Prevalence of scleroderma in Spain: An approach for estimating rare disease prevalence using a disease model

Pharmacoepidemiology and Drug Safety

Volumn 17, Issue 11, 2008, Pages 1100-1107

  Villaverde Hueso, A ^a   de la Paz, M Posada ^a   Martin Arribas M C ^a   Sanchez Valle E ^a   Ramirez Gonzalez A ^a   Biairdi, P ^b  

^a INSTITUTO DE SALUD CARLOS III   (Spain)

^b UNIVERSITY OF PAVIA   (Italy)

Author keywords

Epidemiology; Orphan drugs; Prevalence; Rare diseases; Scleroderma

Indexed keywords

ORPHAN DRUG;

ADULT; ARTICLE; COMPUTER PROGRAM; DECISION MAKING; DISEASE DURATION; DISEASE MODEL; FEMALE; GREECE; HUMAN; INCIDENCE; INTERNATIONAL CLASSIFICATION OF DISEASES; MAJOR CLINICAL STUDY; MALE; MORTALITY; ONSET AGE; POISSON DISTRIBUTION; POPULATION RESEARCH; PREVALENCE; PRIORITY JOURNAL; PUBLIC HEALTH; RARE DISEASE; REMISSION; SCLERODERMA; SPAIN; UNITED KINGDOM;

ADULT; AGE DISTRIBUTION; AGED; FEMALE; GREAT BRITAIN; GREECE; HUMANS; INCIDENCE; MALE; MIDDLE AGED; MODELS, THEORETICAL; POISSON DISTRIBUTION; PREVALENCE; SCLERODERMA, SYSTEMIC; SPAIN;

EID: 57649162575     PISSN: 10538569     EISSN: 10991557     Source Type: Journal    
DOI: 10.1002/pds.1660     Document Type: Article

References (31)

