ArticleCardiovascular diagnosis and therapy2019
Long-term outcomes of childhood onset Noonan compared to sarcomere hypertrophic cardiomyopathy.
Article in Cardiovascular diagnosis and therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 2 of them syntheses that pooled it.
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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.
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Who cites it
23 citing papers in PubMed, 2 syntheses or guidelines pooled it, 38 citations in OpenAlex.
- Trametinib as a targeted treatment in cardiac and lymphatic presentations of Noonan syndrome.Frontiers in pediatrics · 2025Pooled it
- Guidelines on the Diagnosis and Treatment of Hypertrophic Cardiomyopathy - 2024.Arquivos brasileiros de cardiologia · 2024Guideline
- Targeting Interleukin-8-Mediated Cellular Crosstalk Reverses Hypertrophic Cardiomyopathy and Cardiac Fibrosis in Noonan Syndrome.Circulation · 2026Article
- Impact of RASopathy subtype on the early disease course of RASopathy-associated hypertrophic cardiomyopathy: clinical outcomes and genetic insights.Pediatric research · 2026Article
- Characterisation and prognostic implications of the 12-lead electrocardiogram in children with RASopathy-associated hypertrophic cardiomyopathy.Heart (British Cardiac Society) · 2026Article
- Childhood-onset RASopathy-associated hypertrophic cardiomyopathy, diastolic dysfunction, and arrhythmias.European heart journal · 2026Article
- The impact of genotype-phenotype on the prognosis of children with hypertrophic cardiomyopathy.JTCVS open · 2026Article
- Clinical characteristics of 41 children with hypertrophic cardiomyopathy: A single-center retrospective study.The Journal of international medical research · 2025Article
- Cardiovascular aspects of Noonan syndrome and related disorders.Medizinische Genetik : Mitteilungsblatt des Berufsverbandes Medizinische Genetik e.V · 2025Article
- Trametinib alters contractility of paediatric Noonan syndrome-associated hypertrophic myocardial tissue slices.ESC heart failure · 2025Article
- Health outcomes and drug utilisation in children with Noonan syndrome: a European cohort study.Orphanet journal of rare diseases · 2025Article
- Impact of MEK Inhibition on Childhood RASopathy-Associated Hypertrophic Cardiomyopathy.JACC. Basic to translational science · 2025Article
- Biventricular outflow tract obstruction due to hypertrophy related to compound heterozygous variants in LZTR1.ESC heart failure · 2024Article
- Noonan Syndrome: Relation of Genotype to Cardiovascular Phenotype-A Multi-Center Retrospective Study.Genes · 2024Article
- Natural history and outcomes in paediatric RASopathy-associated hypertrophic cardiomyopathy.ESC heart failure · 2024Article
- Natural History of Hypertrophic Cardiomyopathy in Noonan Syndrome With Multiple Lentigines.Circulation. Genomic and precision medicine · 2023Article
- Narrative review of pediatric heart failure in the age of precision medicine.Translational pediatrics · 2023Review
- Multimodality Imaging in Noonan Syndrome: Case Series of Young Children.Radiology. Cardiothoracic imaging · 2023Article
- Cell cycle defects underlie childhood-onset cardiomyopathy associated with Noonan syndrome.iScience · 2022Article
- Hypertrophic Cardiomyopathy in RASopathies: Diagnosis, Clinical Characteristics, Prognostic Implications, and Management.Heart failure clinics · 2022Review
Corrections and comments
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Authors and funding
14 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundTo compare outcome and cardiac pathology between patients with Noonan syndrome (N-HCM) and sarcomere protein-associated (S-HCM) childhood onset hypertrophic cardiomyopathy (HCM).
methodsClinical data were recorded from medical charts. Primary endpoint was survival. Secondary endpoints were survival without hospitalization, without intervention or without arrhythmic events. Functional clinical status and results from genetic testing, imaging, electrocardiographic (ECG) studies, cardiopulmonary exercise testing (CPET) and histopathology were compared between groups.
resultsChildhood HCM was diagnosed in 29 N-HCM and 34 S-HCM patients. Follow-up time was greater than 10 years in more than half of all patients. Mortality was below 7% and not different between groups. Children with N-HCM presented at a younger age and there was less time of survival without hospitalization for heart failure or intervention in N-HCM compared to S-HCM patients. Clinical functional status improved over time in N-HCM patients. On long-term follow-up, left ventricular posterior wall thickness indexed to body surface area decreased in N-HCM and increased in S-HCM patients. There was a trend to lower risk for severe arrhythmic events in N-HCM patients and only S-HCM individuals received an implantable cardioverter-defibrillator. There were no differences between groups in ventricular function, ECG and CPET parameters. Myocardial fibrosis as assessed by histopathology of myocardial specimens and cardiovascular magnetic resonance with late gadolinium enhancement or T1 mapping was present in both groups.
conclusionsWhen compared to S-HCM patients, children with N-HCM have increased morbidity during early disease course, but favorable long-term outcome with low mortality, stagnation of myocardial hypertrophy, and low risk for malignant arrhythmias.
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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.