Therapeutic drug monitoring-guided dosing for pediatric cystic fibrosis patients: recent advances and future outlooks

S Bentley, J Cheong, N Gudka… - Expert Review of …, 2023 - Taylor & Francis
S Bentley, J Cheong, N Gudka, S Makhecha, S Hadjisymeou-Andreou, JF Standing
Expert Review of Clinical Pharmacology, 2023Taylor & Francis
Introduction Medicine use in children with cystic fibrosis (CF) is complicated by inconsistent
pharmacokinetics at variance with the general population, a lack of research into this and its
effects on clinical outcomes. In the absence of established dose regimens, therapeutic drug
monitoring (TDM) is a clinically relevant tool to optimize drug exposure and maximize
therapeutic effect by the bedside. In clinical practice though, use of this is variable and
limited by a lack of expert recommendations. Areas covered We aimed to review the use of …
Introduction
Medicine use in children with cystic fibrosis (CF) is complicated by inconsistent pharmacokinetics at variance with the general population, a lack of research into this and its effects on clinical outcomes. In the absence of established dose regimens, therapeutic drug monitoring (TDM) is a clinically relevant tool to optimize drug exposure and maximize therapeutic effect by the bedside. In clinical practice though, use of this is variable and limited by a lack of expert recommendations.
Areas covered
We aimed to review the use of TDM in children with CF to summarize recent developments, current recommendations, and opportunities for future directions. We searched PubMed for relevant publications using the broad search terms “cystic fibrosis” in combination with the specific terms ”therapeutic drug monitoring (TDM)” and ”children.” Further searches were undertaken using the name of identified drugs combined with the term ”TDM.”
Expert opinion
Further research into the use of Bayesian forecasting and the relationship between exposure and response is required to personalize dosing, with the opportunity for the development of expert recommendations in children with CF. Use of noninvasive methods of TDM has the potential to improve accessibility to TDM in this cohort.
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