PREVALENCE OF TRANSTHYRETIN FAMILIAL AMYLOID POLYNEUROPATHY IN PORTUGAL

Author(s)

Inês M1, Coelho T2, Conceição I3, Duarte-Ramos F4, Carvalho M3, Costa J5
1Instituto de Medicina Molecular, Lisboa, Portugal, 2Unidade Clinica de Paramiloidose, Hospital de Santo Antonio, Porto, Portugal, 3Centro Hospitalar de Lisboa Norte, Lisbon, Portugal, 4Faculty of Pharmacy Lisbon University, Lisbon, Portugal, 5Institute of Molecular Medicine, Lisbon, Portugal

OBJECTIVES: Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) is a rare, progressive, debilitating and life-threatening neurodegenerative disease. Although a rare disease, Portugal has one of the largest TTR-FAP foci in the world but recent Portuguese epidemiological data is lacking. The purpose of this study was to estimate TTR-FAP prevalence in Portugal. METHODS: TTR-FAP patient’s medicines utilization is fully funded by National Health System in Portugal since 2001. Since March 2013 Portuguese electronic prescription system became more generalized, allowing central monitoring and validation of medicines prescription and dispensing. TTR-FAP anonymized patient’s data was requested to Administração Central do Sistema Saúde (ACSS). The most frequent municipality used for medicines dispensing by each patient was identified and used as a proxy for residence. Portuguese population by municipality was obtained from Instituto Nacional Estatística

Conference/Value in Health Info

2015-11, ISPOR Europe 2015, Milan, Italy

Value in Health, Vol. 18, No. 7 (November 2015)

Code

PSY21

Topic

Epidemiology & Public Health

Disease

Rare and Orphan Diseases

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