# Intrathecal baclofen and pediatric dystonia.

> **NIH NIH R01** · BAYLOR COLLEGE OF MEDICINE · 2024 · $680,870

## Abstract

Over 11,000 infants are diagnosed annually with cerebral palsy (CP) with lifetime medical costs >$1.4
million/person. Elevated muscle tone includes dystonia (involuntary muscular contraction) and spasticity
(velocity-dependent muscle stiffness), which often co-exist, greatly impairing function, mobility, and quality of
life and causing pain and bony deformities. After decades of research, dystonia remains poorly understood and
managed. Intrathecal baclofen (ITB) delivered continuously via an implanted pump is widely used to manage
dystonic CP despite equivocal efficacy data and very low evidence supporting ITB use. Co-morbid spasticity is
a major confounder that can mask dystonia; further, multiple patterns of brain malformation/injury cause
dystonia and most studies fail to address dystonia triggers, e.g., pain. A 2023 study, however, found that
despite limited dystonia reduction, ITB improved caregiving ease, pain, comfort, and mobility. This revised
proposal specifically seeks to overcome these identified serious limitations of prior studies and thoroughly
investigate ITB’s effects using an adequately powered, prospective observational cohort design. This clinical
trial will use a standardized titration protocol to optimize ITB dosing. We hypothesize that children with more
severe baseline dystonia, spasticity, and pain, as well as those with white matter brain injury patterns, will
show the best overall response to ITB.
AIM 1) Determine the 12-month effects of ITB on a cohort of 65 clinically representative, ITB naïve, children
with dystonic CP using a standardized titration protocol and a battery of assessments to capture critical
components of the child’s function and well-being.
AIM 2) Complete a detailed characterization of brain malformation and injury patterns in children with dystonic
CP. This will be the first study to combine multiple imaging analysis techniques in this population, including the
MRI classification scale for CP, lesion network mapping, and tract based spatial statistics.
AIM 3) Explore potential differences in outcome patterns as related to patient characteristics and use findings
to guide the development of a highly sensitive multidimensional or composite measure that captures clinically
important areas of change for the child and family.
This revised study builds on new preliminary data and capitalizes on the expertise of an established
transdisciplinary team, a high-resource academic clinical environment, and a diverse Parent-Patient Council.
The study’s findings are likely to have high impact to improve future treatment of children with dystonic CP,
including identifying key patient characteristics associated with more or less positive outcomes and developing
a sensitive, efficient multidimensional composite measure to assess treatment responsiveness.

## Key facts

- **NIH application ID:** 10882869
- **Project number:** 1R01HD112563-01A1
- **Recipient organization:** BAYLOR COLLEGE OF MEDICINE
- **Principal Investigator:** Sruthi Pandipati Thomas
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $680,870
- **Award type:** 1
- **Project period:** 2024-09-01 → 2029-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10882869

## Citation

> US National Institutes of Health, RePORTER application 10882869, Intrathecal baclofen and pediatric dystonia. (1R01HD112563-01A1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10882869. Licensed CC0.

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