{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,26]],"date-time":"2025-10-26T14:56:05Z","timestamp":1761490565604,"version":"3.37.3"},"reference-count":15,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2018,5,27]],"date-time":"2018-05-27T00:00:00Z","timestamp":1527379200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Develop Med Child Neuro"],"published-print":{"date-parts":[[2019,2]]},"abstract":"Metachromatic leukodystrophy (MLD<\/jats:styled-content>) is a rare progressive neurological disorder, often accompanied by motor impairments that are challenging to treat. In this case series, we report the course of treatment with intrathecal baclofen (ITB<\/jats:styled-content>), aimed at improving daily care and comfort in children and young adults with MLD<\/jats:styled-content>. All patients with MLD<\/jats:styled-content> in our centre on ITB<\/jats:styled-content> treatment for a minimum of 6 months were included (n<\/jats:italic>=10; 4 males, 6 females; mean age 10y 8mo [range 6\u201324y]). Eight patients had MLD<\/jats:styled-content> with a predominant spastic movement disorder (sMLD<\/jats:styled-content>) and two were mainly dyskinetic. Patients with sMLD<\/jats:styled-content> were compared with matched patients with spastic cerebral palsy (CP<\/jats:styled-content>). Complication rates related to ITB<\/jats:styled-content> treatment were similar in both groups. ITB<\/jats:styled-content> treatment course in the first 6 months after pump implantation appears to show more dose increase in most patients MLD<\/jats:styled-content>, compared to patients with spastic CP<\/jats:styled-content>. This may be due to the progressive disease in MLD<\/jats:styled-content>. ITB<\/jats:styled-content> is a feasible therapy to improve daily care and comfort in patients with MLD<\/jats:styled-content> and should therefore be considered early.<\/jats:p><\/jats:sec>What this paper adds<\/jats:title>\n\n\nIntrathecal baclofen (ITB) is a feasible therapy to improve comfort and daily care in children and young people with metachromatic leukodystrophy (MLD).<\/jats:p><\/jats:list-item>\n\nIn the first 6 months of ITB treatment, MLD seems to show more dose increase compared to spastic cerebral palsy.<\/jats:p><\/jats:list-item>\n<\/jats:list>\n<\/jats:p><\/jats:sec>","DOI":"10.1111\/dmcn.13919","type":"journal-article","created":{"date-parts":[[2018,5,28]],"date-time":"2018-05-28T06:10:05Z","timestamp":1527487805000},"page":"232-235","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":14,"title":["Intrathecal baclofen in metachromatic leukodystrophy"],"prefix":"10.1111","volume":"61","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6265-856X","authenticated-orcid":false,"given":"Nikki","family":"van der Veldt","sequence":"first","affiliation":[{"name":"Department of Rehabilitation Medicine VU University Medical Center Amsterdam Movement Sciences Amsterdam the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Diane F","family":"van Rappard","sequence":"additional","affiliation":[{"name":"Department of Child Neurology Center for Childhood White Matter Disorders VU University Medical Center and Amsterdam Neuroscience Amsterdam the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Laura A","family":"van de Pol","sequence":"additional","affiliation":[{"name":"Department of Child Neurology Center for Childhood White Matter Disorders VU University Medical Center and Amsterdam Neuroscience Amsterdam the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marjo S","family":"van der Knaap","sequence":"additional","affiliation":[{"name":"Department of Child Neurology Center for Childhood White Matter Disorders VU University Medical Center and Amsterdam Neuroscience Amsterdam the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Willem J R","family":"van Ouwerkerk","sequence":"additional","affiliation":[{"name":"Department of Neurosurgery VU University Medical Center Amsterdam the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jules G","family":"Becher","sequence":"additional","affiliation":[{"name":"Department of Rehabilitation Medicine VU University Medical Center Amsterdam Movement Sciences Amsterdam the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nicole I","family":"Wolf","sequence":"additional","affiliation":[{"name":"Department of Child Neurology Center for Childhood White Matter Disorders VU University Medical Center and Amsterdam Neuroscience Amsterdam the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5662-2843","authenticated-orcid":false,"given":"Annemieke I","family":"Buizer","sequence":"additional","affiliation":[{"name":"Department of Rehabilitation Medicine VU University Medical Center Amsterdam Movement Sciences Amsterdam the Netherlands"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2018,5,27]]},"reference":[{"doi-asserted-by":"publisher","key":"e_1_2_6_2_1","DOI":"10.1038\/bmt.2008.275"},{"key":"e_1_2_6_3_1","first-page":"729","article-title":"Developing therapeutic approaches for metachromatic leukodystrophy","volume":"7","author":"Patil SA","year":"2013","journal-title":"Drug Des Devel 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