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Burden of disease of spinal muscular atrophy linked to chromosome 5q (5q-SMA) in Colombia.
Gil-Rojas, Yaneth; Suárez-Obando, Fernando; Amaya-Granados, Devi; Prieto-Pinto, Laura; Samacá-Samacá, Daniel; Ortiz, Blair; Hernández, Fabián.
Afiliação
  • Gil-Rojas Y; Real World Insights (RWI), IQVIA, Bogotá, Colombia.
  • Suárez-Obando F; Instituto de Genética Humana, School of Medicine, Pontificia Universidad Javeriana, Bogotá, Colombia.
  • Amaya-Granados D; Real World Insights (RWI), IQVIA, Bogotá, Colombia.
  • Prieto-Pinto L; Evidence Generation, Roche, Bogotá, Colombia.
  • Samacá-Samacá D; Real World Insights (RWI), IQVIA, Bogotá, Colombia.
  • Ortiz B; Universidad de Antioquia, Hospital San Vicente Fundación, Medellín, Colombia.
  • Hernández F; Real World Insights (RWI), IQVIA, Bogotá, Colombia.
Article em En | MEDLINE | ID: mdl-37096565
OBJECTIVE: This article estimates the disease burden of 5q-SMA in Colombia by using the disability-adjusted life years (DALYs) metric. METHODS: Epidemiological data were obtained from local databases and medical literature and were adjusted in the DisMod II tool. DALYs were obtained by adding years of life lost due to premature death (YLL) and years lived with disability (YLD). RESULTS: The modeled prevalence of 5q-SMA in Colombia was 0.74 per 100,000 population. The fatality rate for all types was 14.1%. The disease burden of 5q-SMA was estimated at 4,421 DALYs (8.6 DALYs/100,000), corresponding to 4,214 (95.3%) YLLs and 207 (4.7%) YLDs. Most of the DALYs were accounted in the 2-17 age group. Of the total burden, 78% correspond to SMA type 1, 18% to type 2, and 4% to type 3. CONCLUSIONS: Although 5q-SMA is a rare disease, it is linked to a significant disease burden due to premature mortality and severe sequelae. The estimates shown in this article are important inputs to inform public policy decisions on how to ensure adequate health service provision for patients with 5q-SMA.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Efeitos Psicossociais da Doença / Mortalidade Prematura Tipo de estudo: Prognostic_studies / Risk_factors_studies Aspecto: Patient_preference Limite: Humans País/Região como assunto: America do sul / Colombia Idioma: En Revista: Expert Rev Pharmacoecon Outcomes Res Assunto da revista: FARMACOLOGIA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Colômbia País de publicação: Reino Unido

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Efeitos Psicossociais da Doença / Mortalidade Prematura Tipo de estudo: Prognostic_studies / Risk_factors_studies Aspecto: Patient_preference Limite: Humans País/Região como assunto: America do sul / Colombia Idioma: En Revista: Expert Rev Pharmacoecon Outcomes Res Assunto da revista: FARMACOLOGIA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Colômbia País de publicação: Reino Unido