Measuring parent proxy-reported quality of life of 11 rare diseases in children in Zhejiang, China
- PDF / 979,343 Bytes
- 9 Pages / 595.276 x 790.866 pts Page_size
- 1 Downloads / 190 Views
Open Access
RESEARCH
Measuring parent proxy‑reported quality of life of 11 rare diseases in children in Zhejiang, China Qisheng Gao1 , Shanshan Wang2, Jianping Ren2 and Xin Wen3*
Abstract Background: It has become increasingly important to measure the health-related quality of life (HRQoL) of rare diseases in children and adolescents in recent decades. Much attention has been paid to investigate the HROoL of a specific rare disease by self-report in previous studies. This study aimed to evaluate and compare the HROoL of 11 rare diseases in Chinese children by parent proxy-report, to explore the factors associated with HROoL of patients, and to understand the problems of most concern. Methods: A total of 651 children aged from 2 to 18 were enrolled from the Children’s Hospital Affiliated Zhejiang University in 2018. Their parents completed the parent proxy-report version of the Pediatric Quality of Life Inventory™ 4.0 (PedsQL™ 4.0). Independent samples t-test, one-way ANOVA, or Kruskal–Wallis H test was used to compare HROoL scores between groups. Multilevel linear regression models with random intercept were applied to analyze the relationship between socioeconomic variables and both the total score and subdomain scores. Results: The total PedsQL scores of Patent ductus arteriosus (PDA), Infantile agranulocytosis, Autoimmune thrombocytopenia (ITP), Polysyndactyly, Hirschsprung disease, Cleft lip and palate, Tetralogy of fallot, Myasthenia gravis, Guillain–barre syndrome, Glycogen storage disease, and Langerhans cell histiocytosis children were 79.65 ± 5.46, 95.88 ± 3.48, 71.39 ± 3.27, 91.77 ± 6.35, 76.18 ± 6.92, 96.33 ± 4.22, 77.85 ± 8.90, 95.99 ± 3.31, 85.77 ± 4.56, 82.97 ± 4.13 and 77.6 ± 5.15, respectively. Age was significantly associated with physical functioning, school functioning, and psychosocial health scores. The household registration place was significantly related to the total score. The most urgent desire of patients was to reduce the overall medical costs. Conclusions: This study showed that patients with PDA had the lowest physical functioning score, while patients with ITP scored the lowest in the emotional functioning, social functioning, school functioning, psychosocial health, and total scores. Incentive policies should be further adopted to improve orphan drug availability and reduce the economic burden of rare diseases. Background Rare diseases, also known as “orphan diseases”, refer to diseases with a low prevalence but which are seriously debilitating or even life-threatening [1]. There is no *Correspondence: [email protected] 3 Center for Medical Science, Technology and Education of Zhejiang Province, No, 60 Hefang Street, Hangzhou 310006, Zhejiang Province, China Full list of author information is available at the end of the article
universal definition of rare diseases worldwide. Different countries or regions have distinct definitions depending on disease incidence or prevalence, the severity of the disease, and the existence of adequate treatments or drugs. The average prevalenc
Data Loading...
