The impact of seizure frequency on quality of life in patients with Lennox-Gastaut syndrome or Dravet syndrome.

OBJECTIVES: Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS) are rare treatment-resistant epileptic encephalopathies with limited data describing the relationship between seizures and quality of life (QoL). The objective of this cross-sectional pilot study was to assess the impact on QoL of seizures and seizure-free days for the generation of utility values. METHODS: Surveys were conducted in the UK and France, whereby patients and/or caregivers of patients with LGS, DS, or other epilepsies were asked to score health state vignettes for a hypothetical patient with LGS or DS. Respondents evaluated QoL for health states based on the number of seizures and seizure-free days per month, using a visual analog scale (VAS). Visual analog scale scores were converted to the 0-1 scale as a proxy estimate for utility values. Surveys were pilot tested and respondents were recruited from October 2018 to August 2019. RESULTS: Patient respondents were mainly treatment-responsive (n = 43/55) whereas caregiver respondents mainly cared for patients with treatment-resistant epilepsy (n = 38/43). Most respondents and patients were aged ≥18 years. Results from LGS and DS surveys in the UK (n = 58) and France (n = 40) suggested that health states with fewer seizures and more seizure-free days had higher QoL scores for hypothetical patients. For DS, QoL scores for patient health states ranged from 0.20 (32 convulsive seizures and 4 seizure-free days/month, UK) to 0.92 (seizure-free, France). For LGS, scores ranged from 0.14 (130 drop seizures and 1 seizure-free day/month, France) to 0.83 (seizure-free, UK). In all surveys, seizure-free days had a greater impact on QoL than seizure frequency (P < 0.001). CONCLUSIONS: Fewer seizures and additional seizure-free days improved QoL in patients with LGS or DS; seizure-free days had the greatest impact on QoL.

Cost-effectiveness of everolimus plus reduced tacrolimus in de novo liver-recipients in the Italian setting.

INTRODUCTION: Long-term exposure to calcineurin inhibitor-based immunosuppressant (IS) therapy in liver transplant (LT) recipients is associated with renal complications. In the randomized trial H2304, everolimus + reduced-dose tacrolimus (EVR + rTAC) demonstrated equivalent efficacy and superior renal function compared to standard-dose tacrolimus. METHODS: To evaluate the cost-effectiveness of EVR + rTAC vs TAC, in de novo LT patients, a Markov model simulating both liver and kidney function was developed and estimated the long-term outcomes of IS following LT. The analysis used the Italian healthcare payer perspective. RESULTS: Patients treated with EVR + rTAC gained on average 1.92 years and 1.62 quality-adjusted life years (QALYs). The incremental cost-effectiveness ratios (ICER) were €35,851 and €42,567 for LY gained and QALY gained, respectively. For the hepatitis-c sub-population, the ICERs decreased to €22,519 and €30,658, respectively. CONCLUSION: EVR + rTAC improves survival and quality-of-life and is a cost-effective alternative to calcineurin-inhibitor monotherapy for patients requiring LT.