The Patient Perspectives on Future Therapeutic Options in NASH and Patient Needs
The Patient Perspectives on Future Therapeutic Options in NASH and Patient Needs
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DOI：
10.3389/fmed.2019.00061
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发表时间：
2019-04-03
影响因子：
3.9
通讯作者：
Balp, Maria-Magdalena
中科院分区：
文献类型：
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作者：
Cook, Nigel;Geier, Andreas;Balp, Maria-Magdalena
背景：非酒精性脂肪性肝炎（NASH）是一种具有严重并发症的慢性肝脏疾病，目前尚无批准的治疗方法。目前，关于该疾病对患者的总体负担或患者需求和偏好的数据有限。本研究调查了NASH患者对未来潜在治疗方法的偏好。此外，还探讨了与患者相关的因素及其对未来治疗方案的重要性。方法：电话深度访谈（TDIs），在线定量调查30分钟。在线调查包括(1)关于NASH诊断和疾病背景的选择题（mcq）。(2)确定患者对诊断时提供的信息的满意程度，并详细探讨症状学。(3)评估潜在新产品和产品属性的练习，包括“拖放”排序练习和基于自适应选择的联合练习（ACBC）。(4)采用EQ-5D-5L问卷和视觉模拟量表（VAS）来衡量患者的健康状况。(5)收集社会人口统计数据，(6)测量患者对调查满意度的问题。结果：共纳入166例患者，分别来自加拿大[n = 36]、德国[n = 50]、英国[n = 30]和美国[n = 50]。57%的患者[n = 94]进行了肝活检以确认NASH。患者通常无法将其症状与NASH或其他疾病联系起来。ACBC结果显示，疗效，定义为“对肝脏状态的影响”，是未来潜在NASH治疗的最重要属性。其他被认为具有次要重要性的属性包括对体重的影响、症状控制和副作用的存在。EQ-5D效用评分为0.81,VAS = 67.2。结论：“对肝脏状况的影响”是主要的预后指标。患者普遍缺乏对其疾病的了解，似乎不熟悉NASH的长期后果。有必要提高患者对NASH及其进展性的认识，并需要改进确诊诊断和监测。
Background: Non-alcoholic steatohepatitis (NASH) is a chronic liver disease with severe complications and without approved therapies. Currently, there is limited data on the overall burden of the disease for patients or on patient needs and preferences. This study investigates patient preferences in relation to potential future therapies for NASH. In addition, the factors that are relevant to patients and their importance in relation to future treatment options are explored.Method: Telephone in-depth interviews (TDIs) preceded an online 30-min quantitative survey. The online survey included (1) multiple choice questions (MCQs) on NASH diagnosis and disease background. (2) An exercise to determine patients' satisfaction levels with information provided at diagnosis, and to explore symptomatology in detail. (3) Exercises to evaluate potential new products and product attributes, including a "drag and drop" ranking exercise, and an adaptive choice-based conjoint exercise (ACBC). (4) The EQ-5D-5L questionnaire and the Visual Analog Scale (VAS), which measures patients' health status. (5) Collection of socio-demographic data, and (6) Questions to measure patient satisfaction with the survey.Results: There were 166 patients included in this study from Canada [n = 36], Germany [n = 50], the UK [n = 30], and USA [n = 50]. Fifty seven percent of patients [n = 94] had had a liver biopsy for confirmation of NASH. Patients were often unable to link their symptoms to NASH or other conditions. ACBC results showed that efficacy, defined as "impact on liver status" was the single most important attribute of a potential future NASH therapy. Other attributes considered to have secondary importance included impact on weight, symptom control and the presence of side effects. The EQ-5D utility score was 0.81 and VAS = 67.2.Conclusion: "Impact on liver status" is the primary outcome sought. Patients demonstrate a general lack of understanding of their disease and appeared to be unfamiliar with longer-term consequences of NASH. It is necessary to improve patient understanding of NASH and its progressive nature, and there is a need for improving confirmatory diagnosis and monitoring.