Health-Related Quality of Life in Children and Adolescents With Spinal Muscular Atrophy in the Czech Republic
Abstrakt
BACKGROUND: Spinal muscular atrophy is a rare hereditary neuromuscular disorder (with a prevalence of 1 per 30,000) that greatly debilitates patients and, in most cases, shortens their life expectancy. Although there is no causal therapy, improvements in symptomatic therapy have extended patients' life expectancy and increased their quality of life.
Unfortunately, the advancements in care vary from country to country. To improve the care for children with spinal muscular atrophy in the Czech Republic, we created a survey to obtain the baseline information about their quality of life and compared the data with equivalent data from the United States.
METHODS: We used the Pediatric Quality of Life Inventory 3.0 Neuromuscular Measurement Model, which is a health-related quality of life questionnaire specific to children with neuromuscular disorders. The survey was conducted on 35 children with genetically proven spinal muscular atrophy and their parents.
RESULTS: Compared with the US data, the Czech data generally show a lower quality of life, mainly in the family resources part. The greatest score was achieved in the section about communication.
Altogether, the parents' scores are lower than those of the children. CONCLUSION: In the Czech Republic, patients with spinal muscular atrophy and, especially their parents, have a significantly lower quality of life compared with US patients, mostly because of economic factors and a lack of social support.
Our results reveal areas toward which improvement should be directed. The need for family support through social care as well as civic, patient, or organizational support is accentuated.