ArticleBehavioral sciences (Basel, Switzerland)2023
Assessment of Parental Needs and Quality of Life in Children with a Rare Neuromuscular Disease (Pompe Disease): A Quantitative-Qualitative Study.
Article in Behavioral sciences (Basel, Switzerland), 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers, 1 of them a synthesis that pooled it.
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Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
4 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Assessing Psychological Harms on Parents and Primary Caregivers of Children Living with a Rare Disease: A Systematic Review of the Scope and Validity of Surveys Utilized.Clinical child and family psychology review · 2025Pooled it
- Measuring the psychological symptoms of pediatric palliative care patients and their caregivers: a scoping review of available instruments.BMC psychology · 2026Article
- Parents as First Responders: Experiences of Emergency Care in Children with Nemaline Myopathy: A Qualitative Study.Nursing reports (Pavia, Italy) · 2025Article
- Understanding the health-related quality of life impacts of caring for children and adolescents with rare progressive life-limiting conditions: key challenges and future research priorities.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025Article
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Pompe disease (PD) is a rare metabolic disorder with progressive neuromuscular consequences that negatively impact a child's development and quality of life (QoL). Despite an improved prognosis with treatment, the risk for early death due cardiorespiratory crisis remains. Parents not only face physical fatigue and family distress in coping with the child's special needs but also experience emotions, worries, and unexpressed needs (a "humanistic burden") that require supportive interventions. Fourteen parents of children with PD completed an online self-report questionnaire assessing their child's QoL, their own parental burden of care, and disease-related issues. The aim was to estimate the associations between the child's QoL and the caregiver's burden levels. Three mothers were also interviewed. A total of 57.1% of parents lived with moderate/severe burden conditions; worse QoL for the child was associated with higher levels of caregiver burden (
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