1. Rare diseases: understanding this public health priority. Eurordis, 2005; http://www.eurordis.org/ [Accessed 22 May 2007].
2. Orphanet. Enfermedades Raras, Medicamentos Huérfanos. http://www.orpha.net/consor [Accessed 22 May 2007].
3. US Food and Drug Administration (1983) The Orphan Drug Act. http://www.fda.gov/orphan/oda.htm [Accessed 22 May 2007].
4. Commission of the European Communities. Commission staff working document on the experience acquired as a result of the application of regulation (EC) no 141/2000 on orphan medicinal products and account of the public health benefits obtained document on the basis of Article 10 of regulation (EC) no 141/2000. Brussels, 20.6.2006. SEC(2006) 832: 1-32.
5. Posada de la Paz M, Philen RM, Borda AI. Toxic oil syndrome: the perspective after 20 years. Epidemiol Rev 2001; 23: 231-247.
6. Ferri C, Valentín G, Cozzi F, et al. Systemic sclerosis. demographic, clinical, and serologic features and survival in 1. 012 Italian patients. Medicine 2002; 81(2): 139-153.
7. Simeón CP, Armadans L, Fonollosa V, et al. Mortality and prognostic factors in Spanish patients with systemic sclerosis. Rheumatology 2003; 42: 71-75.
8. Allcock RJ, Forrest I, Corris PA, Crook PR, Griffiths ID. A study of the prevalence sclerosis in Northeast England. Rheumatology (Oxford) 2004; 43(5): 596-602.
9. Le Guern V, Mahr A, Mouthon L, Jeanneret D, Carzon M, Guillevin L. Prevalence of systemic sclerosis in a French multi-ethnic county. Rheumatology (Oxford) 2004; 43(9): 1129-1137.
10. Mayes MD, Lacey JV, Beebe-Dimmer J, et al. Prevalence, incidence, survival, and disease characteristics of systemic sclerosis in a large US population. Arthritis Rheum 2003; 48(8): 2246-2255.
11. Geirsson AJ, Steinsson K, Guthmundsson S, Sigurthsson V. Systemic sclerosis in Iceland. A nationwide epidemiological study. Ann Rheum Dis 1994; 53(8): 502-505.
12. Silman AJ, Jannini S, Symmons D, Bacon P. An epidemiological study of scleroderma in the West Midlands. Br J Rheumatol 1988; 27: 286-290.
13. Geirsson AJ,Wollheim FA, Akesson A. Disease severity of 100 patients with systemic sclerosis over a period of 14 years: using a modified Medsger scale. Ann Rheum Dis 2001; 60: 1117-1122.
14. Zurriaga O, Martínez C, Arizo V, et al. Los Registros de Enfermedades en la Investigacín Epidemiológica de las Enfermedades Raras en España. Rev Esp Salud Pública 2006; 80: 249-257.
15. DisMod II. Software developed by J. Barendregt, Department of Public Health, Erasmus University Holland, in collaboration with the WHO. http://www.hsph.harvard.edu/organizations/bdu/DisMod.html [Accessed 22 May 2007].
16. Kruijshaar ME, Barendregt JJ, Hoeymans N. The use of models in the estimation of disease epidemiology. Bull. World Health Organ. 2002; 80: 622-628.
17. Barendregt JJ, van Oortmarssen GJ, Vos T, Murray CJ. A generic model for the assessment of disease epidemiology: the computational basis of DisMod II. Popul Health Metr 2003; 1(1): 4.
18. Kruijshaar ME, Barendregt JJ, van de Poll-Franse LV. Estimating the prevalence of breast cancer using a disease model: data problems and trends. Popul Health Metr 2003; 1: 5.
19. Barendregt JJ, Ott A. Consistency of epidemiologic estimates. Eur J Epidemiol 2005; 20(10): 827-832.
20. Cifras de Población. Revisión del Padrón Municipal 2004. Instituto Nacional de Estadística (INE). http://www.ine.es/inebase/index.html [Accessed 22 May 2007].
21. INE. Fichero de microdatos individuales de defunciones con causa de muerte. Año 2004; Instituto Nacional de Estadística.
22. World Health Organization (WHO). 1992-1994; International Statistical Classification of Diseases and Related Health Problems, (10th revision, ICD-10), Vol 1-3. World Health Organization: Geneva.
23. Alamanos Y, Tsifetaki N, Voulgari PV, et al. Epidemiology of systemic sclerosis in northwest Greece, 1981 to 2002. Semin Arthritis Rheum 2005; 34(5): 714-720.
24. COMP. Public summary of positive opinion for orphan designation of human engineered monoclonal antibody specific for transforming growth factor beta 1 (TGF-beta 1) for the treatment of systemic sclerosis. EMEA/ COMP/74/02 Rev. 1, 2003.
25. Magnant J, de Monte M, Guilmot JL, et al. Relationship between occupational risk factors and severity markers of systemic sclerosis. J Rheumatol 2005; 32(9): 1713-1718.
26. Mérida Férnandez SM, Margolles Martins M. Enfermedades Raras: La Esclerosis Sistémica (Esclerodermia) en Asturias, 1996-2003. Gac Sanit 2005; 19(Suppl. 1): 72.
27. Murray CJL, Lopez AD (eds). The global burden of disease: a comprehensive assessment of mortality and disability form diseases, injuries and risk factors in 1990 and projected to 2020. Global Burden of Disease and Injury. Harvard University Press: Harvard School of Public Health on behalf of WHO, Cambridge, MA, 1996.
28. Cubo E, Álvarez E, Morant C, et al. Burden of disease related to Parkinson's disease in Spain in the year 2000. Mov Disord 2005; 20: 1481-1487.
29. Wild S, Roglic G, Green A, Sicree R, King H. Global prevalence of diabetes: estimates for the year 2000 and projections for 2030. Diabetes Care 2004; 27(5): 1047-1053.
30. Arnett FC, Howard RF, Tan F, et al. Increased prevalence of systemic sclerosis in a Native American tribe in Oklahoma. Association with an Amerindian HLA haplotype. Arthritis Rheum 1996; 39(8): 1362-1370.
31. Silman AJ, Howard Y, Hickl AJ, Black C. Geographical clustering of scleroderma in south and west London. Br J Rheumatol 1990; 29(2): 93-96.