USE OF THE CLINICAL PRACTICE RESEARCH DATALINK (CPRD) TO ASSESS ‘REAL-WORLD' MANAGEMENT OF TUBEROUS SCLEROSIS COMPLEX (TSC) IN THE UK
Author(s)
Demuth D*1, Nasuti P1, Lucchese L2, Richards C1, Gray L3, Price L3, Magestro M4 1IMS Health, London, United Kingdom, 2IMS Health, London, UK, London, United Kingdom, 3Novartis Pharmaceuticals UK Limited, Frimley, United Kingdom, 4Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA
OBJECTIVES: TSC is a multi-system genetic disorder associated with benign lesions throughout the body, neurological manifestations, and impaired cognition. As many as 8,000 people in the UK may have TSC; many cases likely go undiagnosed. Long-term morbidity and treatment burden associated with TSC are significant, suggesting substantial economic burden. This preliminary study assessed presentation patterns of select TSC manifestations in CPRD. METHODS: TSC patients were retrospectively identified in CPRD (Read: PK5..00) and linked Hospital Episodes Statistics (HES; ICD-10: Q85.1) databases. CPRD includes over 5 million active patients from UK primary care practices (~8% coverage). 55% of practices (375) are linked to HES, allowing events in secondary care to be analysed (e.g. hospitalisations, procedures). Patients not linked to HES were excluded. Available history was extracted for each patient; descriptive statistics for select TSC-related diagnoses and procedures are presented. All ages reported below are median values.
RESULTS: A total of 244 patients (49% male) with a TSC diagnosis were identified; age at diagnosis was 8 years, with 70% under 18 years. Patient data history was 20 years; 3 and 11 years pre/post initial TSC diagnosis. By age 4, 72% had a record of epilepsy; by 16 years, 9% had a record of subependymal giant-cell astrocytoma (SEGA); by 18 years, 1% had obstructive hydrocephalus; by 43 years, 4% had renal angiomyolipoma (AML) [median age at initial recorded diagnosis]. CONCLUSIONS: Preliminary analyses affirm the utility of CPRD in a real-world study of TSC, and the many emergent TSC-related manifestations, in a longitudinal fashion. Data are suggestive of evolving diagnostic and treatment patterns (30% adults) and may highlight a need for better coordination of adult care. Relatively low prevalence of AML was unexpected and warrants further investigation. Robust analyses are planned to comprehensively describe the clinical and economic burden of TSC in the UK.
Conference/Value in Health Info
2013-11, ISPOR Europe 2013, The Convention Centre Dublin
Value in Health, Vol. 16, No. 7 (November 2013)
Code
PRM40
Topic
Real World Data & Information Systems
Topic Subcategory
Reproducibility & Replicability
Disease
Rare and Orphan Diseases