Examining the Role of Precision Medicine with Oral Baclofen in Pediatric Patients with Cerebral Palsy

  • Matthew J. McLaughlinEmail author
  • Susan Abdel-Rahman
  • J. Steven Leeder
Pediatric Rehabilitation Medicine (A Houtrow and M Fuentes, Section Editors)
Part of the following topical collections:
  1. Topical Collection on Pediatric Rehabilitation Medicine


Purpose of Review

Despite its widespread use, oral baclofen requires a critical review of the pharmacology to determine potential precision medicine applications to improve medication administration. Discussing the dose→exposure→response relationship of oral baclofen allows a conceptual framework in which designing clinical trials would become more successful. This paper seeks to examine some of the areas where variability in exposures can exist and lead to undesired clinical responses.

Recent Findings

Several factors are at play to implement precision medicine with oral baclofen in the pediatric patient with cerebral palsy. Variations in intestinal absorption, oral baclofen clearance, pharmacogenomic variants, and distribution of this medication into the cerebrospinal fluid cause differences in the amount of baclofen available at the GABA-B receptor site, causing a clinical response.


Oral baclofen has significant variability in disposition and clinical response. Research to determine the causes for this variability and controlling for these factors would allow improvement in clinical outcomes.


Cerebral palsy Baclofen Precision medicine Rehabilitation Pharmacogenetics Pediatrics 


Compliance with Ethical Standards

Conflict of Interest

Matthew McLaughlin reports grants from NICHD T32 HD069038 during the conduct of the study. Susan Abdel-Rahman and J. Steven Leeder declare no conflicts of interest relevant to this manuscript.

Human and Animal Rights and Informed Consent

This article does not contain any studies with human or animal subjects performed by any of the authors.


Papers of particular interest, published recently, have been highlighted as: • Of importance •• Of major importance

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Copyright information

© Springer Science+Business Media, LLC, part of Springer Nature 2019

Authors and Affiliations

  • Matthew J. McLaughlin
    • 1
    Email author
  • Susan Abdel-Rahman
    • 1
  • J. Steven Leeder
    • 1
  1. 1.Children’s Mercy HospitalKansas CityUSA

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