14800-DvRappard

64 Chapter 4 spinal fluid by an intrathecal catheter. To reach an adequate effect, the dose is increased in a stepwise manner in continuous delivery or, when repeated vanishing effect occurs, by a flexible program with periodic boluses. 13,14 During the last decades, experience with ITB has been increasing, and it is now known as an effective therapy to treat spasticity related problems in daily life in spastic cerebral palsy (SCP). 15-17 Positive effects in dyskinetic cerebral palsy have also been reported, but current evidence is limited. 18 ITB has recently been described to have beneficial effects in treatment of spasticity in progressive neurological diseases like MLD, 16,19 and in our center, ITB is frequently used as therapy in children with cerebral palsy and progressive neurological disorders. As little is known about the clinical course of ITB treatment in MLD, our aim is to describe the course of ITB treatment in juvenile MLD patients and contrast it with SCP patients. We were interested whether the progressive nature of the disease makes other dose adaptations necessary than normally applied in static encephalopathies in children and young adults. METHODS Design This study is a retrospective cross-sectional cohort study with matched-control SCP patients. Setting and participants All patients with juvenile MLD (diagnosis established by typical clinical andMRI findings, ASA activity and ARSA mutation analysis) who were treated with ITB in our center between February 2002 and December 2016, and had a baclofenpump for at least six months, were included. All children were non-walking (GMFC-MLD level 3 to 6). For the SMLD group, a matched control group of patients with SCP treated with ITB was composed, out of all ITB patients treated in our center. This SCP group was matched with the SMLD group for age, sex and functional mobility level (non-walking, GMFCS 4 and 5). Only the MLD patients with a spastic movement disorder were matched with SCP patients, as patients with spasticity seem to require different dosing than dyskinetic patients, 20 and the DMLD group (n=2) was too small to match.