Health utilities and quality of life in individuals with tuberous sclerosis complex (TSC) who experience epileptic seizures: A web-based survey Theo Tritton a, ⁎, Bryan Bennett a , Elaine Brohan a , Laura Grant a , Alison Cooper b , Carla Fladrowski c , Cathy Evanochko d , Maureen Neary e , João Vieira f , Viviana Buffa f , Chris Kingswood g a Adelphi Values, Adelphi Mill, Bollington, Cheshire SK10 5JB, United Kingdom b Tuberous Sclerosis Association, CAN Mezzanine 32-36 Loman Street, London SE1 0EH, United Kingdom c European Tuberous Sclerosis Association, In den Birken, 30, 45711 Dattein, Germany d Tuberous Sclerosis Canada Sclérose Tubéreuse, 35057 Essa Road RO, Barrie, ON L4N 5Z2, Canada e Novartis Pharmaceuticals Corporation, One Health Plaza, East Hanover, NJ 07936-1080, United States f Novartis Pharmaceuticals UK Limited, 200 Frimley Business Park, GB-Frimley/Camberley, Surrey GU16 7SR, United Kingdom g St George's Hospital, Blackshaw Road, London SW17 0QT, United Kingdom abstract article info Article history: Received 18 September 2018 Revised 18 October 2018 Accepted 19 November 2018 Available online xxxx Objectives: Individuals with tuberous sclerosis complex (TSC) experience a wide range of health impacts, in- cluding epileptic seizures, negatively impacting their health-related quality of life (HRQoL). Health state utility values (HSUVs) are index values representing HRQoL and are used as key inputs for health economic analyses. Such data are currently very limited in the TSC population. The objective of this study was to generate HSUVs for TSC health states, defined by the number and type of seizures experienced in the previous week, and to com- pare with UK normative values. Methods: This cross-sectional study involved 186 participants (individuals with TSC = 61, caregivers reporting for individuals with TSC = 125) from Europe and North America who completed a web-based survey. Partici- pants completed the [EuroQol - 5 dimensions - 3 levels] (self-report version for individuals with TSC or proxy ver- sion 1 for caregivers). Results: The mean age of individuals with TSC was 27.3 years (self-reported: 41.3 years, caregiver-reported: 20.5 years); 56% were males. Most individuals with TSC (71%) reported experiencing between one and ten seizures in the week prior to participating in the study. The most frequently reported type of seizure was focal: simple partial (50%). Across all participants (combined self-report and caregiver-report), the mean HSUV was 0.474 (95% confidence interval [CI]: 0.424–0.524), significantly lower than the UK norm (0.856, 95%CI: 0.848–0.864) [1]. Mean HSUV and HRQoL scores were consistently lower when reported by caregivers than when self-reported by individuals with TSC (HSUV = 0.351 vs. 0.727). This is in part because caregivers reported for individuals with TSC who experienced more frequent and severe seizures than those who were able to self- report. HSUVs incrementally decreased with the experience of more frequent (1–5 per week: HSUV = 0.666 vs. N 20: HSUV = 0.290) and more severe seizures (focal: simple partial: HSUV = 0.450 vs. generalized: convulsive: HSUV = 0.194). Conclusions: The HRQoL and HSUV index scores indicate substantial impairment among individuals with TSC; HSUVs were shown to decrease considerably with increases in seizure frequency or seizure severity, indicating that more burdensome seizure health states are associated with poorer HRQoL. © 2018 Elsevier Inc. All rights reserved. Keywords: TSC (Tuberous Sclerosis Complex) QoL (Quality of Life) Utilities Seizures Web-based survey 1. Introduction Tuberous Sclerosis Complex (TSC) is a rare genetic condition charac- terized by the growth of benign tumors in various organs of the body [1]; TSC occurs in between 1 in 5000 and 1 in 10,000 live births [2–6]. No single feature of TSC is considered definitively diagnostic [7], indeed individuals with TSC experience a wide range of health impacts that in- clude epileptic seizures, learning difficulties, and behavioral problems [8]. The majority of TSC cases are diagnosed during childhood [9]. Epileptic seizures occur in 85% of patients with TSC [10] and are con- sidered one of the most impactful symptoms on the health-related qual- ity of life (HRQoL) of individuals with TSC [11]. Furthermore, incidents of epileptic seizures are likely to contribute to developmental delay among individuals with TSC [12]. A higher burden of TSC-associated epilepsy is Epilepsy & Behavior 92 (2019) 213–220 ⁎ Corresponding author at: Adelphi Values, Adelphi Mill, Grimshaw Lane, Bollington, Cheshire, United Kingdom. E-mail address: theo.tritton@adelphivalues.com (T. Tritton). https://doi.org/10.1016/j.yebeh.2018.11.021 1525-5050/© 2018 Elsevier Inc. All rights reserved. Contents lists available at ScienceDirect Epilepsy & Behavior journal homepage: www.elsevier.com/locate/yebeh