Academic Department of Neurosciences, Royal Hallamshire Hospital and University of Sheffield, Sheffield, UK.
Department of Infection, Immunity and Cardiovascular Disease, University of Sheffield, Sheffield, UK.
Cerebellum. 2021 Jun;20(3):454-461. doi: 10.1007/s12311-020-01222-7. Epub 2021 Jan 6.
Superficial siderosis describes haemosiderin deposition on the surface of the brain. When present on infratentorial structures, it can cause ataxia, sensorineural hearing loss and pyramidal signs. There is no proven treatment and patients experience slow progression of symptoms. Iron-chelating agents have been suggested as a therapeutic option and deferiprone is suited as it crosses the blood-brain barrier. However, deferiprone is reported to have a 1-2% risk of agranulocytosis. We performed a systematic review on treatment of infratentorial superficial siderosis with deferiprone based on PRISMA guidelines. Studies were included if in English or an English language translation was available, were about human subjects and referred to patients with ataxia. Studies were excluded if they did not possess an English translation, included animal studies or did not have ataxia. Studies were excluded if they discussed cerebral amyloid angiopathy or siderosis of other regions. Eleven papers were included. We identified 69 patients. Seventeen patients (25%) discontinued the drug. The most encountered adverse effect was anaemia (21.7%). Neutropaenia was observed in 8.7% and agranulocytosis in 5.8% of patients. Clinically, response varied, and stability or improvement was seen across neurological domains in 6 studies while 5 showed a mixed response. On imaging, 13 (28.9%) patients improved, 24 (53.3%) stabilised and 8 (17.8%) deteriorated. A prospective international centralised register of patients should be developed to inform the design and conduct of a multicentre, placebo-controlled, randomised clinical trial to evaluate the efficacy of deferiprone. The evidence from this systematic review is that deferiprone is a promising intervention.
脑表铁沉积症是指铁蛋白在脑表面沉积。当发生于后颅窝结构时，可引起共济失调、感音神经性聋和锥体束征。目前尚无有效的治疗方法，患者症状逐渐加重。铁螯合剂已被提议作为一种治疗选择，而二乙撑三胺五乙酸（DFP）适合作为药物，因为它可以穿过血脑屏障。然而，DFP 有 1-2%的粒细胞缺乏症风险。我们根据 PRISMA 指南，对 DFP 治疗后颅窝表浅铁沉积症进行了系统评价。纳入标准为：英语或英语翻译版本、研究对象为人以及涉及共济失调患者的研究。排除标准为：无英语翻译、动物研究或不伴有共济失调的研究；涉及脑淀粉样血管病或其他部位铁沉积症的研究。共纳入 11 篇文献。我们共识别出 69 例患者。17 例（25%）患者停药。最常见的不良反应是贫血（21.7%）。中性粒细胞减少症发生率为 8.7%，粒细胞缺乏症发生率为 5.8%。临床上，反应不一，6 项研究中所有神经学领域均显示稳定或改善，5 项研究显示混合反应。影像学方面，13 例（28.9%）患者改善，24 例（53.3%）稳定，8 例（17.8%）恶化。应开发一个前瞻性国际集中患者登记处，为设计和开展多中心、安慰剂对照、随机临床试验提供信息，以评估 DFP 的疗效。本系统评价的证据表明，DFP 是一种有前途的干预措施。
