Novartis Pharmaceuticals as an employee. Ms. Neary has received personal compensation for activities with Novartis. Ms. Neary holds stock and/or stock options in Novartis. Dr. Herbst has received personal compensation for activities with Novartis as an employee. Dr. Herbst holds stock and/or stock options in Novartis. Dr. Sully has received personal compensation from Adelphi Values as an employee. Dr. Brohan has received personal compensation for activities with Adelphi Values as an employee. Dr. Lawson has received personal compensation for travel support and honoraria from Novartis.