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

Abstract

BackgroundDystonic 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/DesignA 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.DiscussionThe results of this study will provide data for evidence-based use of intrathecal baclofen in dystonic cerebral palsy.Trial registrationNederlands Trial Register, NTR3642

Understanding the reasons for delayed referral for intrathecal baclofen therapy in pediatric patients with severe spasticity

“…Many of our patients have had multiple orthopedic surgeries for contractures that have developed from poorly controlled spasticity. Pharmacological therapies that are initially tried are reported to have limited effect [6,19]. Interestingly, our data shows a much greater delay in patients who have cerebral palsy due to perinatal causes than in patients who had brain or spine injuries later in life.…”

“…Pharmacological, non-invasive treatment of spastic, dystonic, and mixed-movement types of movement disorder has been reported to be rather ineffective in most cases [6]. This includes botulinum toxin injections, alcohol blocks, oral baclofen as well as other oral medications.…”

CXCL12 chemokine and GABA neurotransmitter systems crosstalk and their putative roles

“…Baclofen is currently approved for the treatment of spasticity in patients with spinal cord injury, cerebral palsy, traumatic brain injury, multiple sclerosis and other disorders (Plassat et al, 2004; Guglani and Lodha, 2007; Kolaski and Logan, 2008; Rekand and Gronning, 2011). Recently, it has been used in the treatment of alcohol dependence and withdrawal (Addolorato et al, 2006).…”

Intrathecal baclofen in dyskinetic cerebral palsy: effects on function and activity

“…One such study is underway, which will hopefully shed light on the effects of ITB and their magnitude in dyskinetic CP. 26 preparation, and/or publication decisions. The authors have stated that they had no interests that might be perceived as posing a conflict or bias.…”