Trial reportThe Journal of pediatrics2019
Health-Related Quality of Life in Children and Young Adults with Marfan Syndrome.
Trial report in The Journal of pediatrics, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers, 1 of them a synthesis 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
21 citing papers in PubMed, 1 synthesis or guideline pooled it, 47 citations in OpenAlex.
- Quality of life of pediatric and adult individuals with osteogenesis imperfecta: a meta-analysis.Orphanet journal of rare diseases · 2023Pooled it
- Prediction of vulnerability to mental health symptoms in children with congenital ectopia lentis: development and validation of a prediction model.Eye (London, England) · 2025Article
- Family functioning and health-related quality of life in children and young adults with Marfan syndrome.Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation · 2025Article
- Management of aortic disease in children with FBN1-related Marfan syndrome.European heart journal · 2024Review
- Health-related quality of life in children and adolescents with Marfan syndrome or related disorders: a controlled cross-sectional study.Orphanet journal of rare diseases · 2024Article
- In vivo phenotypic vascular dysfunction extends beyond the aorta in a mouse model for fibrillin-1 (Fbn1) mutation.Scientific reports · 2024Article
- Cerebral microvascular density, blood-brain barrier permeability, and support for neuroinflammation indicate early aging in a Marfan syndrome mouse model.Frontiers in physiology · 2024Article
- Article
- Health-related quality of life in children with congenital vascular malformations.European journal of pediatrics · 2023Article
- Heritable connective tissue disorders in childhood: Decreased health-related quality of life and mental health.American journal of medical genetics. Part A · 2022Observational
- How to Distinguish Marfan Syndrome from Marfanoid Habitus in a Physical Examination-Comparison of External Features in Patients with Marfan Syndrome and Marfanoid Habitus.International journal of environmental research and public health · 2022Article
- Development of a Quality-of-Life Survey for Patients With Succinic Semialdehyde Dehydrogenase Deficiency, a Rare Disorder of GABA Metabolism.Journal of child neurology · 2021Article
- Marfan syndrome.Nature reviews. Disease primers · 2021Review
- Quality of life in patients with Marfan syndrome: a cross-sectional study of 102 adult patients.Cardiovascular diagnosis and therapy · 2021Article
- Male-female differences in quality of life and coping style in patients with Marfan syndrome and hereditary thoracic aortic diseases.Journal of genetic counseling · 2020Article
- Health-related quality of life in Marfan syndrome: a 10-year follow-up.Health and quality of life outcomes · 2020Article
- A Baseline Measurement of Quality of Life in 322 Adults With Osteogenesis Imperfecta.JBMR plus · 2020Article
- Social Media Use Among Young Adults With Connective Tissue Disorders: Cross-Sectional Pilot Study.JMIR pediatrics and parenting · 2020Article
- A scoping review presenting a wide variety of research on paediatric and adolescent patients with Marfan syndrome.Acta paediatrica (Oslo, Norway : 1992) · 2020Article
- Marfan syndrome in adolescence: adolescents' perspectives on (physical) functioning, disability, contextual factors and support needs.European journal of pediatrics · 2019Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
27 authors at 20 institutions in 3 countries.
Funding
Abstract
objectiveTo assess health-related quality of life (HRQOL) in a large multicenter cohort of children and young adults with Marfan syndrome participating in the Pediatric Heart Network Marfan Trial. STUDY
designThe Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales were administered to 321 subjects with Marfan syndrome (5-25 years). PedsQL scores were compared with healthy population norms. The impact of treatment arm (atenolol vs losartan), severity of clinical features, and number of patient-reported symptoms on HRQOL was assessed by general linear models.
resultsMean PedsQL scores in children (5-18 years) with Marfan syndrome were lower than healthy population norms for physical (P ≤ .003) and psychosocial (P < .001) domains; mean psychosocial scores for adults (19-25 years) were greater than healthy norms (P < .001). HRQOL across multiple domains correlated inversely with frequency of patient-reported symptoms (r = 0.30-0.38, P < .0001). Those <18 years of age with neurodevelopmental disorders (mainly learning disability, attention-deficit/hyperactivity disorder) had lower mean PedsQL scores (5.5-7.4 lower, P < .04). A multivariable model found age, sex, patient-reported symptoms, and neurodevelopmental disorder to be independent predictors of HRQOL. There were no differences in HRQOL scores by treatment arm, aortic root z score, number of skeletal features, or presence of ectopia lentis.
conclusionsChildren and adolescents with Marfan syndrome were at high risk for impaired HRQOL. Patient-reported symptoms and neurodevelopmental disorder, but not treatment arm or severity of Marfan syndrome-related physical findings, were associated with lower HRQOL.
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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.