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In total, 510 MRI scans were collected from 12 intravenous (IV)\u00a0rhASA\u2010treated children with MLD, 24 IT rhASA\u2010treated children with MLD, 32 children with untreated MLD, and 156 normally developing children. Linear mixed models were fitted to analyze the time courses of gray matter (GM) volume and fractional anisotropy (FA) in the posterior limb of the internal capsule. Time courses for demyelination load and FA in the centrum semiovale were visualized using locally estimated scatterplot smoothing regression curves. All assessed imaging parameters demonstrated structural evidence of neurological deterioration in children with MLD. GM volume was significantly lower at follow\u2010up (median duration, 104\u2009weeks) in IV rhASA\u2010treated versus IT rhASA\u2010treated children. GM volume decline over time was steeper in children receiving low\u2010dose (10 or 30\u2009mg) versus high\u2010dose (100\u2009mg) IT rhASA. Similar effects were observed for demyelination. FA in the posterior limb of the internal capsule showed a higher trend over time in IT rhASA\u2010treated versus children with untreated MLD, but FA parameters were not different between children receiving the low doses\u00a0versus those receiving the high dose. GM volume in IT rhASA\u2010treated children showed a strong positive correlation with 88\u2010item Gross Motor Function Measure score over time. In some children with MLD, IT administration of high\u2010dose rhASA may delay neurological deterioration (assessed using MRI), offering potential therapeutic benefit.<\/jats:p>","DOI":"10.1002\/jimd.12706","type":"journal-article","created":{"date-parts":[[2024,2,7]],"date-time":"2024-02-07T01:26:07Z","timestamp":1707269167000},"page":"778-791","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["The effect of intrathecal recombinant arylsulfatase A therapy on structural brain magnetic resonance imaging in children with metachromatic leukodystrophy"],"prefix":"10.1002","volume":"47","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2706-7163","authenticated-orcid":false,"given":"Samuel","family":"Groeschel","sequence":"first","affiliation":[{"name":"Department of Pediatric Neurology University Children's Hospital T\u00fcbingen  T\u00fcbingen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2945-6784","authenticated-orcid":false,"given":"Shanice","family":"Beerepoot","sequence":"additional","affiliation":[{"name":"Amsterdam Leukodystrophy Center, Department of Child Neurology Emma Children's Hospital, Amsterdam University Medical Centers, Vrije Universiteit and Amsterdam Neuroscience\u2014Cellular and Molecular Mechanisms, Vrije Universiteit  Amsterdam Netherlands"},{"name":"Center for Translational Immunology University Medical Center Utrecht  Utrecht Netherlands"},{"name":"Princess M\u00e1xima Center for Pediatric Oncology  Utrecht Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lucas Bastian","family":"Amedick","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology University Children's Hospital T\u00fcbingen  T\u00fcbingen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ingeborg","family":"Kr\u04d3geloh\u2010Mann","sequence":"additional","affiliation":[{"name":"Department of Pediatric Neurology University Children's Hospital T\u00fcbingen  T\u00fcbingen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jing","family":"Li","sequence":"additional","affiliation":[{"name":"Takeda Development Center Americas, Inc.  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