Lancet neurology
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Randomized Controlled Trial
Safety and efficacy of pitolisant in children aged 6 years or older with narcolepsy with or without cataplexy: a double-blind, randomised, placebo-controlled trial.
Narcolepsy is a life-long disorder characterised by excessive daytime sleepiness and cataplexy, often arising in childhood or adolescence. Pitolisant, a selective histamine H3 receptor inverse agonist, has been approved in Europe and USA for adults with narcolepsy with or without cataplexy, with a favourable safety profile. This phase 3 study aimed to assess the safety and efficacy of pitolisant in children with narcolepsy with or without cataplexy. ⋯ Bioprojet.
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Multiple sclerosis is often diagnosed in patients who are planning on having children. Although multiple sclerosis does not negatively influence most pregnancy outcomes, less is known regarding the effects of fetal exposure to novel disease-modifying therapies (DMTs). The withdrawal of some DMTs during pregnancy can modify the natural history of multiple sclerosis, resulting in a substantial risk of pregnancy-related relapse and disability. ⋯ Emerging data reporting outcomes in neonates exposed to DMTs in utero and through breastfeeding will allow for more careful and individualised treatment decisions. This emerging research is particularly important to guide decision making in women with high disease activity or who are treated with DMTs associated with risk of discontinuation rebound. As increasing data are generated in this field, periodic updates will be required to provide the most up to date guidance on how best to achieve multiple sclerosis stability during pregnancy and post partum, balanced with fetal and newborn safety.
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Basilar artery occlusion is a rare and severe condition. The effectiveness of endovascular thrombectomy in patients with basilar artery occlusion was unclear until recently, because these patients were excluded from most trials of endovascular thrombectomy for large-vessel occlusion ischaemic stroke. ⋯ The Basilar Artery International Cooperation Study (BASICS) and the Basilar Artery Occlusion Endovascular Intervention versus Standard Medical Treatment (BEST) trials, specifically designed to investigate the benefit of thrombectomy in patients with basilar artery occlusion, did not find significant evidence of a benefit of endovascular thrombectomy in terms of disability outcomes at 3 months after stroke. However, these trials suggested a potential benefit of endovascular thrombectomy in patients presenting with moderate-to-severe symptoms. Subsequently, the Endovascular Treatment for Acute Basilar Artery Occlusion (ATTENTION) and the Basilar Artery Occlusion Chinese Endovascular (BAOCHE) trials, which compared endovascular thrombectomy versus medical therapy within 24 h of onset, showed clear benefit of endovascular thrombectomy in reducing disability and mortality, particularly in patients with moderate-to-severe symptoms. The risk of intracranial haemorrhage with endovascular thrombectomy was similar to the risk in anterior circulation stroke. Thrombectomy was beneficial regardless of age, baseline characteristics, the presence of intracranial atherosclerotic disease, and time from symptom onset to randomisation. Therefore, the question of whether endovascular thrombectomy is beneficial in basilar artery occlusion now appears to be settled in patients with moderate-to-severe symptoms, and endovascular thrombectomy should be offered to eligible patients. WHERE NEXT?: Key outstanding issues are the potential benefits of endovascular thrombectomy in patients with mild symptoms, the use of intravenous thrombolysis in an extended time window (ie, after 4·5 h of symptom onset), and the optimal endovascular technique for thrombectomy. Dedicated training programmes and automated software to assist with the assessment of imaging prognostic markers could be useful in the selection of patients who might benefit from endovascular thrombectomy. Large international research networks should be built to address knowledge gaps in this field and allow the conduct of clinical trials with fast and consecutive enrolment and a diverse ethnic representation.