欧洲额颞叶变性相关综合征患者入住养老院的预测因素和生存率。
Predictors of Care Home Admission and Survival Rate in Patients With Syndromes Associated With Frontotemporal Lobar Degeneration in Europe.
机构信息
From the Department of Clinical and Experimental Sciences (B.B.), University of Brescia; Department of Continuity of Care and Frailty (B.B., A.C.A.), ASST Spedali Civili, Brescia; Medical and Genomic Statistics Unit (B.T., M.G.), Department of Brain and Behavioural Sciences, University of Pavia, Italy; Division of Neurogeriatrics (C.G.), Department NVS, Karolinska Institutet, Solna; Unit for Hereditary Dementia (C.G.), Theme Inflammation and Aging, Karolinska University Hospital-Solna, Stockholm, Sweden; Research Unit of Clinical Medicine (J.K., S.A.-S., A.M.P.), Neurology, University of Oulu; MRC (J.K., A.M.P.), Oulu University Hospital; Neurocenter (J.K.), Neurology, Oulu University Hospital, Finland; Department of Neurology (A.C.L., M.O.), University of Ulm; Deutsches Zentrum für Neurodegenerative Erkrankungen (DZNE) (A.C.L.), Ulm, Germany; Cognitive Disorders Unit (F.M., M.B., A.G.), Department of Neurology, Hospital Universitario Donostia; Neuroscience Area (F.M., M.B., A.G.), Biogipuzkoa Health Research Institute, San Sebastian, Spain; Department of Neurology (M.O.), Martin Luther University, University Hospital, Halle (Saale), Germany; MRC Cognition and Brain Sciences Unit (J.B.R., A.G.M., T.R.), Department of Clinical Neurosciences, and Cambridge University Hospitals NHS Trust, University of Cambridge, Cambridge, United Kingdom; Department of Neurology and Alzheimer Center Erasmus MC (H.S., E.V.D.E., J.C.V.S.), Erasmus MC University Medical Center, Rotterdam, the Netherlands; Neurology (E. Solje, P.H.), Institute of Clinical Medicine, University of Eastern Finland; Neurocenter (E. Solje), Neurology, Kuopio University Hospital, Finland; Neurology Clinic (E. Stefanova, G.M.S.), Faculty of Medicine, University Clinical Center, University of Belgrade; UH Alexandrovska (L.D.T., S.M.), Department of Neurology, Medical University Sofia, Bulgaria; Theme Inflammation and Aging (V.J.), Medical Unit Aging Brain, Karolinska University Hospital Huddinge, Solna; Division of Clinical Geriatrics (V.J.), Department NVS, Karolinska Institutet, Huddinge, Sweden; Molecular Markers Laboratory (R.G.), IRCCS Istituto Centro San Giovanni di Dio Fatebenefratelli, Brescia; and Center for Neurodegenerative Diseases and the Aging Brain (M.T.D., C.Z., G.L.), Pia Fondazione Cardinale Giovanni Panico, University of Bari-Aldo Moro, Italy.
出版信息
Neurology. 2024 Oct 8;103(7):e209793. doi: 10.1212/WNL.0000000000209793. Epub 2024 Sep 3.
BACKGROUND AND OBJECTIVES
Data on care home admission and survival rates of patients with syndromes associated with frontotemporal lobar degeneration (FTLD) are limited. However, their estimation is essential to plan trials and assess the efficacy of intervention. Population-based registers provide unique samples for this estimate. The aim of this study was to assess care home admission rate, survival rate, and their predictors in incident patients with FTLD-associated syndromes from the European FRONTIERS register-based study.
