{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,22]],"date-time":"2026-07-22T14:30:47Z","timestamp":1784730647953,"version":"3.55.0"},"reference-count":21,"publisher":"BMJ","issue":"5","content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["BMJ Case Rep"],"accepted":{"date-parts":[[2021,4,7]]},"published-print":{"date-parts":[[2021,5]]},"abstract":"<jats:p>We report on a male subject with a diagnosis of Niemann-Pick type C (NPC). He received an experimental medicinal product intrathecally initially via lumbar puncture (LP) and eventually via intrathecal drug delivery device. Shortly after implantation, the device catheter migrated outside of the intrathecal space and coiled subcutaneously. The treatment continued via LP after removal of the device. A subdural haematoma developed after repeated LPs. It was surgically evacuated and the patient recovered with sequelae. Surgically implanted drug delivery devices are designed to bypass the blood\u2013brain barrier and deliver a medicinal product directly into the cerebrospinal fluid circulation. Their use has extended into the field of neurodegenerative disorders. Significant adverse events can occur at any given time after implantation including neurological injury, dislodgement or displacement of any of its components, infection and drug-related complications; all can significantly affect the quality of life of patients. Repeated LPs also carry significant risk.<\/jats:p>","DOI":"10.1136\/bcr-2021-241786","type":"journal-article","created":{"date-parts":[[2021,5,7]],"date-time":"2021-05-07T11:55:47Z","timestamp":1620388547000},"page":"e241786","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":4,"title":["Complications associated with intrathecal drug delivery in a paediatric patient with Niemann-Pick type C"],"prefix":"10.1136","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7771-5560","authenticated-orcid":false,"given":"Jose Ignacio Rodriguez","family":"Rodriguez Ciancio","sequence":"first","affiliation":[{"name":"Metabolic Medicine, Great Ormond Street Hospital for Children, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristian","family":"Aquilina","sequence":"additional","affiliation":[{"name":"Neurosurgery, Great Ormond Street Hospital for Children, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"239","published-online":{"date-parts":[[2021,5,7]]},"reference":[{"key":"2025082803221062000_14.5.e241786.1","doi-asserted-by":"crossref","DOI":"10.1186\/s13023-018-0785-7","article-title":"Consensus clinical management guidelines for Niemann-Pick disease type C","volume":"13","author":"Geberhiwot","year":"2018","journal-title":"Orphanet J Rare Dis"},{"key":"2025082803221062000_14.5.e241786.2","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-5-16"},{"key":"2025082803221062000_14.5.e241786.3","doi-asserted-by":"crossref","DOI":"10.1186\/s13023-019-0996-6","article-title":"Disease characteristics, prognosis and miglustat treatment effects on disease progression in patients with Niemann-Pick disease type C: an international, multicenter, retrospective chart review","volume":"14","author":"Pineda","year":"2019","journal-title":"Orphanet J Rare Dis"},{"key":"2025082803221062000_14.5.e241786.4","doi-asserted-by":"crossref","DOI":"10.1016\/j.ijpharm.2020.119440","article-title":"Recent advances in the treatment of Niemann Pick disease type C: a mini-review","volume":"584","author":"Matencio","year":"2020","journal-title":"Int J Pharm"},{"key":"2025082803221062000_14.5.e241786.5","doi-asserted-by":"crossref","first-page":"80","DOI":"10.1016\/j.ymgme.2019.11.007","article-title":"Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study","volume":"129","author":"Chen","year":"2020","journal-title":"Mol Genet Metab"},{"key":"2025082803221062000_14.5.e241786.6","doi-asserted-by":"publisher","DOI":"10.1038\/gim.2015.36"},{"key":"2025082803221062000_14.5.e241786.7","doi-asserted-by":"crossref","DOI":"10.1016\/j.ymgme.2020.07.002","article-title":"Safety of intrathecal delivery of recombinant human arylsulfatase A in children with metachromatic leukodystrophy: results from a phase 1\/2 clinical trial","volume":"131","author":"\u00cd Dali","year":"2020","journal-title":"Mol Genet Metab"},{"key":"2025082803221062000_14.5.e241786.8","unstructured":"K.K. 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