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The Participant-Reported Implementation Update and Score PRIUS: A Novel Method for Capturing Implementation-Related Data Over Time
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Considerations for Implementing Precision Therapeutics for Children.

Matthew J McLaughlin1, Jonathan Wagner1, Valentina Shakhnovich1

  • 1Children's Mercy Hospital, Kansas City, Missouri, USA.

Clinical and Translational Science
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PubMed
Summary
This summary is machine-generated.

Optimizing pediatric pharmacologic agents requires considering genetic and developmental factors. Focusing on drug response, not just dose, is crucial for personalized pediatric medicine.

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Area of Science:

  • Pharmacology
  • Pediatric Medicine
  • Genetics

Background:

  • Individual variability in drug response is influenced by genetic factors and ontogeny (age-specific developmental changes).
  • Pediatric patients exhibit unique developmental trajectories impacting drug disposition and efficacy.
  • Current approaches often focus on dose-exposure relationships, potentially overlooking individual response variations.

Purpose of the Study:

  • To emphasize a shift towards response-based therapeutic strategies in pediatric pharmacologic agent utilization.
  • To highlight the importance of understanding both genetic and ontogenic contributions to drug variability.
  • To advocate for the development of tools for individualizing drug response in children.

Main Methods:

  • Reviewing the impact of genetic factors on drug metabolism and target receptor sites.
  • Analyzing age-specific developmental changes (ontogeny) affecting drug exposure and response.
  • Discussing the utility of concentration-controlled studies for identifying optimal therapeutic exposures.

Main Results:

  • Genetic and ontogenic factors significantly contribute to inter-individual variability in pediatric drug response.
  • Understanding drug target receptor expression and binding affinity is key to achieving desired therapeutic outcomes.
  • Concentration-controlled studies offer a pathway to individualize drug exposure based on patient-specific factors.

Conclusions:

  • A response-centered approach, informed by genetics and ontogeny, is essential for optimizing pediatric pharmacologic treatments.
  • Developing tools to personalize drug delivery across the pediatric age spectrum is a critical unmet need.
  • Individualized pediatric medicine can lead to significant, lifelong benefits for patients.