BACKGROUND AND OBJECTIVES
Representative enrollment of racial and ethnic minoritized populations in biomedical research ensures the generalizability of results and equitable access to novel therapies. Previous studies on pediatric clinical trial diversity are limited to subsets of journals or disciplines. We aimed to evaluate race and ethnicity reporting and representation in all US pediatric clinical trials on ClinicalTrials.gov.
METHODS
We performed a cross-sectional study of US-based clinical trials registered on ClinicalTrials.gov that enrolled participants aged <18 years old between October 2007 and March 2020. We used descriptive statistics, compound annual growth rates, and multivariable logistic regression for data analysis. Estimates of US population statistics and disease burden were calculated with the US Census, Kids’ Inpatient Database, and National Survey of Children’s Health.
RESULTS
Among 1183 trials encompassing 405 376 participants, race and ethnicity reporting significantly increased from 27% in 2007 to 87% in 2018 (P < .001). The median proportional enrollment of Asian American children was 0.6% (interquartile range [IQR], 0%–3.7%); American Indian, 0% (IQR, 0%–0%); Black, 12% (IQR, 2.9%–28.4%); Hispanic, 7.1% (IQR, 0%–18.6%); and white 66.4% (IQR, 41.5%–81.6%). Asian American, Black, and Hispanic participants were underrepresented relative to US population demographics. Compared with expected proportions based on disease prevalence and hospitalizations, Asian American and Hispanic participants were most consistently underrepresented across diagnoses.
CONCLUSIONS
While race and ethnicity reporting in pediatric clinical trials has improved, the representative enrollment of minoritized participants remains an ongoing challenge. Evidence-based and policy solutions are needed to address these disparities to advance biomedical innovation for all children.
Axenfeld–Rieger syndrome is a rare multi-system disorder associated with cardiac anomalies. All patients with a diagnosis of Axenfeld–Rieger syndrome were identified from our electronic medical record. Chart review was performed to document the presence and types of CHD. Out of 58 patients, 14 (24.1%) had CHD and a wide variety of cardiac lesions were identified.
Background and Objective: Improvement in the affordability and convenience of genetic testing has rapidly expanded the understanding of the mechanistic causes of various pediatric cardiomyopathies.Concurrently, new therapies are being developed to better-target specific pathologies as opposed to classic therapies that treat the maladaptive processes of chronic heart failure. This review will discuss the advances in genetic testing and specific therapies that have been shown to benefit or potentially benefit genetically distinct subsets of the pediatric population with heart failure or at risk of developing heart failure.Methods: We undertook a comprehensive database search (January 2000-August 2022) of PubMed, utilizing terms 'pediatric', 'cardiomyopathy', 'heart failure', 'genetics', and 'precision medicine'. Additional notable studies were obtained from ClinicalTrials.gov. Studies published in English that examine genetic basis and treatment modalities of pediatric heart failure.
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