International Psychogeriatric Association consensus statement on defining and measuring treatment benefits in dementia
International Psychogeriatric Association consensus statement on defining and measuring treatment benefits in dementia
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DOI：
10.1017/s1041610207005145
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发表时间：
2007-06-01
影响因子：
7
通讯作者：
Chiu, Edmond
中科院分区：
文献类型：
--
作者：
Katona, Cornelius;Livingston, Gill;Chiu, Edmond
目前对痴呆症的对症治疗只有适度的疗效。评估这种渐进性综合征的有意义的益处是复杂和困难的。这份共识声明是由一个代表护理人员、组织和具有痴呆症专业知识的专业人士的国际小组提出的。我们建议在治疗试验中声明明确、预先定义的诊断和严重程度标准以及结局指标，包括功能和执行能力。结果可以包括对痴呆症（PWD）患者在认知，行为和心理症状，生活质量，整体评估和日常生活活动方面的影响，并且必须根据参与者的教育和文化进行调整。结果也可以适当地包括对照顾者的影响。可能需要新的工具，因为许多现有的措施已经在相对同质的人群中得到验证。治疗目标可以是预防痴呆症的出现，或在那些已确诊的痴呆症患者中减缓恶化，并推迟，稳定或改善症状。治疗组之间的比较应基于临床相关指标，报告所有受试者的风险和获益，无论他们是否在整个试验期间继续接受治疗。迄今为止，对治疗反应良好的任何组的表征都不成功，但将来可能通过测量推定的生物标志物来促进。尽管最近取得了相当大的进展和几个“候选”生物标志物，但没有一个在确定诊断、严重程度、进展或预测反应方面令人满意。为了提供有意义的数据，经济分析应使用最新的、国家特定的数据。卫生经济学指标应作为次要结局纳入所有3期试验，因为卫生系统关注成本效益和临床结局。健康效用的措施，但是，验证令人满意的痴呆症，从而质疑以前的卫生经济分析。虽然目前的药物似乎减少了PWD所需的家庭照顾者时间，但这些费用主要落在老年人身上，他们通常很少发挥政治影响力，而不是整个社会。传统上，老年人在政治进程中被边缘化。世界各地老年人口的增长及其增加政治权力的潜力可能导致对痴呆症治疗经济学的彻底重新评估。
Current symptomatic treatments for dementia have only modest efficacy. Assessing meaningful benefits in this variably progressive syndrome is complex and difficult. This consensus statement was generated by an international group representing caregivers, organizations and professionals with expertise in dementia.We recommend the statement of clear, pre-defined diagnostic and severity criteria and outcome measures, which include functional and executive capacity, in treatment trials. Outcomes can include effects on people with dementia (PWD) with regard to cognition, behavioral and psychological symptoms, quality of life, global assessments, and activities of daily living, and must be tailored to the education and culture of the participants. Outcomes can also appropriately encompass effects on caregivers. New instruments may be needed, as validation of many existing measures has been in relatively homogenous populations. Treatment goals can be to prevent dementia emerging, or in those with established dementia to slow deterioration, and to postpone, stabilize or improve symptoms. Comparisons between treatment groups should be on the basis of clinically relevant measures with both risk and benefit reported for all participants regardless of whether or not they continue to receive treatment throughout the trial. Characterization of any groups that respond well to treatment has been unsuccessful to date, but may be facilitated in the future by measurement of putative biomarkers. Despite considerable recent progress and several 'candidate' biomarkers, none is yet satisfactory for determining diagnosis, severity, progression or prediction of response.To provide meaningful data, economic analyses should use up-to-date, country-specific data. Health economic measures should be incorporated as secondary outcomes in all Phase 3 trials since health systems are concerned with cost-effectiveness as well as clinical outcome. Health utility measures are not, however, validated satisfactorily in dementia, thus calling into question previous health economic analyses. While current drugs appear to reduce the amount of family caregiver time required by PWD, these costs fall in the main on older individuals who often exert little political leverage, rather than on society at large. Traditionally, elderly people have been marginalized in the political process. The growth in the older population across the world, and their potential for increasing political empowerment may lead to a radical re-evaluation of the economics of treatment in dementia.