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转移性前列腺癌治疗的获益与危害方面患者及一般人群的偏好:一项离散选择实验

Patient and General Population Preferences Regarding the Benefits and Harms of Treatment for Metastatic Prostate Cancer: A Discrete Choice Experiment.

作者信息

Menges Dominik, Piatti Michela C, Omlin Aurelius, Cathomas Richard, Benamran Daniel, Fischer Stefanie, Iselin Christophe, Küng Marc, Lorch Anja, Prause Lukas, Rothermundt Christian, O'Meara Stern Alix, Zihler Deborah, Lippuner Max, Braun Julia, Cerny Thomas, Puhan Milo A

机构信息

Epidemiology, Biostatistics and Prevention Institute, University of Zurich, Zurich, Switzerland.

Department of Medical Oncology and Hematology, Kantonsspital St. Gallen, St. Gallen, Switzerland.

出版信息

Eur Urol Open Sci. 2023 Mar 22;51:26-38. doi: 10.1016/j.euros.2023.03.001. eCollection 2023 May.

Abstract

BACKGROUND

Patient preferences for treatment outcomes are important to guide decision-making in clinical practice, but little is known about the preferences of patients with metastatic hormone-sensitive prostate cancer (mHSPC).

OBJECTIVE

To evaluate patient preferences regarding the attributed benefits and harms of systemic treatments for mHSPC and preference heterogeneity between individuals and specific subgroups.

DESIGN SETTING AND PARTICIPANTS

We conducted an online discrete choice experiment (DCE) preference survey among 77 patients with metastatic prostate cancer (mPC) and 311 men from the general population in Switzerland between November 2021 and August 2022.

OUTCOME MEASUREMENTS AND STATISTICAL ANALYSIS

We evaluated preferences and preference heterogeneity related to survival benefits and treatment-related adverse effects using mixed multinomial logit models and estimated the maximum survival time participants were willing to trade to avert specific adverse effects. We further assessed characteristics associated with different preference patterns via subgroup and latent class analyses.

RESULTS AND LIMITATIONS

Patients with mPC showed an overall stronger preference for survival benefits in comparison to men from the general population ( = 0.004), with substantial preference heterogeneity between individuals within the two samples (both  < 0.001). There was no evidence of differences in preferences for men aged 45-65 yr versus ≥65 yr, patients with mPC in different disease stages or with different adverse effect experiences, or general population participants with and without experiences with cancer. Latent class analyses suggested the presence of two groups strongly preferring either survival or the absence of adverse effects, with no specific characteristic clearly associated with belonging to either group. Potential biases due to participant selection, cognitive burden, and hypothetical choice scenarios may limit the study results.

CONCLUSIONS

Given the relevant heterogeneity in participant preferences regarding the benefits and harms of treatment for mHSPC, patient preferences should be explicitly discussed during decision-making in clinical practice and reflected in clinical practice guidelines and regulatory assessment regarding treatment for mHSPC.

PATIENT SUMMARY

We examined the preferences (values and perceptions) of patients and men from the general population regarding the benefits and harms of treatment for metastatic prostate cancer. There were large differences between men in how they balanced the expected survival benefits and potential adverse effects. While some men strongly valued survival, others more strongly valued the absence of adverse effects. Therefore, it is important to discuss patient preferences in clinical practice.

摘要

背景

患者对治疗结果的偏好对于指导临床实践中的决策很重要,但对于转移性激素敏感性前列腺癌(mHSPC)患者的偏好知之甚少。

目的

评估患者对mHSPC全身治疗的归因益处和危害的偏好,以及个体和特定亚组之间的偏好异质性。

设计、设置和参与者:2021年11月至2022年8月期间,我们在瑞士对77例转移性前列腺癌(mPC)患者和311名普通男性进行了一项在线离散选择实验(DCE)偏好调查。

结果测量和统计分析

我们使用混合多项logit模型评估了与生存益处和治疗相关不良反应相关的偏好和偏好异质性,并估计了参与者为避免特定不良反应而愿意牺牲的最长生存时间。我们通过亚组分析和潜在类别分析进一步评估了与不同偏好模式相关的特征。

结果和局限性

与普通男性相比,mPC患者总体上对生存益处的偏好更强(P = 0.004),两个样本中的个体之间存在显著的偏好异质性(均P < 0.001)。没有证据表明45 - 65岁男性与≥65岁男性、不同疾病阶段或有不同不良反应经历的mPC患者,以及有和没有癌症经历的普通人群参与者在偏好上存在差异。潜在类别分析表明存在两组,一组强烈偏好生存,另一组强烈偏好无不良反应,没有明确的特定特征与属于哪一组相关。由于参与者选择、认知负担和假设选择场景导致的潜在偏差可能会限制研究结果。

结论

鉴于参与者对mHSPC治疗益处和危害的偏好存在相关异质性,在临床实践决策过程中应明确讨论患者偏好,并在mHSPC治疗的临床实践指南和监管评估中予以体现。

患者总结

我们研究了患者和普通男性对转移性前列腺癌治疗益处和危害的偏好(价值观和认知)。男性在平衡预期生存益处和潜在不良反应方面存在很大差异。一些男性非常重视生存,而另一些男性更重视无不良反应。因此,在临床实践中讨论患者偏好很重要。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/73ca/10175729/174e5017ddbe/gr1.jpg

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