Perspectives from the Society for Pediatric Research: pharmacogenetics for pediatricians

Girdwood, Sonya Tang; Rossow, Katelyn M; Driest, Sara L. Van; Ramsey, Laura B.

doi:10.1038/s41390-021-01499-2

Cited by 5 publications

(1 citation statement)

References 127 publications

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“…While the unique characteristics of children necessitate child-specific evidence, they also impose distinct challenges to gathering this evidence. The most prominent issue identified in the literature is the rarity of childhood disease and the implications this has for reaching statistical significance in clinical trials [18][19][20][21][23][24][25][26]28,[38][39][40][41][42][43][44][45][46][47][48][49][50][51][52][53][54]. The challenge of small sample size is compounded by genotyping, which further subdivides eligible participants into molecular-based strata [41,44,53,54].…”

Section: Study Design Challenges and Potential Solutionsmentioning

confidence: 99%

Translating Precision Health for Pediatrics: A Scoping Review

et al. 2023

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Precision health aims to personalize treatment and prevention strategies based on individual genetic differences. While it has significantly improved healthcare for specific patient groups, broader translation faces challenges with evidence development, evidence appraisal, and implementation. These challenges are compounded in child health as existing methods fail to incorporate the physiology and socio-biology unique to childhood. This scoping review synthesizes the existing literature on evidence development, appraisal, prioritization, and implementation of precision child health. PubMed, Scopus, Web of Science, and Embase were searched. The included articles were related to pediatrics, precision health, and the translational pathway. Articles were excluded if they were too narrow in scope. In total, 74 articles identified challenges and solutions for putting pediatric precision health interventions into practice. The literature reinforced the unique attributes of children and their implications for study design and identified major themes for the value assessment of precision health interventions for children, including clinical benefit, cost-effectiveness, stakeholder values and preferences, and ethics and equity. Tackling these identified challenges will require developing international data networks and guidelines, re-thinking methods for value assessment, and broadening stakeholder support for the effective implementation of precision health within healthcare organizations. This research was funded by the SickKids Precision Child Health Catalyst Grant.

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Section: Study Design Challenges and Potential Solutionsmentioning

confidence: 99%

Translating Precision Health for Pediatrics: A Scoping Review

et al. 2023

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Acid-Lowering Drugs for the Treatment of Gastro-esophageal Reflux Disease

Scarpignato

2022

Gastroesophageal Reflux in Children

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Molecular Characterisation of the Mechanism of Action of Stimulant Drugs Lisdexamfetamine and Methylphenidate on ADHD Neurobiology: A Review

2022

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Attention-deficit/hyperactivity disorder (ADHD) is a common childhood-onset neurodevelopmental disorder characterised by persistent inattention, hyperactivity and impulsivity. Moreover, ADHD is commonly associated with other comorbid diseases (depression, anxiety, bipolar disorder, etc.). The ADHD symptomatology interferes with subject function and development. The treatment of ADHD requires a multidisciplinary approach based on a combination of non-pharmacological and pharmacological treatments with the aim of ameliorating the symptomatology; among first-line pharmacological treatments are stimulants [such as methylphenidate (MPH) and lisdexamfetamine dimesylate (LDX)]. In this review we explored recent ADHD- and stimulants-related literature, with the aim of compiling available descriptions of molecular pathways altered in ADHD, and molecular mechanisms of current first-line stimulants MPH and LDX. While conducting the narrative review, we applied structured search strategies covering PubMed/MEDLINE database and performed handsearching of reference lists on the results of those searches. The aetiology and pathophysiology of ADHD are incompletely understood; both genetic and environmental factors have been associated with the disorder and its grade of burden, and also the relationship between the molecular mechanisms of pharmacological treatments and their clinical implications. The lack of comprehensive understanding of the underlying molecular pathology makes both the diagnosis and treatment difficult. Few published studies evaluating molecular data on the mechanism of action (MoA) of MPH and LDX on ADHD are available and most of them are based on animal models. Further studies are necessary to improve the knowledge of ADHD pathophysiology and how the MoAs of MPH and LDX differentially modulate ADHD pathophysiology and control ADHD symptomatology.

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Perspectives from the Society for Pediatric Research: pharmacogenetics for pediatricians

Cited by 5 publications

References 127 publications

Translating Precision Health for Pediatrics: A Scoping Review

Translating Precision Health for Pediatrics: A Scoping Review

Acid-Lowering Drugs for the Treatment of Gastro-esophageal Reflux Disease

Molecular Characterisation of the Mechanism of Action of Stimulant Drugs Lisdexamfetamine and Methylphenidate on ADHD Neurobiology: A Review

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