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量化患者对难治性慢性自发性荨麻疹治疗方法的偏好。

Quantifying patient preferences for treatments for refractory chronic spontaneous urticaria.

作者信息

Babalola Olufemi, Hass Richard, McAna John, Segal Manav, Gonzalez Juan Marcos, Fadugba Olajumoke

机构信息

Jefferson College of Population Health, Thomas Jefferson University, Philadelphia, Pa.

Chestnut Hill Allergy and Asthma Associates, Wyndmoor, Pa.

出版信息

J Allergy Clin Immunol Glob. 2025 Mar 31;4(3):100468. doi: 10.1016/j.jacig.2025.100468. eCollection 2025 Aug.

Abstract

BACKGROUND

In recent years, it has become increasingly common to incorporate the patient perspective into drug development and regulatory decision making.

OBJECTIVE

This study aimed to measure and quantify patient preferences (priorities and trade-offs) for attributes that characterize current and emerging refractory chronic spontaneous urticaria (rCSU) treatments.

METHODS

Adult patients with self-reported rCSU symptoms completed an online discrete choice experiment survey. The survey included 10 questions that asked respondents to choose between 2 hypothetical rCSU treatment profiles having similar attributes with varying levels. The attributes included the following: chance of control of symptoms, time to symptom control, return of symptoms after discontinuation of therapy (complete remission), allergic reaction, risk of kidney dysfunction (usually reversible), and mode and frequency of administration. Relative attribute importance and maximum acceptable risks were calculated.

RESULTS

A total of 213 subjects with a mean age of 51 years completed the survey. Efficacy (symptom control) and mode of administration were the 2 most important attributes to treatment choice, followed by risk of kidney dysfunction and time to achieve symptom control. Complete remission of symptoms and risk of allergic reaction were identified as least important. With regard to mode of administration, topical treatment was the most preferred option and infusion therapy was least preferred. Respondents who were presented with a scenario of refractory and severe chronic spontaneous urticaria were willing to accept increased risk of reversible kidney dysfunction in exchange for improvement in symptom control or complete remission. Respondents were willing to accept infusion over topical treatment if there was significant increase in treatment efficacy.

CONCLUSION

These study results can be used to inform development and evaluation of future rCSU therapies by product developers and regulatory authorities, respectively.

摘要

背景

近年来,将患者视角纳入药物研发和监管决策已变得越来越普遍。

目的

本研究旨在衡量和量化患者对当前及新兴难治性慢性自发性荨麻疹(rCSU)治疗特征属性的偏好(优先级和权衡)。

方法

自我报告有rCSU症状的成年患者完成了一项在线离散选择实验调查。该调查包括10个问题,要求受访者在2种具有不同水平相似属性的假设性rCSU治疗方案之间进行选择。这些属性包括:症状控制的机会、症状控制时间、治疗中断后症状复发(完全缓解)、过敏反应、肾功能障碍风险(通常可逆)以及给药方式和频率。计算了相对属性重要性和最大可接受风险。

结果

共有213名平均年龄为51岁的受试者完成了调查。疗效(症状控制)和给药方式是治疗选择中最重要的2个属性,其次是肾功能障碍风险和实现症状控制的时间。症状完全缓解和过敏反应风险被确定为最不重要。关于给药方式,局部治疗是最优选的选项,而输液治疗是最不优选的。面对难治性和严重慢性自发性荨麻疹情况的受访者愿意接受可逆性肾功能障碍风险增加,以换取症状控制的改善或完全缓解。如果治疗疗效显著提高,受访者愿意接受输液而非局部治疗。

结论

这些研究结果可分别用于为产品开发者和监管机构提供有关未来rCSU疗法研发和评估的信息。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/d30b/12051122/dac19b01f572/gr1.jpg

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