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患者对慢性痛风研究核心结局指标的偏好是否支持综合反应标准的有效性?

Do patient preferences for core outcome domains for chronic gout studies support the validity of composite response criteria?

机构信息

Department of Medicine, University of Otago, Wellington, New Zealand.

出版信息

Arthritis Care Res (Hoboken). 2013 Aug;65(8):1259-64. doi: 10.1002/acr.21955.

DOI:10.1002/acr.21955
PMID:23335569
Abstract

OBJECTIVE

To determine patient-derived weights or prioritization for core outcome domains in chronic gout clinical studies.

METHODS

Three patient groups participated in a conjoint decision-making exercise using 1000Minds software, which asked participants to make repeated judgments regarding which of 2 hypothetical patients with gout represented the best response to treatment. Each scenario compared 2 patients on the basis of change in 2 of 5 core outcome domains at a time. Agreement of 80% of the group was required to answer each scenario. Re-voting was performed once after discussion in instances of disagreement.

RESULTS

The relative importance accorded to each outcome domain was different across the 3 groups of patients. There was some consistency that tophi was the least or second to least important outcome domain for every group and pain between attacks was ranked in the bottom third of priority for all groups. Gout attacks were ranked as the second or third most important domain in each group. However, the relative importance of serum urate (SUA) and activity limitations was quite different among the 3 groups, with 1 group ranking SUA as the most important outcome and 1 group ranking it as the second to least important outcome.

CONCLUSION

Despite some consistency in the relative value of some outcome domains for chronic gout studies, there is sufficient disagreement in the relative importance of other domains of outcome to challenge the validity of constructing a composite index of response that would be applicable to most gout patients.

摘要

目的

确定慢性痛风临床研究中患者衍生权重或核心结局领域的优先级。

方法

三组患者使用 1000Minds 软件参与了联合决策制定练习,该练习要求参与者对两名患有痛风的假设患者中哪一位对治疗的反应最佳进行反复判断。每次情景比较 2 名患者时,根据 5 个核心结局领域中的 2 个领域的变化。每个场景的回答需要小组 80%的成员达成一致。在出现分歧的情况下,讨论后会进行一次重新投票。

结果

不同患者组对每个结局领域的重视程度不同。有一些一致性,即对于每个组,痛风石都是最不重要或第二不重要的结局领域,发作间疼痛在所有组中都排在优先级的倒数第三位。痛风发作在每个组中均被列为第二或第三重要的领域。然而,三组之间血清尿酸(SUA)和活动受限的相对重要性差异很大,一组将 SUA 列为最重要的结局,一组将其列为第二至最不重要的结局。

结论

尽管慢性痛风研究中某些结局领域的相对价值存在一定的一致性,但其他结局领域的相对重要性存在足够的分歧,这对构建适用于大多数痛风患者的反应综合指数的有效性提出了挑战。

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