--- title: "Prospective study protocol: intrathecal baclofen and ventral-dorsal rhizotomy for dystonia in chil" id: "bmj-open-15-investigating-the-effects-of-intrathecal-baclofen-and-ventral-dorsal-rhizotomy" canonical_url: "https://medichelpline.com/clinical-feed/bmj-open-15-investigating-the-effects-of-intrathecal-baclofen-and-ventral-dorsal-rhizotomy" content_type: "clinical_feed_article" specialty: "Neurology" source_name: "BMJ Open" source_url: "http://bmjopen.bmj.com/cgi/content/short/16/10/e123926?rss=1" published_at: "2026-10-06T11:59:03.000Z" evidence_level: "Journal Feed" license: "CC-BY-NC-4.0 / Informational Use" --- # Prospective study protocol: intrathecal baclofen and ventral-dorsal rhizotomy for dystonia in chil ## Provenance & Clinical Metadata - **Canonical URL:** https://medichelpline.com/clinical-feed/bmj-open-15-investigating-the-effects-of-intrathecal-baclofen-and-ventral-dorsal-rhizotomy - **Specialty:** [Neurology](https://medichelpline.com/clinical-feed/neurology.md) - **Primary Source:** BMJ Open - **Source URL:** [Original Journal Publication](http://bmjopen.bmj.com/cgi/content/short/16/10/e123926?rss=1) - **Published At:** 2026-10-06T11:59:03.000Z - **Evidence Rating:** Journal Feed ## Executive GIST (TL;DR) - This protocol describes a prospective observational clinical trial (PREDICT-Dystonia) evaluating two neurosurgical treatments for dystonia in children with cerebral palsy: **intrathecal baclofen (ITB)** and **ventral-dorsal rhizotomy (VDR)**. - The study targets children with moderate to severe dystonia defined as a **Barry Albright Dystonia Scale (BADS)** score >14. - Planned enrollment is 52 children across three US pediatric centers: Texas Children's Hospital (Houston), Lurie Children's Hospital (Chicago) and C.S. Mott Children's Hospital (Ann Arbor). - Treatment groups are ITB (n=35) and VDR (n=17) as observed in routine clinical care rather than randomized assignment. - Primary outcome is change in BADS scores from baseline at 3, 6 and 12 months using repeated measures models. - Secondary outcomes include measures of **spasticity**, gross and fine motor function, and multiple patient-reported outcomes covering quality of life, participation, depression, anxiety and pain. - Detailed neuroimaging-based characterisation of brain malformation and injury patterns will be collected to support subgroup analyses. - Secondary analyses will assess other outcome measures over time and explore child-level characteristics associated with positive response to ITB or VDR; a multidimensional or composite outcome measure will be developed. - Study received primary ethics approval from Baylor College of Medicine IRB (H-54449); caregivers provided consent and participants with capacity were asked to assent. - Results will be disseminated via scientific conferences and open access publication; clinical trial registration: NCT06606574. ## Clinical Analysis & Structured Key Points Introduction Dystonia, a form of elevated muscle tone in CP, significantly impairs function and quality of life. Multiple neurosurgical interventions are used to reduce dystonia, including intrathecal baclofen (ITB) and ventral dorsal rhizotomy (VDR). ITB is used most often for moderate to severe generalised dystonia in CP; however, the evidence supporting its effectiveness is weak due to patient variability and study limitations. Further, VDR is becoming more prevalent but there is minimal data available on its outcomes. This proposal aims to evaluate ITB and VDR's ability to reduce quantitative and functionally impactful dystonia as well as reduce spasticity, improve motor function, improve mood and improve quality of life. Secondary analysis will focus on identifying child characteristics of a positive response to ITB and VDR to allow for better patient selection. Methods and analysis We will conduct a prospective observational study of 52 children with CP and moderate-severe dystonia (Barry Albright Dystonia Scale (BADS) greater than 14) who receive ITB (n=35) and VDR (n=17). Children will be enrolled at three sites in the USA: Texas Children's Hospital (Houston), Lurie Children's Hospital (Chicago) and C.S. Mott Children's Hospital (Ann Arbor). Changes will be measured in dystonia, spasticity, gross and fine motor function and multiple patient-reported outcomes related to quality of life, participation, depression, anxiety and pain. Children enrolled in the study will undergo detailed characterisation of brain malformation and injury patterns. The primary analysis will use repeated measures models to estimate changes from baseline in BADS scores at 3, 6 and 12 months. Secondary analyses will apply the same strategies to the other outcome measures. Subgroup analyses will investigate unique characteristics of children with a positive response to either intervention. A multidimensional or composite measure will also be created. Ethics and dissemination Primary ethics approval was provided by the Baylor College of Medicine Institutional Review Board (H-54449). Primary caregivers of all participants were consented for the study. Participants with capacity were asked to assent. Results of the study will be disseminated via peer-reviewed presentations at scientific conferences and open access publication. 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