TY - JOUR
T1 - Burden of disease of spinal muscular atrophy linked to chromosome 5q (5q-SMA) in Colombia
AU - Gil-Rojas, Yaneth
AU - Suárez-Obando, Fernando
AU - Amaya-Granados, Devi
AU - Prieto-Pinto, Laura
AU - Samacá-Samacá, Daniel
AU - Ortiz, Blair
AU - Hernández, Fabián
N1 - Publisher Copyright:
© 2023 Informa UK Limited, trading as Taylor & Francis Group.
PY - 2023
Y1 - 2023
N2 - 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.
AB - 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.
KW - Burden of disease
KW - Colombia
KW - disability weights
KW - disability-adjusted life years (DALYs)
KW - spinal muscular atrophy linked to chromosome 5q
UR - http://www.scopus.com/inward/record.url?scp=85158841745&partnerID=8YFLogxK
U2 - 10.1080/14737167.2023.2206569
DO - 10.1080/14737167.2023.2206569
M3 - Article
C2 - 37096565
AN - SCOPUS:85158841745
SN - 1473-7167
VL - 23
SP - 671
EP - 682
JO - Expert Review of Pharmacoeconomics and Outcomes Research
JF - Expert Review of Pharmacoeconomics and Outcomes Research
IS - 6
ER -
