Background: Dystonic cerebral palsy is primarily caused by damage to the basal ganglia and central cortex. The\r\ndaily care of these patients can be difficult due to dystonic movements. Intrathecal baclofen treatment is a\r\npotential treatment option for dystonia and has become common practice. Despite this widespread adoption, high\r\nquality evidence on the effects of intrathecal baclofen treatment on daily activities is lacking and prospective data\r\nare needed to judge the usefulness and indications for dystonic cerebral palsy. The primary aim of this study is to\r\nprovide level one clinical evidence for the effects of intrathecal baclofen treatment on the level of activities and\r\nparticipation in dystonic cerebral palsy patients. Furthermore, we hope to identify clinical characteristics that will\r\npredict a beneficial effect of intrathecal baclofen in an individual patient.\r\nMethods/Design: A double blind placebo-controlled multi-center randomized clinical trial will be performed in\r\n30 children with dystonic cerebral palsy. Patients aged between 4 and 25 years old with a confirmed diagnosis of\r\ndystonic cerebral palsy, Gross Motor Functioning Classification System level IV or V, with lesions in the cerebral\r\nwhite matter, basal ganglia or central cortex and who are eligible for intrathecal baclofen treatment will be\r\nincluded. Group A will receive three months of continuous intrathecal baclofen treatment and group B will receive\r\nthree months of placebo treatment, both via an implanted pump. After this three month period, all patients will\r\nreceive intrathecal baclofen treatment, with a follow-up after nine months. The primary outcome measurement will\r\nbe the effect on activities of and participation in daily life measured by Goal Attainment Scaling. Secondary outcome\r\nmeasurements on the level of body functions include dystonia, spasticity, pain, comfort and sleep-related breathing\r\ndisorders. Side effects will be monitored and we will study whether patient characteristics influence outcome.\r\nDiscussion: The results of this study will provide data for evidence-based use of intrathecal baclofen in dystonic\r\ncerebral palsy.
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