{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T10:32:46Z","timestamp":1790764366482,"version":"4.1.0"},"reference-count":10,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2019,10,16]],"date-time":"2019-10-16T00:00:00Z","timestamp":1571184000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2019,10,16]],"date-time":"2019-10-16T00:00:00Z","timestamp":1571184000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J Neurol"],"published-print":{"date-parts":[[2020,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n<jats:sec>\n<jats:title>Objective<\/jats:title>\n<jats:p>Deferiprone is an iron chelator that has recently been used to treat patients with infratentorial superficial siderosis (iSS). It is considered to have a generally favourable safety profile but concerns have been raised due to the risk of agranulocytosis. We aimed to evaluate the safety and tolerability of oral deferiprone as a treatment for patients with iSS.<\/jats:p>\n<\/jats:sec>\n<jats:sec>\n<jats:title>Methods<\/jats:title>\n<jats:p>We present a case series of 10 consecutive patients presenting with classical iSS treated with deferiprone.<\/jats:p>\n<\/jats:sec>\n<jats:sec>\n<jats:title>Results<\/jats:title>\n<jats:p>Ten patients were followed up for a mean period of 2.3\u00a0years (range 0.5\u20135.5\u00a0years). Four patients (40%) were withdrawn from treatment because of treatment-related side effects. The\u00a0reasons for treatment discontinuation were neutropenic sepsis (<jats:italic>n<\/jats:italic>\u2009=\u20093) and fatigue (<jats:italic>n<\/jats:italic>\u2009=\u20091). In 2 out of the 3 cases\u00a0of neutropenic sepsis, patients initially developed neutropenia\u00a0without sepsis. The mean time to neutropenic sepsis following deferiprone was 1.2\u00a0years (range 0.3\u20132.5) with mean neutrophil count of 0.4 (range 0.3\u20130.5). Six patients (60%) reported no change in neurological function while on treatment, and four patients (40%) reported that\u00a0their condition deteriorated.<\/jats:p>\n<\/jats:sec>\n<jats:sec>\n<jats:title>Conclusions<\/jats:title>\n<jats:p>Deferiprone was poorly tolerated, with 40% of patients withdrawing from treatment, most commonly due to neutropenic sepsis, after an average of 2\u00a0years on treatment. This study increases the number of reported cases of agranulocytosis in patients with iSS treated with deferiprone. Clinicians treating iSS patients with deferiprone should be aware that this drug\u00a0has a\u00a0potentially life-threatening side effect\u00a0of neutropenic sepsis, and should\u00a0ensure that appropriate haematological\u00a0monitoring is in place.<\/jats:p>\n<\/jats:sec>","DOI":"10.1007\/s00415-019-09577-6","type":"journal-article","created":{"date-parts":[[2019,10,16]],"date-time":"2019-10-16T19:13:18Z","timestamp":1571253198000},"page":"239-243","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":26,"title":["Risks associated with oral deferiprone in the treatment of infratentorial superficial siderosis"],"prefix":"10.1007","volume":"267","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9921-9130","authenticated-orcid":false,"given":"Y.","family":"Sammaraiee","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"G.","family":"Banerjee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"S.","family":"Farmer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B.","family":"Hylton","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"P.","family":"Cowley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"P.","family":"Eleftheriou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J.","family":"Porter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"D. 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The authors report no disclosures related to the manuscript.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflicts of interest"}},{"value":"The project involved a retrospective review of routinely collected clinical data from March 2013 until January 2019, and was approved as a clinical service evaluation by the Clinical Governance department of the National Hospital for Neurology and Neurosurgery.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}}]}}