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卡介苗无反应性非肌层浸润性膀胱癌患者的治疗偏好:一项跨国选择实验

Patient Preferences for Treatment of Bacillus Calmette-Guérin-unresponsive Non-muscle-invasive Bladder Cancer: A Cross-country Choice Experiment.

作者信息

Collacott Hannah, Krucien Nicolas, Heidenreich Sebastian, Catto James W F, Ghatnekar Ola

机构信息

Evidera, London, UK.

Department of Oncology, University of Sheffield, Sheffield, UK.

出版信息

Eur Urol Open Sci. 2023 Jan 31;49:92-99. doi: 10.1016/j.euros.2022.12.016. eCollection 2023 Mar.

Abstract

BACKGROUND

Patients with non-muscle-invasive bladder cancer (NMIBC) that is unresponsive to bacillus Calmette-Guérin (BCG) immunotherapy face a difficult choice. Immediate radical cystectomy (RC) is effective but might represent overtreatment. Continuing bladder preservation with medical therapy is an alternative, but it risks progression to muscle-invasive bladder cancer (MIBC) and a reduction in survival.

OBJECTIVE

To understand the trade-offs patients are willing to make in selecting treatments for BCG-unresponsive NMIBC.

DESIGN SETTING AND PARTICIPANTS

Adults with NMIBC from the UK, France, Germany, and Canada who reported current receipt of BCG, disease unresponsive to BCG, or receipt of RC in the previous 12 mo after failure of BCG were recruited to participate in an online choice experiment. Patients were asked to make repeated choices between two hypothetical medical treatments and the option to undergo immediate RC. The medical treatments required trade-offs between the time to RC, the mode and frequency of administration, the risk of experiencing serious side effects, and the risk of disease progression.

OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS

Error component logit models were used to calculate relative attribute importance (RAI) scores as the maximum percentage contribution to a preference and acceptable benefit-risk trade-offs.

RESULTS AND LIMITATIONS

Most of the 107 participants (average age 63 yr) never selected RC (89%) as their preferred option in the choice experiment. Preferences were most affected by time to RC (RAI 55%), followed by risk of progressing to MIBC (RAI 25%), medication administration (RAI 12%), and the risk of serious side effects (RAI 8%). To increase the time to RC from 1 yr to 6 yr, patients accepted a 43.8% increase in the risk of progression and a 66.1% increase in the risk of serious side effects.

CONCLUSIONS

Patients with BCG-treated NMIBC valued bladder-sparing treatments and were willing to make substantial benefit-risk trade-offs to delay RC.

PATIENT SUMMARY

Adults with bladder cancer not invading the bladder muscle completed an online experiment in which they chose between hypothetical medications and bladder removal. The results show that patients would be willing to accept different risks associated with medications to delay bladder removal. Patients considered disease progression the most important risk of medicinal treatment.

摘要

背景

对卡介苗(BCG)免疫疗法无反应的非肌层浸润性膀胱癌(NMIBC)患者面临艰难抉择。立即进行根治性膀胱切除术(RC)有效,但可能属于过度治疗。采用药物治疗继续保留膀胱是一种替代方案,但存在进展为肌层浸润性膀胱癌(MIBC)并降低生存率的风险。

目的

了解患者在为对BCG无反应的NMIBC选择治疗方案时愿意做出的权衡。

设计、设置和参与者:招募来自英国、法国、德国和加拿大的患有NMIBC的成年人,这些人报告目前正在接受BCG治疗、对BCG无反应或在BCG治疗失败后的前12个月内接受了RC治疗,参与一项在线选择实验。要求患者在两种假设的医学治疗方法和立即接受RC治疗的选项之间反复做出选择。医学治疗方法需要在进行RC的时间、给药方式和频率、出现严重副作用的风险以及疾病进展的风险之间进行权衡。

结果测量和统计分析

使用误差成分逻辑模型计算相对属性重要性(RAI)分数,作为对偏好和可接受的获益-风险权衡的最大百分比贡献。

结果与局限性

在选择实验中,107名参与者(平均年龄63岁)中的大多数(89%)从未选择RC作为他们的首选方案。偏好受进行RC的时间影响最大(RAI为55%),其次是进展为MIBC的风险(RAI为25%)、药物给药(RAI为12%)和严重副作用的风险(RAI为8%)。为了将进行RC的时间从1年延长至6年,患者接受了疾病进展风险增加43.8%和严重副作用风险增加66.1%。

结论

接受BCG治疗的NMIBC患者重视保留膀胱的治疗方法,并愿意做出重大的获益-风险权衡以推迟RC。

患者总结

患有未侵犯膀胱肌肉的膀胱癌的成年人完成了一项在线实验,在实验中他们在假设的药物治疗和膀胱切除之间进行选择。结果表明,患者愿意接受与药物治疗相关的不同风险以推迟膀胱切除。患者认为疾病进展是药物治疗最重要的风险。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/85c8/9974996/42ca48be5a1d/gr1.jpg

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