IntroductionEpilepsy is highly prevalent in tuberous sclerosis complex (TSC), a multi-system genetic disorder. The clinical and economic burden of this condition is expected to be substantial due to treatment challenges, debilitating co-morbidities and the relationship between TSC-related manifestations. This study estimated healthcare resource utilisation (HCRU) and costs for patients with TSC with epilepsy (TSC+E) in the UK.MethodsPatients with TSC+E in the Clinical Practice Research Datalink (CPRD) linked to Hospital Episodes Statistics were identified from April 1997 to March 2012. Clinical data were extracted over the entire history, and costs were reported over the most recent 3-year period. HCRU was compared with a matched Comparator cohort, and the key cost drivers were identified by regression modelling.ResultsIn total, 209 patients with TSC+E were identified, of which 40% recorded ≥2 other primary organ system manifestations and 42% had learning disability. Treatment with ≥2 concomitant antiepileptic drugs (AEDs) was prevalent (60%), potentially suggesting refractory epilepsy. Notwithstanding, many patients with TSC+E (12%) had no record of AED use in their entire history, which may indicate undertreatment for these patients.Brain surgery was recorded in 12% of patients. Routine electroencephalography and MRI were infrequently performed (30% of patients), yet general practitioner visits, hospitalisations and outpatient visits were more frequent in patients with TSC+E than the Comparator. This translated to threefold higher clinical costs (£14 335 vs £4448), which significantly increased with each additional primary manifestation (p<0.0001).ConclusionsPatients with TSC+E have increased HCRU compared with the general CPRD population, likely related to manifestations in several organ systems, substantial cognitive impairment and severe epilepsy, which is challenging to treat and may be intractable. Disease surveillance and testing appears to be inadequate with few treatments trialled.Multidisciplinary care in TSC clinics with specialist neurologist input may alleviate some of the morbidity of patients, but more innovative treatment and management options should be sought.

Background: Tuberous sclerosis complex (TSC) is a multi-system genetic disorder characterized by the development of diverse clinical manifestations. The complexity of this disease is likely to result in substantial challenges and costs in disease management throughout the patient's lifetime. This retrospective database study aims to quantify healthcare resources utilized by TSC patients. Methods: TSC patients in the Clinical Practice Research Datalink linked to the Hospital Episodes Statistics database were identified between January 1987 and June 2013. Analyses were conducted over the most recent 3-year period of data and stratified by pediatric (< 18) and adult patients. Prescriptions, procedures, diagnostic tests, and healthcare encounters were reported in comparison with a matched comparator cohort. Costs and key economic drivers by primary organ system manifestations were also examined. Results: A total of 286 patients with TSC were identified and consistently reported 2-fold greater resource use than the matched presumably healthy controls. Despite this comparatively greater resource use, half of TSC patients did not record any procedures, and 20% of patients did not record any diagnostic tests; however, inpatient hospitalizations were greater for the TSC cohort (3.1 vs 1.3), but length of stay was comparable. TSC patients had costs totaling £12,681 per patient over the 3-year period, a figure 2.7-fold greater than the total costs in the comparator cohort (£4,777). Costs for patients with specific primary manifestations were even greater, with brain manifestations incurring £22,139 per affected patient. Kidney and nervous system manifestations were the main cost drivers. Conclusions: The economic burden of TSC and its impact on NHS healthcare resources is mostly attributable to the broad spectrum of manifestations that develop within multiple organ systems. TSC patients may benefit from co-ordinated care based on their requirement for high numbers of healthcare visits across specialties.

Objective: This retrospective database analysis complements previous research to understand treatment patterns for German patients newly-initiating or switching to subsequent GLP-1 RAs. Methods: Adult patients (!18 years) initiating GLP-1 RA (Cohort 1 [C1]) or switching from a previous GLP-1 RA (Cohort 2 [C2]) to exenatide twice-daily (exBID), exenatide once-weekly (exQW), dulaglutide (DULA), or liraglutide (LIRA) were included in this analysis using IQVIA LRx from January 1, 2014-March 31, 2017. Patients were required to have !1 oral anti-hyperglycemic prescription during the 6-month pre-index period and !12 months follow-up. Persistence and treatment modifications were assessed within and beyond 12 months follow-up. Average daily/weekly dosage (ADD/AWD) was calculated during persistence. Results: C1 included 13,417 patients, while C2 included 4,264 patients. Mean ± standard deviation (SD) age was similar (57.7 ± 11.1 years [C1], 58.9 ± 10.1 years [C2]). Most patients using DULA in C2 had switched from LIRA (56.6%). For C1, mean ADD for LIRA was 1.41 ± 0.10 mg, slightly higher in C2, and increased over time. ADD for exBID was 16.9 ± 1.0 mcg, slightly greater in C2. AWD was 2.00 ± 0.05 mg for exQW users and 1.42 ± 0.03 mg for DULA users in C1, similar to C2. For C1, 27.0% exBID, 35.3% exQW, 50.9% DULA, and 48.1% LIRA users remained persistent at 12 months. Patients using DULA had a higher probability of remaining persistent over time (Kaplan-Meier) for both cohorts. Conclusions: Patients using DULA had the highest probability of remaining persistent over time, followed by LIRA. ADD/AWD for DULA, exQW, and exBID were aligned with the recommended combination therapy dose; LIRA ADD suggests some patients use the 1.8 mg dose.

