ArticleBMJ open2018
Financial, opportunity and psychosocial costs of spinal muscular atrophy: an exploratory qualitative analysis of Australian carer perspectives.
Article in BMJ open, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 36 papers, 5 of them syntheses that pooled it.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
36 citing papers in PubMed, 5 syntheses or guidelines pooled it, 70 citations in OpenAlex.
- Caregiver Burden of Spinal Muscular Atrophy: A Systematic Review.PharmacoEconomics · 2023Pooled it
- Parents as informal caregivers of children and adolescents with spinal muscular atrophy: a systematic review of quantitative and qualitative data on the psychosocial situation, caregiver burden, and family needs.Orphanet journal of rare diseases · 2022Pooled it
- Systematic Literature Review to Identify Utility Values in Patients with Spinal Muscular Atrophy (SMA) and Their Caregivers.PharmacoEconomics · 2022Pooled it
- Systematic Literature Review to Assess the Cost and Resource Use Associated with Spinal Muscular Atrophy Management.PharmacoEconomics · 2022Pooled it
- Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments.Orphanet journal of rare diseases · 2021Pooled it
- Parental illness intrusiveness in parents of children with neuromuscular disorders.Journal of neuromuscular diseases · 2026Article
- Economic Burden of Spinal Muscular Atrophy in Singapore: A Societal Perspective Cost-of-Illness Study.PharmacoEconomics - open · 2026Article
- Adaptive functioning in school-aged children with spinal muscular atrophy in the treatment era: a non-randomised cohort study.The Lancet regional health. Western Pacific · 2026Article
- Data-Related and Methodological Challenges in Assessing the Cost-Effectiveness of Traditional and Genomic Newborn Screening Programs.Applied health economics and health policy · 2026Review
- Counting the Cost: The Hidden Financial Realities of Neuromuscular Disease Through Patient and Family Perspectives.Health expectations : an international journal of public participation in health care and health policy · 2025Article
- Review
- "…They were just treating her Symptom by Symptom": maternal experiences of having a child with spinal muscular atrophy in Ghana.BMC palliative care · 2025Article
- Use of the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND) in Spinal Muscular Atrophy.Journal of clinical medicine · 2024Article
- Spinal muscular atrophy type 1 in the Caribbean: the first case report from the Dominican Republic.Frontiers in neuroscience · 2024Article
- Gratitude Experience in Ten Patients with Spinal Muscular Atrophy: A Qualitative Study.Psychology research and behavior management · 2024Article
- Experiences of Health Care and Psychosocial Needs in Parents of Children with Spinal Muscular Atrophy.International journal of environmental research and public health · 2023Article
- Assessment of health-related quality of life in patients with spina muscular atrophy in China.Intractable & rare diseases research · 2022Article
- Investigating attitudes toward prenatal diagnosis and fetal therapy for spinal muscular atrophy.Prenatal diagnosis · 2022Article
- A mixed method study on the impact of living with spinal muscular atrophy in Malaysia from patients' and caregivers' perspectives.Orphanet journal of rare diseases · 2022Article
- The involvement of rare disease patient organisations in therapeutic innovation across rare paediatric neurological conditions: a narrative review.Orphanet journal of rare diseases · 2022Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesSpinal muscular atrophy (SMA) has profound implications for patients and families. The aim of the present study was to gain insights into the effects caring for a child with SMA has on the costs incurred by families caring for a child with SMA from carer perspectives to identify gaps in provision of care, inform public policy and cost-effectiveness analyses.
designInterpretive phenomenological analysis guided the delivery and analysis of semi-structured interviews undertaken to explore the financial, opportunity and psychosocial costs associated with caring for children with SMA. PARTICIPANTS AND
settingParents of children with SMA types II and III from a single Australian paediatric neuromuscular clinic participated in this study.
resultsA range of experiences were reported and information saturation (n=7) was reached endorsing themes, including: significant financial and caregiving burdens, adjusted career choices and limitations on career progression and a complex landscape of access to funding, equipment, support and resources. Opportunity costs of foregone employment, purchases and leisure activities were substantial, as were emotional and social impacts. Participants voiced determination and resilience, and called for continued efforts to improve supportive care services and resources.
conclusionsThe range and nature of costs met by families caring for a child with SMA were found to be expansive and not typically recognised. These include high direct costs associated with goods and services, indirect costs associated with voluntary care, substantial and long-term opportunities foregone in paid employment and career progression and unmeasured or hidden costs associated with mental health burden.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.