How many patients are eligible for disease-modifying treatment in Alzheimer's disease? A French national observational study over 5 years

How many patients are eligible for disease-modifying treatment in Alzheimer's disease? A French national observational study over 5 years
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DOI:
10.1136/bmjopen-2019-029663
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发表时间:
2019-06-01
期刊:
影响因子:
2.9
通讯作者:
Dubois, Bruno
Dubois, Bruno
中科院分区:
医学3区
文献类型:
--
作者:
Epelbaum, Stephane;Paquet, Claire;Dubois, Bruno

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目的我们的目的是研究前驱期患者的流行病学谁是有资格的临床试验与diseasemodifying therapies.Settings我们分析了两个大型的互补数据库,研究发病率和特征,这一人群在全国范围内在法国从2014年至2018年。国家阿尔茨海默病数据库包含来自357个记忆中心和90名私人神经学家的数据。分析了2014年至2018年的数据。参与者包括50-85岁的AD患者,他们的简易精神状态检查(MMSE)评分>= 20。我们排除了混合型和非AD neurocognitive disorders.Primary结局指标描述性统计的人群的利益是主要measure.Results在国家阿尔茨海默病数据库,550 198例患者进行了评估。其中,72174(13.1%)被诊断为AD,MMSE ≥ 20。使用校正AD临床诊断的特异性,我们估计约50 000(9.1%)有前驱或轻度AD。在11个法国专家记忆中心的联合电子临床记录数据库中,通过使用脑脊液AD生物标志物认证的前驱期或轻度AD的诊断可以在14596例患者中的195例(1.3%)中建立。结论AD在2014年至2018年在法国的前驱期或轻度痴呆阶段不常被诊断。诊断很少依赖于病理生理学标记,即使在专家记忆中心。国家数据库将是有价值的,以监测早期AD诊断疗效记忆中心时,疾病修饰治疗变得可用。
Objective We aimed to study the epidemiology of the prodromal patients who are eligible for clinical trials with diseasemodifying therapies.Settings We analysed two large complementary databases to study the incidence and characteristics of this population on a nationwide scope in France from 2014 to 2018. The National Alzheimer Database contains data from 357 memory centres and 90 private neurologists. Data from 2014 to 2018 have been analysed.Participants Patients, 50-85 years old, diagnosed with AD who had an Mini-Mental State Exam (MMSE) score of >= 20 were included. We excluded patients with mixed and non-AD neurocognitive disorders.Primary outcome measure Descriptive statistics of the population of interest was the primary measure.Results In the National Alzheimer Database, 550 198 patients were assessed. Among them, 72 174 (13.1%) were diagnosed with AD and had an MMSE >= 20. Using corrections for specificity of clinical diagnosis of AD, we estimated that about 50 000 (9.1%) had a prodromal or mild AD. In the combined electronic clinical records database of 11 French expert memory centres, a diagnosis of prodromal or mild AD, certified by the use of cerebrospinal fluid AD biomarkers, could be established in 195 (1.3%) out of 14 596 patients.Conclusions AD was not frequently diagnosed at a prodromal or mild dementia stage in France in 2014 to 2018. Diagnosis rarely relied on a pathophysiological marker even in expert memory centres. National databases will be valuable to monitor early stage AD diagnosis efficacy in memory centres when a disease-modifying treatment becomes available.