阿尔法-1 抗胰蛋白酶缺乏症患者对临床试验的态度

IF 3.5 3区 医学 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS
Kristen E. Holm , Robert A. Sandhaus , Sheri Allison , Charlie Strange
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引用次数: 0

摘要

目的 本研究探讨了影响α-1抗胰蛋白酶缺乏症(AATD)患者参与兴趣的临床试验特征。程序 AATD患者完成了一项横断面调查。34个项目描述了临床试验的特点,评分范围从1分(不会参加)到5分(非常有兴趣参加)。结果 1664 名参与者(91.6% 患有肺病,16.3% 患有肝病,14.9% 患有肺病和肝病)提供了数据。近三分之一(31.8%)的参与者表示,如果有机会获得安慰剂,他们不会参加试验。如果试验包括三次肝活检,53.3% 的人不会参加。超过三分之二(69.8%)正在使用增强疗法的参与者不会参加需要停药 12 个月的试验。上一年病情加重两次或两次以上的患者更有可能对试验产生浓厚的兴趣(OR = 1.4,95 % CI = 1.1-1.7,p = 0.009)。此外,慢性阻塞性肺病评估测试得分为 10 分或更高的人更有可能对试验产生浓厚兴趣(OR = 1.4,95 % CI = 1.1-1.8,p = 0.010)。结论相当大比例的参与者表示,他们不会参加包括安慰剂、涉及多次肝活检或涉及停止增强疗法的临床试验。受 AATD 影响较大的人比受影响较小的人更有兴趣参与试验。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Clinical trial attitudes among individuals with Alpha-1 antitrypsin deficiency

Purpose

This study examined characteristics of clinical trials that influence interest in participation among individuals with alpha-1 antitrypsin deficiency (AATD).

Procedures

A cross-sectional survey was completed by individuals with AATD. Thirty-four items described characteristics of clinical trials, which were rated from 1 (would not participate) to 5 (highly interested in participating). Logistic regression was used to compare participants with high interest in trials (defined as scores of 4 or 5 on ≥50 % of responses) to all remaining participants.

Results

Data were provided by 1664 participants (91.6 % with lung disease, 16.3 % with liver disease, 14.9 % with lung and liver disease). Nearly one-third (31.8 %) indicated that they would not participate in a trial if there was a chance of getting a placebo. If the trial included three liver biopsies, 53.3 % would not participate. More than two-thirds (69.8 %) of participants who were using augmentation therapy would not participate in a trial that required twelve months off therapy. Individuals with two or more exacerbations in the prior year were more likely to have high interest in trials (OR = 1.4, 95 % CI = 1.1–1.7, p = 0.009). In addition, individuals with a score of 10 or higher on the COPD Assessment Test were more likely to have high interest (OR = 1.4, 95 % CI = 1.1–1.8, p = 0.010).

Conclusions

A sizeable percentage of participants indicated that they would not participate in clinical trials that include a placebo, involve multiple liver biopsies, or involve discontinuing augmentation therapy. Individuals who are more affected by AATD have more interest in trial participation than individuals who are less affected.

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来源期刊
Respiratory medicine
Respiratory medicine 医学-呼吸系统
CiteScore
7.50
自引率
0.00%
发文量
199
审稿时长
38 days
期刊介绍: Respiratory Medicine is an internationally-renowned journal devoted to the rapid publication of clinically-relevant respiratory medicine research. It combines cutting-edge original research with state-of-the-art reviews dealing with all aspects of respiratory diseases and therapeutic interventions. Topics include adult and paediatric medicine, epidemiology, immunology and cell biology, physiology, occupational disorders, and the role of allergens and pollutants. Respiratory Medicine is increasingly the journal of choice for publication of phased trial work, commenting on effectiveness, dosage and methods of action.
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