患有早期症状性阿尔茨海默病的专科记忆诊所患者对lecanemab的态度：欧洲一项多中心调查的结果

摘要(中文译文)

仑卡奈单抗在欧洲联盟（EU）的获批是在延迟之后获得的。这一延迟引发了众多利益相关方的担忧，但接受专科记忆门诊诊疗的早期症状性阿尔茨海默病患者的態度仍未得到充分评估。因此，我们评估了欧洲专科记忆门诊早期症状性阿尔茨海默病患者对仑卡奈单抗的态度。在这项于2024年10月14日至2025年2月18日开展的匿名、国际、多中心、横断面调查中，一份由专家制定的标准化问卷评估了对仑卡奈单抗治疗及欧盟获批的态度。在回答四个二分类问题之前，参与者收到了关于预期临床获益、淀粉样蛋白相关影像学异常（ARIA）以及与APOE ε4纯合子相关的ARIA风险增加的简要说明信息。该调查在欧洲阿尔茨海默病联盟（EADC）、德国记忆门诊网络（DNG）和奥地利记忆中心的专科记忆门诊内开展。由于调查为匿名且在当地分发，可获得的招募流程数据仅限于已完成的问卷。281例早期症状性阿尔茨海默病患者完成了调查。网络层面样本量为EADC n=202、DNG n=60、奥地利 n=19；EADC内各国具体样本量及应答率无法获得。对仑卡奈单抗个体治疗（81.9%，95%置信区间[CI] 76.8-86.2）和欧盟总体获批（91.8%，95% CI 87.9-94.7）的支持率均较高。在APOE ε4纯合子情境下，支持率仍然可观但有所降低（治疗：61.2%，95% CI 55.2-66.9；获批：76.5%，95% CI 71.1-81.3）。与获批相关的问题获得的赞成率高于与治疗相关的问题（84% vs. 72%；p<0.001）。在监管建议将该群体排除后，对APOE ε4纯合子获批的支持率下降（从87%降至73%；p=0.025）；该比较反映的是在2024年11月14日之前与之后完成匿名调查的独立受访者。网络层面的比较为描述性分析，对地理推断而言检验效能不足。该专科记忆门诊样本中的高支持率提示患者认为获得仑卡奈单抗具有价值。对获批的支持率高于对个体治疗的支持率，可能反映了对超越个人治疗选择的治疗可及性的支持，但不能排除其他解释，如社会期望偏倚、默认应答、误解或调查信息的影响。研究结果不应推广至专科记忆门诊患者之外，并应结合潜在的选择偏倚和应答偏倚、缺乏应答率数据以及简短、未经效度验证的二分类问卷等因素进行解读。

Abstract

Lecanemab approval in the European Union (EU) was granted after a delay. This delay resulted in concerns from many stakeholders, but attitudes of patients with early symptomatic Alzheimer's disease receiving specialist memory-clinic care remained insufficiently assessed. Therefore, we evaluated attitudes of specialist memory-clinic patients with early symptomatic Alzheimer's disease towards lecanemab in Europe. In this anonymous, international, multicentre, cross-sectional survey conducted from October 14, 2024 to February 18, 2025, a standardized, expert-developed questionnaire assessed attitudes towards lecanemab treatment and EU approval. Before answering four binary questions, participants received brief explanatory information on expected clinical benefit, amyloid-related imaging abnormalities (ARIA), and the increased ARIA risk associated with APOE ε4 homozygosity. The survey was conducted in specialist memory clinics within the European Alzheimer's Disease Consortium (EADC), the German memory clinic network (DNG), and Austrian memory centers. Available recruitment-flow data were limited to completed questionnaires because the survey was anonymous and distributed locally. 281 patients with early symptomatic Alzheimer's disease completed the survey. Network-level sample sizes were EADC n = 202, DNG n = 60, and Austria n = 19; country-specific sample sizes within the EADC and response rates were not available. Endorsement was high for both individual treatment with lecanemab (81.9%, 95% confidence interval [CI] 76.8-86.2) and general EU approval (91.8%, 95% CI 87.9-94.7). Endorsement remained substantial, but was lower, in the context of APOE ε4 homozygosity (treatment: 61.2%, 95% CI 55.2-66.9; approval: 76.5%, 95% CI 71.1-81.3). Approval-related questions received higher endorsement than treatment-related questions (84% vs. 72%; p < 0.001). Support for approval for APOE ε4 homozygotes declined after regulatory recommendations excluded this group (from 87% to 73%; p = 0.025); this comparison reflects independent respondents completing the anonymous survey before versus after November 14, 2024. Network-level comparisons were descriptive and underpowered for geographic inference. High endorsement within this specialist memory-clinic sample suggests perceived value of access to lecanemab. Greater endorsement for approval than for individual treatment may reflect support for treatment access beyond personal treatment choice, but alternative explanations such as social desirability, acquiescence, misunderstanding, or effects of the survey information cannot be excluded. The findings should not be generalized beyond specialist memory-clinic patients and should be interpreted in view of potential selection and response biases, absent response-rate data, and the brief, non-validated binary questionnaire.

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