Document Type

Article

Publication Date

2-2025

Identifier

DOI: 10.1111/cts.70171; PMCID: PMC11839397

Abstract

While multiple oral medications are used to treat dystonia, limited information exists on current prescribing practices. This study analyzes real-world prescribing practices for pediatric dystonia in the United States, evaluating prescription frequency, dosing, and the impact of comorbidities. Oracle electronic health record real-world data were queried from 2014 to 2019 for encounters of patients under age 18 with a dystonia diagnosis and available medication records. Information was extracted on prescriptions for dystonia medications (baclofen, clonidine, carbidopa-levodopa, gabapentin, tetrabenazine, trihexyphenidyl, and select benzodiazepines), dosing, and comorbid diagnoses of cerebral palsy (CP), epilepsy, or spasticity. A total of 4010 pediatric patients with dystonia were included. Benzodiazepines were most commonly prescribed (midazolam in 53.5% of patients, diazepam 46.7%, lorazepam 41.9%, clonazepam 28.3%). This was followed by baclofen (33.4%), clonidine (26.3%), and gabapentin (19.7%). Dystonia patients with epilepsy were more commonly prescribed benzodiazepines than patients without epilepsy (diazepam 79.1% vs. 29%; clonazepam 50.9% vs. 16%) and baclofen was more often prescribed in patients with CP (59.4%) or spasticity (63.8%) than those without (17%). All medications showed decreased milligram per kilogram dosage as patient weight increased. Benzodiazepines, baclofen, and clonidine were the most common medications prescribed to pediatric patients with dystonia in the United States, although medical comorbidities impact prescribing practices. There was significant variability in weight-based dosing of all medications. There remains a need to determine which dystonia medications are most effective for each patient and the necessary drug exposure to maximize therapeutic efficacy and minimize adverse effects.

Journal Title

Clin Transl Sci

Volume

18

Issue

2

First Page

70171

Last Page

70171

MeSH Keywords

Humans; Child; United States; Practice Patterns, Physicians'; Male; Female; Adolescent; Child, Preschool; Dystonia; Clonidine; Baclofen; Infant; Drug Prescriptions; Epilepsy; Benzodiazepines; Gabapentin; Levodopa; Carbidopa; Comorbidity; Cerebral Palsy; Trihexyphenidyl; Electronic Health Records; Drug Combinations

PubMed ID

39972534

Keywords

data analysis; dosing; dystonia; neurology; pediatric; population analysis

Comments

This is an open access article under the terms of the Creative Commons Attribution-NonCommercial License, which permits use, distribution and reproduction in any medium, provided the original work is properly cited and is not used for commercial purposes.

Publisher's Link: https://ascpt.onlinelibrary.wiley.com/doi/10.1111/cts.70171

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