ReviewQuality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation2018
Measuring quality of life in children with spinal muscular atrophy: a systematic literature review.
Review in Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 14 papers, 3 of them syntheses that pooled it.
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Who cites it
14 citing papers in PubMed, 3 syntheses or guidelines pooled it, 42 citations in OpenAlex.
- 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
- Health-related quality of life scores of typically developing children and adolescents around the world: a meta-analysis with meta-regression.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2020Pooled it
- Quality of life measures in older adults after traumatic brain injury: a systematic review.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2019Pooled it
- Longitudinal Efficacy of Nusinersen Treatment on Health-Related Quality of Life and Independence in Children With Later-Onset Spinal Muscular Atrophy.Muscle & nerve · 2025Article
- The Socio-Economic Burden of Spinal Muscular Atrophy: A Cost-of-Illness Study in Bulgaria.Healthcare (Basel, Switzerland) · 2025Article
- Quality of life in patients with spinal muscular atrophy in Brazil: patient self-assessment and carer perception.Revista paulista de pediatria : orgao oficial da Sociedade de Pediatria de Sao Paulo · 2025Observational
- Quality of Life Assessment in Romanian Patients with Spinal Muscular Atrophy Undergoing Nusinersen Treatment.Neurology international · 2024Article
- Identification of the most relevant aspects of spinal muscular atrophy (SMA) with impact on the quality of life of SMA patients and their caregivers: the PROfuture project, a qualitative study.Journal of patient-reported outcomes · 2024Article
- Measurement properties of the EQ-5D-Y-3L, PedsQL 4.0, and PROMIS-25 Profile v2.0 in pediatric patients with spinal muscular atrophy.Health and quality of life outcomes · 2024Article
- Assessing validity of the EQ-5D-5L proxy in children and adolescents with Duchenne muscular dystrophy or spinal muscular atrophy.The European journal of health economics : HEPAC : health economics in prevention and care · 2024Article
- Pain in Children and Adolescents with Spinal Muscular Atrophy: A Longitudinal Study from a Patient Registry.Children (Basel, Switzerland) · 2023Article
- The quality of life in children with spinal muscular atrophy: a case-control study.BMC pediatrics · 2022Article
- Drug Screening and Drug Repositioning as Promising Therapeutic Approaches for Spinal Muscular Atrophy Treatment.Frontiers in pharmacology · 2020Review
- Patient characteristics and hospitalisation costs of spinal muscular atrophy in Spain: a retrospective multicentre database analysis.BMJ open · 2019Article
Corrections and comments
- Erratum issued
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
objectivesSpinal muscular atrophy (SMA) is a rare, hereditary, autosomal recessive neuromuscular disorder that, in its most severe forms, impacts infants and children. Once symptomatic, it is characterized clinically by a distinct inability to achieve motor milestones, such as the ability to lift the head, sit, stand, or walk. Quality of life (QOL) measurement in very young infants presents a particular challenge. Therefore, this review aims to highlight commonly used measurement tools and identifies future research opportunities for QOL measurement in SMA.
methodsA systematic literature review was carried out focusing on the various tools used to measure QOL in children < 18 years of age with formally diagnosed SMA type I, II, or III. Although the disease area of interest was SMA, data on Duchenne's muscular dystrophy were also included because of the rare nature of SMA.
resultsThe Pediatric Quality of Life Inventory was the most commonly utilized tool to measure QOL in children; this included the generic and neuromuscular modules. No disease-specific tool to capture QOL in children with SMA was identified. Additionally, no measurement tools exist for very young infants (i.e., under 12 months) with SMA Type 1.
conclusionsEvolving standards of care will lead to increased interest by stakeholders, on the methods used to measure QOL in infants and children across all types of SMA. Generic tools may not adequately capture QOL changes in SMA, especially given the age group affected by the disease. Further research is required to explore the scope for a disease-focused approach.
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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.