ArticleOrphanet journal of rare diseases2021
Quality of life and its contributors among adults with late-onset Pompe disease in China.
Article in Orphanet journal of rare diseases, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
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Who cites it
11 citing papers in PubMed, 21 citations in OpenAlex.
- Quality of life and its contributors among patients with homozygous familial hypercholesterolemia in China.Frontiers in public health · 2025Article
- Exploring Quality of Life in Adults Living With Late-onset Pompe Disease: A Combined Quantitative and Qualitative Analysis of Patient Perceptions from Australia, France, Italy, and the Netherlands.Journal of health economics and outcomes research · 2025Article
- Measurement properties of the EQ-5D-3L, EQ-5D-5L, and SF-6Dv2 in patients with late-onset Pompe disease.The European journal of health economics : HEPAC : health economics in prevention and care · 2024Article
- Article
- Measuring health related quality of life (HRQoL) in Lysosomal Storage Disorders (LSDs): a rapid scoping review of available tools and domains.Orphanet journal of rare diseases · 2024Article
- Article
- Glycogen storage diseases.Nature reviews. Disease primers · 2023Review
- Work participation in adults with rare genetic diseases - a scoping review.BMC public health · 2023Article
- Association of migration status with quality of life among rural and urban adults with rare diseases: A cross-sectional study from China.Frontiers in public health · 2022Article
- Rare disease emerging as a global public health priority.Frontiers in public health · 2022Review
- Valuing the "Burden" and Impact of Rare Diseases: A Scoping Review.Frontiers in pharmacology · 2022Article
Corrections and comments
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Authors and funding
5 authors at 3 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundPompe disease (PD) is a rare inherited disorder caused by the deficiency of acid-α glucosidase, which leads to the impairment of organ and tissue functions and causes disabilities. As the first national survey on patients with late-onset PD (LOPD) in China, this study investigated the quality of life (QOL) of adult patients with LOPD in China and explored its contributors.
methodsData were derived from a nation-based, cross-sectional, self-response survey on rare diseases (RDs) in early 2018. Answers from 68 adult Chinese patients with LOPD were used for data analysis. QOL was measured using the World Health Organization Quality of Life: Brief Version. Covariates included age, gender, education, employment, reliance on assistive devices, medication history, social support, and disease economic burden. Data were analyzed using linear regression in R.
resultsFor adult patients with LOPD, the average scores and standard deviations (SD) of the four dimensions of QOL were physical health = 33.77 (SD = 18.28), psychological health = 43.81 (SD = 21.70), environmental health = 39.43 (SD = 16.93), and social relationship = 46.20 (SD = 19.76); the scoring for each dimension was evaluated on a scale of 0 to 100. At the significance level of p < 0.05, with increasing age, the patients experienced a significant decrease in physical health QOL (β = - 0.75) and environmental health QOL (β = - 0.79). Those who relied heavily on assistive devices had lower perceived physical health (β = - 17.8), psychological health (β = - 22.76), environmental health (β = - 17.8), and social relationships (β = - 22.12) than those who did not. A one-unit increase in the amount of social support, as a form of social interaction, led to a significant increase in physical health (β = 0.28), psychological health (β = 0.71), environmental health (β = 0.72), and social relationships (β = 0.70).
conclusionAdult Chinese patients with LOPD had a lower physical health and QOL compared to their counterparts with other RDs. Being employed was found to affect the QOL of adult Chinese patients with LOPD in almost all dimensions. Encouraging adult Chinese patients with LOPD to be socially active and help them become more involved in social life might improve their QOL.
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