METHODS
We conducted a prospective longitudinal multinational observational registry study, considering incident patients with FTLD-associated syndromes diagnosed between June 1, 2018, and May 31, 2019, and followed for up to 5 years till May 31, 2023. We enrolled patients fulfilling diagnosis of the behavioral variant frontotemporal dementia (bvFTD), primary progressive aphasia (PPA), progressive supranuclear palsy (PSP) or corticobasal syndrome (CBS), and FTD with motor neuron disease (FTD-MND). Kaplan-Meier analysis and Cox multivariable regression models were used to assess care home admission and survival rates. The survival probability score (SPS) was computed based on independent predictors of survivorship.
RESULTS
A total of 266 incident patients with FTLD were included (mean age ± SD = 66.7 ± 9.0; female = 41.4%). The median care home admission rate was 97 months (95% CIs 86-98) from disease onset and 57 months (95% CIs 56-58) from diagnosis. The median survival was 90 months (95% CIs 77-97) from disease onset and 49 months (95% CIs 44-58) from diagnosis. Survival from diagnosis was shorter in FTD-MND (hazard ratio [HR] 4.59, 95% CIs 2.49-8.76, < 0.001) and PSP/CBS (HR 1.56, 95% CIs 1.01-2.42, = 0.044) compared with bvFTD; no differences between PPA and bvFTD were found. The SPS proved high accuracy in predicting 1-year survival probability (area under the receiver operating characteristic curve = 0.789, 95% CIs 0.69-0.87), when defined by age, European area of residency, extrapyramidal symptoms, and MND at diagnosis.
DISCUSSION
In FTLD-associated syndromes, survival rates differ according to clinical features and geography. The SPS was able to predict prognosis at individual patient level with an accuracy of ∼80% and may help to improve patient stratification in clinical trials. Future confirmatory studies considering different populations are needed.
背景与目的
关于与额颞叶变性（FTLD）相关综合征患者入住养老院的比率和生存率的数据有限。然而，这些估计对于计划试验和评估干预措施的效果至关重要。基于人群的登记处为这种估计提供了独特的样本。本研究的目的是评估欧洲 FRONTIERS 基于登记处的研究中，与 FTLD 相关综合征的首发患者的入住养老院比率、生存率及其预测因素。
方法
我们进行了一项前瞻性、纵向、多国观察性登记研究，纳入 2018 年 6 月 1 日至 2019 年 5 月 31 日期间诊断的、与 FTLD 相关的综合征首发患者，并进行长达 5 年的随访，截至 2023 年 5 月 31 日。我们纳入了符合行为变异额颞叶痴呆（bvFTD）、原发性进行性失语症（PPA）、进行性核上性麻痹（PSP）或皮质基底节综合征（CBS）以及伴有运动神经元病的额颞叶痴呆（FTD-MND）诊断标准的患者。使用 Kaplan-Meier 分析和 Cox 多变量回归模型评估入住养老院率和生存率。根据生存预测因素计算生存概率评分（SPS）。
结果
共纳入 266 例 FTLD 首发患者（平均年龄 ± 标准差=66.7±9.0；女性=41.4%）。从疾病发病到入住养老院的中位比率为 97 个月（95%CI 86-98），从诊断到入住养老院的中位比率为 57 个月（95%CI 56-58）。从疾病发病到中位生存率为 90 个月（95%CI 77-97），从诊断到中位生存率为 49 个月（95%CI 44-58）。与 bvFTD 相比，FTD-MND（风险比[HR]4.59，95%CI 2.49-8.76，<0.001）和 PSP/CBS（HR 1.56，95%CI 1.01-2.42，=0.044）患者从诊断到生存率更短，而 PPA 与 bvFTD 之间则无差异。SPS 当根据年龄、居住的欧洲地区、锥体外系症状和 MND 在诊断时的存在来定义时，在预测 1 年生存率方面具有较高的准确性（受试者工作特征曲线下面积=0.789，95%CI 0.69-0.87）。
讨论
在与 FTLD 相关的综合征中，生存率因临床特征和地理位置而异。SPS 能够以约 80%的准确性预测个体患者的预后，可能有助于改善临床试验中的患者分层。需要进行考虑不同人群的未来确认性研究。
Zotero 插件