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

  • Pediatric Rehabilitation Medicine (A Houtrow and M Fuentes, Section Editors)
  • Published:
Current Physical Medicine and Rehabilitation Reports Aims and scope Submit manuscript

Abstract

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.

Summary

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.

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Authors and Affiliations

  1. Children’s Mercy Hospital, 2401 Gillham Road, Kansas City, MO, 64108, USA

    Matthew J. McLaughlin, Susan Abdel-Rahman & J. Steven Leeder

Authors
  1. Matthew J. McLaughlin
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  2. Susan Abdel-Rahman
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  3. J. Steven Leeder
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Correspondence to Matthew J. McLaughlin.

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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.

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

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This article is part of the Topical Collection on Pediatric Rehabilitation Medicine

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McLaughlin, M.J., Abdel-Rahman, S. & Leeder, J.S. Examining the Role of Precision Medicine with Oral Baclofen in Pediatric Patients with Cerebral Palsy. Curr Phys Med Rehabil Rep 7, 40–45 (2019). https://doi.org/10.1007/s40141-019-0212-6

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  • DOI: https://doi.org/10.1007/s40141-019-0212-6

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