A systematic review of studies that estimated the burden of chronic non-communicable rare diseases using disability-adjusted life years.

Autor: Oliveira CC; Department of Public Health, Erasmus MC University Medical Center, Rotterdam, The Netherlands. c.cruzoliveira@erasmusmc.nl.; Netherlands Institute for Health Sciences, Erasmus University Rotterdam, Rotterdam, The Netherlands. c.cruzoliveira@erasmusmc.nl., Charalampous P; Department of Public Health, Erasmus MC University Medical Center, Rotterdam, The Netherlands., Delaye J; European Organisation for Rare Diseases (EURORDIS), Paris, France., Grad DA; Department of Public Health, Babes-Bolyai University, Cluj-Napoca, Romania., Kolkhir P; Institute of Allergology, Charité - Universitätsmedizin Berlin, Corporate Member of Freie Universität Berlin, Humboldt-Universität zu Berlin, Berlin, Germany.; Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, Allergology and Immunology, Berlin, Germany., Mechili EA; Clinic of Social and Family Medicine, School of Medicine, University of Crete, Crete, Greece.; Department of Healthcare, Faculty of Public Health, University of Vlora, Vlora, Albania., Unim B; Department of Cardiovascular, Endocrine-Metabolic Diseases and Aging, Istituto Superiore Di Sanità, Rome, Italy., Devleesschauwer B; Department of Epidemiology and Public Health, Sciensano, Brussels, Belgium.; Department of Translational Physiology, Infectiology and Public Health, Ghent University, Merelbeke, Belgium., Haagsma JA; Department of Public Health, Erasmus MC University Medical Center, Rotterdam, The Netherlands.
Jazyk: angličtina
Zdroj: Orphanet journal of rare diseases [Orphanet J Rare Dis] 2024 Sep 09; Vol. 19 (1), pp. 333. Date of Electronic Publication: 2024 Sep 09.
DOI: 10.1186/s13023-024-03342-3
Abstrakt: Background: Initiatives aiming to assess the impact of rare diseases on population health might be hampered due to the complexity of disability-adjusted life years (DALYs) estimation. This study aimed to give insight into the epidemiological data sources and methodological approaches used in studies that estimated DALYs for chronic non-communicable rare diseases (CNCRD), and compare its results.
Methods: A literature strategy was developed for peer-review search in Embase and Medline, and also performed on grey literature databases and population health and/or rare disease-focused websites. We included studies that determined the burden of CNCRD listed on the Orphanet's and/or the Genetic and Rare Diseases information center (GARD) websites. We excluded communicable and occupational diseases, rare cancers, and cost-effectiveness/benefit studies. Two researchers independently screened the identified records and extracted data from the final included studies. We used the Guidelines for Accurate and Transparent Health Estimates Reporting (GATHER) statement to assess the quality of reporting of the included studies. The data synthesis depicted the studies' characteristics, their distribution by geographic coverage and the group of disease(s) they focused on, the methods and data input sources used and estimated DALY per case.
Results: In total, 533 titles were screened, and 18 studies were included. These studies covered 19 different CNCRDs, of which most fell in the disease category "Diseases of the nervous system". Diverse methodological approaches and data input sources were observed among burden of CNCRD studies. A wide range of DALY per case was observed across the different studies and diseases included.
Conclusions: A low number of burden of CNCRD studies was observed and most estimates resulted from multi-country studies, underlining the importance of international cooperation to further CNCRD research. This study revealed a lack of epidemiological data and harmonization of methods which hampers comparisons across burden of CNCRD studies.
(© 2024. The Author(s).)
Databáze: MEDLINE
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