PP125 为什么说了解人口负担至关重要?

IF 2.6 4区 医学 Q2 HEALTH CARE SCIENCES & SERVICES International Journal of Technology Assessment in Health Care Pub Date : 2023-12-14 DOI:10.1017/s0266462323002386
Franziska J. Schöb, Julie Hviid Hahn-Pedersen
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引用次数: 0

摘要

尽管在过去的二十年里,人们在开发新的治疗方法方面做出了巨大的努力,但对症治疗仍然是绝大多数阿尔茨海默病(AD)痴呆患者的唯一选择。对于减缓或阻止AD进展的疾病修饰疗法(dmt)的需求仍未得到满足。正在开发的dmt针对阿尔茨海默病的早期阶段(临床前、轻度认知障碍和轻度痴呆阶段),从而为患者、临床医生和付款人创造了一种全新的治疗模式。一个关键的挑战将是在异质性很大的人群中确定合适的治疗患者。我们进行了一项文献综述,以更好地了解和定义AD人群,以期在未来实现更有针对性的治疗。方法检索sembase、MEDLINE和Cochrane图书馆,以确定2010-2021年间发表的关于患病率和亚组鉴定证据的观察性研究,包括鉴定的临床可行性。搜索只限于英语语言。结果共纳入45项研究,主要来自欧洲、美国和亚洲。人群主要根据一般人口统计学因素(如年龄、性别、性别)、AD分期、合并症或生物标志物进行分组。6个亚群的患病率数据:痴呆前期、轻度痴呆、年龄、载脂蛋白E (APOE)基因型、合并症肥胖和高血压。在这些研究中,根据研究设计和原产国的不同,患病率数据存在差异,轻度AD痴呆患者的患病率在6600万至1.02亿之间,或者作为另一个例子,APOE基因型携带者的患病率在4600万至9200万之间。结论AD人群患病率的异质性和不确定性给临床医生和支付者带来了巨大的挑战。未来关于新疗法的目标患者识别的讨论应该与当前的临床实践保持一致和整合,例如利用经过验证的生物标志物作为诊断工具。需要进一步研究一种综合方法,以确定哪些患者将从dmt中获益最多。
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PP125 Why Understanding The Burden Of The Population Is Fundamental
Introduction

Despite intense efforts in development of new treatments over the last two decades, symptomatic treatments remain the only option for the vast majority patients diagnosed with dementia due to Alzheimer’s disease (AD). There remains a significant unmet need for disease modifying therapies (DMTs) to slow or stop AD progression. DMTs in development are targeting early stages of AD (pre-clinical, mild cognitive impairment and mild dementia stages), thereby creating an entirely new treatment paradigm for patients, clinicians, and payers. A key challenge will be in identifying the appropriate patient for treatment in a very heterogenous population. We have performed a literature review to better understand and define the AD population, with a view to enabling more targeted treatment in future.

Methods

Embase, MEDLINE and the Cochrane Library were searched to identify publications between 2010-2021 on observational studies reporting evidence on prevalence and subgroup identification, including clinical feasibility of identification. The search was restricted to English language.

Results

We identified 45 studies, mostly from Europe, USA and Asia. Populations were primarily grouped based on generic demographic factors (e.g., age, sex, gender), AD staging, comorbidities or biomarkers. Prevalence data was available for six subpopulations: pre-dementia stage, mild dementia, age, Apolipoprotein E (APOE) genotype, comorbid obesity and hypertension. Across these, data on prevalence were heterogenous depending on study design and country of origin, and ranging between 66 million to 102 million for people with mild AD dementia, or as another example, ranging between 46 million to 92 million for APOE genotype carriers worldwide.

Conclusions

The heterogeneity and the uncertainty in prevalence of the AD population represent big challenges to clinicians and payers. Future discussions on target patient identification for new treatments should be aligned and integrated with current clinical practice e.g. leveraging validated biomarkers as diagnostic tools. Additional research on an integrated approach to identify patients who would benefit the most from DMTs will be needed.

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来源期刊
International Journal of Technology Assessment in Health Care
International Journal of Technology Assessment in Health Care 医学-公共卫生、环境卫生与职业卫生
CiteScore
4.40
自引率
15.60%
发文量
116
审稿时长
6-12 weeks
期刊介绍: International Journal of Technology Assessment in Health Care serves as a forum for the wide range of health policy makers and professionals interested in the economic, social, ethical, medical and public health implications of health technology. It covers the development, evaluation, diffusion and use of health technology, as well as its impact on the organization and management of health care systems and public health. In addition to general essays and research reports, regular columns on technology assessment reports and thematic sections are published.
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