PREDICT-ITB: Predicting response in children with dystonic CP to ITB - study protocol

Over 11,000 infants are diagnosed with cerebral palsy (CP) each year, with lifetime medical costs exceeding $1.4 million per person. Elevated muscle tone in CP, including dystonia and spasticity, significantly impairs function and quality of life. Intrathecal baclofen (ITB) is commonly used to manage dystonic CP, though evidence supporting its effectiveness is weak due to patient variability and study limitations. Spasticity may obscure dystonia symptoms, and factors like brain injury patterns and pain triggers are often overlooked in research. Despite uncertain effects on dystonia, ITB has shown benefits in pain relief, comfort, and caregiving ease. This proposal aims to evaluate ITB's overall impact on children with dystonic CP, identify responders, and develop a comprehensive outcome measure using a prospective cohort study.

We will conduct a prospective, observational study of 65 children with dystonia (Barry Albright Dystonia Scale (BADS) greater than 15) and CP who receive ITB. Changes in dystonia, spasticity, gross and fine motor function, and multiple patient-reported outcomes related to quality-of-life, depression, anxiety, pain and more. The primary analysis will use repeated measures models to estimate short and long-term changes from baseline in BADS scores at 3, 6 and 12-months. Secondary analysis will apply the same strategies to the other outcome measures. We will also conduct subgroup analysis and develop a multidimensional or composite measure.

Primary ethic approval was provided by the Baylor College of Medicine Institutional Review Board (H-54449). Results of the study will be disseminated via peer-reviewed presentations at scientific conferences and open access publication.

NCT06606574 ( clinicaltrials.gov ).

Unlike previous studies on ITB in dystonia and CP, a strength of this study is that it will directly measure effects of ITB beyond just dystonia, while also considering the child's co- existing spasticity if present, known triggers of dystonia, including pain, and CNS injury patterns contributing to dystonia.We consider multiple endpoints or the "total child" within the ICF Framework and whether concurrent therapeutic interventions appear to influence outcomes.A limitation of the study is the lack of randomization to placebo or blinding.