@article{773c0d50d56d42fc96dd5981bc6f6599,
title = "The Effect of Intrathecal Baclofen in Dyskinetic Cerebral Palsy: The IDYS Trial",
abstract = "Objective Intrathecal baclofen treatment is used for the treatment of dystonia in patients with severe dyskinetic cerebral palsy; however, the current level of evidence for the effect is low. The primary aim of this study was to provide evidence for the effect of intrathecal baclofen treatment on individual goals in patients with severe dyskinetic cerebral palsy. Methods This multicenter, randomized, double-blind, placebo-controlled trial was performed at 2 university medical centers in the Netherlands. Patients with severe dyskinetic cerebral palsy (Gross Motor Functioning Classification System level IV-V) aged 4 to 24 years who were eligible for intrathecal baclofen were included. Patients were assigned by block randomization (2:2) for treatment with intrathecal baclofen or placebo for 3 months via an implanted microinfusion pump. The primary outcome was goal attainment scaling of individual treatment goals (GAS T score). A linear regression model was used for statistical analysis with study site as a covariate. Safety analyses were done for number and type of (serious) adverse events. Results Thirty-six patients were recruited from January 1, 2013, to March 31, 2018. Data for final analysis were available for 17 patients in the intrathecal baclofen group and 16 in the placebo group. Mean (standard deviation) GAS T score at 3 months was 38.9 (13.2) for intrathecal baclofen and 21.0 (4.6) for placebo (regression coefficient = 17.8, 95% confidence interval = 10.4-25.0, p <0.001). Number and types of (serious) adverse events were similar between groups. Interpretation Intrathecal baclofen treatment is superior to placebo in achieving treatment goals in patients with severe dyskinetic cerebral palsy. ANN NEUROL 2019",
keywords = "CHILDREN, DYSTONIA, CHOREOATHETOSIS, PREVALENCE, CHILDHOOD, VALIDITY, LIFE",
author = "Bonouvrie, {Laura A.} and Becher, {Jules G.} and Vles, {Johan S. H.} and Vermeulen, {R. Jeroen} and Karin Boeschoten and Geytenbeek, {Johanna J. M.} and {de Groot}, Vincent and {van de Pol}, {Laura A.} and {van Ouwerkerk}, {Willem J. R.} and Slot, {K. M.} and Peerdeman, {S. M.} and Strijers, {Rob L. M.} and Foncke, {Elisabeth M. J.} and Twisk, {Jos W. R.} and {van de Ven}, Peter and Dan Soudant and Sabine Fleuren and Teernstra, {Onno P.} and {IDYS Study Group}",
note = "Funding Information: This study was funded by the Phelps Stichting voor Spastici (project numbers2011037 and 99.047), het Rev-alidatiefonds (project number R2011032/), Kinderrevalidatie Fonds de Adriaanstichting (project number 11.02.17-2011/ 0035), and het Johanna KinderFonds (project number 2011/0035-357). Funding Information: This study was funded by the Phelps Stichting voor Spastici (project numbers2011037 and 99.047), het Revalidatiefonds (project number R2011032/), Kinderrevalidatie Fonds de Adriaanstichting (project number 11.02.17-2011/0035), and het Johanna KinderFonds (project number 2011/0035-357). The funders had no role in the study design, data collection, data analysis, data interpretation, or writing of the article. The corresponding author had full access to all study data, and all main authors (L.B., J.B., J.S.H.V., R.J.V., A.B.) had final responsibility for the decision to submit for publication. We thank Dr S. van der Vossen for her help in conducting measurements during the absence of L.A.B.; T. van Ams and S. de Vries for taking care of the logistics regarding study medication, appointments, and pump fillings; and H. Haberfehlner for her enthusiastic and innovative ideas. L.B., J.B., J.S.H.V., and R.J.V. contributed to the conception and design of the study; L.B., J.B., R.J.V., and A.B. contributed to the acquisition and analysis of data; L.B., J.B., J.S.H.V., R.J.V., and A.B. contributed to drafting the text and preparing the figures. IDYS study group members are, from the Amsterdam University Medical Center, Free University Amsterdam, Amsterdam, the Netherlands: Laura A. Bonouvri?, MD, Jules G. Becher, MD, Annemieke I. Buizer, MD, Karin Boeschoten, Johanna J. M. Geytenbeek, PhD, Vincent de Groot, MD (Rehabilitation Medicine), Laura A. van de Pol, MD (Child Neurology), Willem J. R. van Ouwerkerk, MD, K. M. Slot, MD, S. M. Peerdeman, MD (Neurosurgery), Rob L. M. Strijers, MD (Clinical Neurophysiology), Elisabeth M. J. Foncke, MD (Neurology), Jos W. R. Twisk, PhD, Peter van de Ven, PhD (Epidemiology and Biostatistics); and from Maastricht University Medical Center, Maastricht, the Netherlands: R. Jeroen Vermeulen, MD, Johan S. H. Vles, MD, Dan Soudant, MANP, Sabine Fleuren (Neurology), and Onno P. Teernstra, MD (Neurosurgery). Publisher Copyright: {\textcopyright} 2019 The Authors. Annals of Neurology published by Wiley Periodicals, Inc. on behalf of American Neurological Association.",
year = "2019",
month = jul,
doi = "10.1002/ana.25498",
language = "English",
volume = "86",
pages = "79--90",
journal = "Annals of Neurology",
issn = "0364-5134",
publisher = "Wiley",
number = "1",
}