| アブストラクト | INTRODUCTION: Epilepsy 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. METHODS: Patients 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. RESULTS: In 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 ( pound14 335 vs pound4448), which significantly increased with each additional primary manifestation (p<0.0001). CONCLUSIONS: Patients 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. |
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| ジャーナル名 | BMJ open |
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| 投稿日 | 2017/10/7 |
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| 投稿者 | Shepherd, Charles; Koepp, Matthias; Myland, Melissa; Patel, Keyur; Miglio, Cristiana; Siva, Vathani; Gray, Elizabeth; Neary, Maureen |
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| 組織名 | Neurology, Nobles Hospital, Braddan, Isle of Man.;National Hospital for Neurology and Neurosurgery, University College London;Hospital, London, UK.;QuintilesIMS, RWES, London, UK.;Novartis Pharmaceuticals UK Ltd, Camberley, UK.;Novartis Pharmaceuticals Corp, East Hanover, New Jersey, USA. |
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| Pubmed リンク | https://www.ncbi.nlm.nih.gov/pubmed/28982809/ |
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