Epidemiology of transthyretin familial amyloid

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where the majority of dispenses occurred was used as a proxy for residence. ... The geographical information system QGIS 2.10.1 Pisa was used to depict ...

Epidemiology of transthyretin familial amyloid polyneuropathy in Portugal Mónica Inês1, Teresa Coelho2, Isabel Conceição1,3, Filipa Duarte-Ramos4, Mamede de Carvalho1,3, João Costa5,6,7 1 Translational and Clinical Physiology Unit, Instituto de Medicina Molecular, Lisbon, Portugal. 2 Unidade Corino de Andrade and Neurophysiology Service, Department of Neurosciences, Hospital de Santo António - CHP, Porto, Portugal. 3 Department of Neurosciences, Hospital de Santa Maria-CHLN, Lisbon, Portugal. 4 Social Pharmacy Department, Faculty of Pharmacy, University of Lisbon, Lisbon, Portugal. 5 Clinical Pharmacology Unit, Instituto de Medicina Molecular, Lisbon, Portugal. 6 Laboratory of Clinical Pharmacology and Therapeutics, Faculty of Medicine, University of Lisbon, Lisbon, Portugal. 7 Center for Evidence Based-Medicine, Faculty of Medicine, L29 University of Lisbon, Lisbon, Portugal.

Objectives ● The main objective of this study is to generate Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) epidemiological data for Portugal, the country with the largest patient’s cluster worldwide.

Methods ● In Portugal, TTR-FAP symptomatic patient’s medicines utilization is fully reimbursed by National Health Service (NHS) since 20011. ● Since March 2013 Portuguese electronic prescription system2 became more generalized, allowing central monitoring and validation of medicines prescription and dispensing. ● TTR-FAP de-identified patient’s electronic prescription data, provided from Administração Central do Sistema Saúde (ACSS, I.P.)3 was analyzed. ● Every prescription has information regarding the local where medication was dispensed. For each patient, the municipality where the majority of dispenses occurred was used as a proxy for residence. ● Portuguese mainland population in year 2014 was obtained from the National Statistics Institute4 and it was used to estimate prevalence and incidence. ● In European Union countries, any disease affecting less than 5 people in 10 000 inhabitants is considered rare5, equivalent to less than 50 cases when reported by 100 000 inhabitants. ● Number of new TTR-FAP cases diagnosed during year 2014 was collected from the two Portuguese TTR-FAP medical referral centers6 located at Lisbon and Porto. ● The geographical information system QGIS 2.10.1 Pisa was used to depict TTR-FAP prevalence data in the Portuguese map based on the 2014 CAOP (Carta Administrativa Oficial de Portugal)7 territorial municipalities limits.

Results Prevalence ● Trough year 2014, a total of 70 286 medicines electronic dispensing acts were extracted from ACSS I.P. database, corresponding to a total of 2013 individuals recipients of NHS TTRFAP full reimbursement , including liver transplant patients. ● TTR-FAP prevalence of 20,40 patients per 100 000 inhabitants was estimated based on a country mainland population of 9 869 783 inhabitants (2014). ● The disease is currently spread across 162 (58%) of the 278 Portuguese mainland municipalities, with a peculiar geographical dispersion. ● In 19 municipalities TTR-FAP affects more than 50 per 100 000 inhabitants, being not a rare disease in these municipalities. ● Municipalities with higher TTR-FAP prevalence per 100 000 inhabitants are Póvoa de Varzim, Pampilhosa da Serra and Seia followed by Esposende, Vila do Conde, Figueira da Foz, Amares, Boticas and Barcelos. ● Póvoa de Varzim and Vila do Conde conjoint prevalence in year 1991 was estimated as 90,2 patients per 100 000 inhabitants8. This geographic area has currently a TTR-FAP prevalence of 163,12 patients per 100 000 inhabitants, a 81% increase during the last 23 years. Incidence ● The two Portuguese centers reported a total of 138 new TTR-FAP cases during year 2014, being 63 (46%) new diagnosed symptomatic patients and 75 (54%) new asymptomatic disease carriers. ● A TTR-FAP symptomatic incidence of 0,64 patients and 0,76 carriers per 100 000 inhabitants was estimated.

Conclusion

● This study generates new and relevant data that confirms Portugal as the country with the greatest worldwide TTR-FAP prevalence. ● TTR-FAP disease has a prevalence of 20,40 and an incidence of 0,64 patients per 100 000 inhabitants in Portugal. ● The disease affects in 2014 more than half of the Portuguese municipalities and as compared with 1991 geographical distribution8 we can conclude that is more disseminated across the country, with more municipalities affected in South Portugal area and in Serra da Estrela – Figueira da Foz vector area. ● Póvoa de Varzim and Vila do Conde area historically persists as the most impacted area with a prevalence eight times higher than overall country prevalence that has increased 81% in the last two decades.

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Figure 1. Portugal TTR-FAP 2014 prevalence (by municipality) and incidence Acknowledgements We acknowledge ACSS I.P. for access to the electronic prescription database, Isaura Vieira for support with prescription data questions, Rui Alves for support with the QGIS data integration and Margarida Fernandes, Vanessa Gomes da Costa and Carla Rodrigues for support with Portuguese TTR-FAP center's data.

Declaration of interests No funding or sponsorship was received for this study. This research was conducted under the doctoral programme of the Lisbon Academic Medical Centre (CAML). Mónica Inês is a Pfizer employee at the time this study was conducted.

References 1. Ministerial order 4521/2001, January 31; 2. Ministerial order 4322/2013, March 25; 3. www.acss.min-saude.pt [data on file]; 4. www.ine.pt [accessed 21 Jun 2015]; 5. http://www.who.int/bulletin/volumes/90/6/12-020612/en/; 6. Administrative rule 194/2014, September 30; 7. http://www.dgterritorio.pt [accessed 1 Oct 2015]; 8. Sousa A, et al. Am J Med Genet 1995;60:512–21.

ISPOR 18th Annual European Congress, Milan, November 2015

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