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Open Access Highly Accessed Study protocol

Intrathecal baclofen treatment in dystonic cerebral palsy: a randomized clinical trial: the IDYS trial

Laura A Bonouvrié1*, Jules G Becher1, Johannes SH Vles2, Karin Boeschoten1, Dan Soudant2, Vincent de Groot1, Willem JR van Ouwerkerk3, Rob LM Strijers4, Elisabeth Foncke5, Joke Geytenbeek1, Peter M van de Ven6, Onno Teernstra7 and R Jeroen Vermeulen8

Author Affiliations

1 Department of Rehabilitation Medicine, VU University Medical Center, Postbus 7057, 1007, MB Amsterdam, The Netherlands

2 Department of Child Neurology, Maastricht University Medical Center, Amsterdam, The Netherlands

3 Department of Neurosurgery, VU University Medical Center, Amsterdam, The Netherlands

4 Department of Clinical Neurophysiology, VU University Medical Center, Amsterdam, The Netherlands

5 Department of Neurology, VU University Medical Center, Amsterdam, The Netherlands

6 Department of Epidemiology and Biostatistics, VU University Medical Center, Amsterdam, The Netherlands

7 Department of Neurosurgery, Maastricht University Medical Center, Amsterdam, The Netherlands

8 Department of Child Neurology, Neuroscience Campus Amsterdam, VU University Medical Center, Amsterdam, The Netherlands

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BMC Pediatrics 2013, 13:175  doi:10.1186/1471-2431-13-175

Published: 28 October 2013

Abstract

Background

Dystonic cerebral palsy is primarily caused by damage to the basal ganglia and central cortex. The daily care of these patients can be difficult due to dystonic movements. Intrathecal baclofen treatment is a potential treatment option for dystonia and has become common practice. Despite this widespread adoption, high quality evidence on the effects of intrathecal baclofen treatment on daily activities is lacking and prospective data are needed to judge the usefulness and indications for dystonic cerebral palsy. The primary aim of this study is to provide level one clinical evidence for the effects of intrathecal baclofen treatment on the level of activities and participation in dystonic cerebral palsy patients. Furthermore, we hope to identify clinical characteristics that will predict a beneficial effect of intrathecal baclofen in an individual patient.

Methods/Design

A double blind placebo-controlled multi-center randomized clinical trial will be performed in 30 children with dystonic cerebral palsy. Patients aged between 4 and 25 years old with a confirmed diagnosis of dystonic cerebral palsy, Gross Motor Functioning Classification System level IV or V, with lesions in the cerebral white matter, basal ganglia or central cortex and who are eligible for intrathecal baclofen treatment will be included. Group A will receive three months of continuous intrathecal baclofen treatment and group B will receive three months of placebo treatment, both via an implanted pump. After this three month period, all patients will receive intrathecal baclofen treatment, with a follow-up after nine months. The primary outcome measurement will be the effect on activities of and participation in daily life measured by Goal Attainment Scaling. Secondary outcome measurements on the level of body functions include dystonia, spasticity, pain, comfort and sleep-related breathing disorders. Side effects will be monitored and we will study whether patient characteristics influence outcome.

Discussion

The results of this study will provide data for evidence-based use of intrathecal baclofen in dystonic cerebral palsy.

Trial registration

Nederlands Trial Register, NTR3642

Keywords:
Cerebral palsy; Dystonia; Dyskinesia; Goal attainment scaling; Intrathecal baclofen; Randomized controlled trial