Introduction: Maintenance treatment strategies in COPD recommend inhaled corticosteroid (ICS) ? long-acting muscarinic antagonist (LAMA) ? long-acting b 2-agonist (LABA) triple therapy after initial dual therapy. Little is known about how treatment pathways to triple therapy vary across countries in clinical practice. Methods: This multi-country, retrospective cohort study (conducted 1 January 2005-1 May 2016) included patients with a COPD diagnosis, and (UK only) evidence of smoking history, or (France, Italy, Germany, and Australia) an indicator confirming COPD diagnosis, a first instance of triple therapy recorded during the study period and C 12 months of data prior to this date. Treatment pathways to triple therapy were analyzed in patients whose first instance of triple therapy was on or after the initial COPD diagnosis. The proportion of patients who initiated triple therapy prior to initial COPD diagnosis was also estimated. Meta-analyses of the main results were performed. Results: In 130,729 patients across all countries, mean age (standard deviation) ranged from 63.4 (10.4) years (Germany) to 69.8 (9.9) years (Italy), and median time (interquartile range) from initial COPD diagnosis to first prescription of triple therapy ranged from 16.9 (5.7-36.2) months (Australia) to 42.5 (13.9-87.4) months (UK). ICS ? LABA was the most common treatment pathway prior to triple therapy in the UK, Germany, and Italy (27.3%-31.6%); no previous maintenance therapy prior to triple therapy was the most common pathway in France and Australia (32.5% and 37.9%, respectively). Meta-analyses provided a pooled estimate of 20.4% (95% confidence interval: 13.8%-29.1%) for the proportion of patients initiating triple therapy at or before initial COPD diagnosis. Conclusions: In this retrospective cohort study, treatment pathways to triple therapy were

Background: Tuberous sclerosis complex (TSC) is a multi-system genetic disorder in which renal manifestations occur in 50% of children and 80% of adults. Since these often present alongside other manifestations, renal TSC is likely to incur significant costs. This study aims to quantify healthcare resource use (HCRU) and costs for renal TSC patients in the UK. Methods: TSC patients in the Clinical Practice Research Datalink (CPRD) linked to Hospital Episodes Statistics were identified from January 1987-June 2013. Clinical data were extracted over the entire history and costs were reported over the most recent 3-year period. HCRU was compared with a matched comparator cohort. Incremental costs were reported and the key cost drivers by primary manifestation category were identified by regression modeling. Results: A total of 79 renal TSC patients were identified with manifestations including chronic kidney disease stage 3-5 (with prevalence increasing with age) and renal angiomyolipoma. Renal TSC patients consistently reported greater HCRU than the comparator. Inpatient hospitalizations were more frequent for renal TSC patients (3.2 vs 1.6), but length of stay was comparable; however, 70.9% of renal TSC patients recorded no kidney-related procedures ever and averaged <1 test per year in the 3-year period. Average costs for renal TSC patients were nearly 3-fold greater than the comparator (£15,162 vs £5672). Costs increased with additional manifestation categories (£3600: only renal; £27,531: renal with 4 additional manifestation categories [25% of patients]). Additional nervous system and dermatology/ psychiatric manifestations significantly (p < 0.028) affected costs. Conclusions: Renal TSC patients have greater HCRU than the general CPRD population, likely to result from progression of renal disease and additional manifestations; however, surveillance for disease progression appears to be deficient. Inadequate monitoring may contribute to a lack of co-ordinated care and increased healthcare-associated costs. Efforts should be made to follow the TSC guidelines to effectively monitor and treat patients.