intrathecal baclofen and pediatric dystonia

Conditions: Dystonic Cerebral Palsy

Summary: the goal of this clinical trial is to better understand the effects of intrathecal baclofen (itb) on children with dystonic cerebral palsy (cp). the main questions this study aims to answer are: (1) determine if itb reduces dystonia while identifying other potential benefits, (2) identify the characteristics of children with the best response to itb (3) develop a holistically representative composite outcome measure for dystonic cp. this study will evaluate patient improvement by using a standardized titration, or medication management, protocol to gradually increase the childs itb dosages over a 12-month period until they achieve maximum benefit with minimal to no side effects. this titration protocol mimics what is currently done through routine care but with more precision. this study will also directly measure the global effects of itb, taking into account spasticity, known dystonia triggers (e.g. pain), and patterns of cns injury that cause dystonia. participants will: 1. complete a total of 4 additional clinic visits outside usual care. these appointments will be with physical and occupational therapists as well as the study pi to complete evaluations for dystonia, spasticity, and function. 2. complete several questionnaires at these visits. the total duration of the study for an individual child will be 12 months.

cerebral palsy (cp) is the most common cause of physical disability in childhood and affects \~1 in 323 in the us per the cdc. the lifetime medical costs for an individual with cp was expected to be \~$1.4 million (inflation adjusted). an effective way to improve quality of life and decrease disease burden, both in childhood and adulthood, is to significantly reduce or eliminate abnormal tone. as muscle tone subsides, it is easier to move the body both passively and actively. a reduction in tone can improve quality of life, reduce pain, ease the burden of daily care and performance of day-to-day activities, and reduce healthcare costs. intrathecal baclofen ( itb) is used widely to manage dystonia in cp even though the data on exact efficacy is mixed. therefore, the goal of this study, is to understand the effects of itb on a heterogenous population of children with dystonic cp. the goal of the proposed research is to complete a longitudinal prospective cohort study investigating the effects of intrathecal baclofen (itb) on children with dystonic cerebral palsy (cp). this 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. specifically, this protocol aims to determine the 12-month effects of itb in a cohort of 65 clinically representative, itb naïve, children with dystonic cp using a standardized titration protocol. additionally, this protocol aims to complete a detailed characterization of brain malformation and injury patterns in children with dystonic cp and explore potential differences in outcome patterns as related to patient characteristics and use findings to guide development of a sensitive multidimensional or composite measure that captures clinically important areas of change for the child and family. our study site will be texas children's hospital (tch) in houston, tx, our pi sruthi thomas' home institution. tch has over 860 active patients with cp and just over 200 patients with itb. houston, tx, is the most diverse city in the united states, allowing us to recruit a very diverse patient population based on ethnicity and socioeconomic groups.

Eligibility criteria:
inclusion criteria: * child must be large enough to have an itb pump implanted (typically \~18 kg/4 years old) * they have a diagnosis of cp * presence of dystonia verified with a documented hypertonia assessment tool (hat) and they must have a barry-albright dystonia scale (bads) score \&gt;15 * they have been identified by a physician for itb treatment for tone management and the family/child have agreed to proceed with implantation; note: an itb trial dose is not necessary to qualify * child and family are willing to participate in full schedule of formal assessments, spanning pre-implantation, every 2 weeks until ideal intrathecal baclofen pump dosing is reached, and follow-up assessments exclusion criteria: * emergent need for itb such as status dystonicus or paroxysmal sympathetic hyperactivity * botulinum injections within 3 months or phenol injections within 6 months of enrollment * foster care placement or incarceration

Keywords: intrathecal baclofen spasticity dystonia cerebral palsy