Patient preferences for stages II–IV melanoma treatments in the UK: results from a cross-sectional study

To understand patient preferences for treatment attributes in adjuvant and metastatic melanoma in the UK. Patients with stages II–IV melanoma completed an online survey from November 2022 to February 2023 comprising two discrete choice experiments (DCE) exploring efficacy, safety, and dosing regimen. Out of 104 respondents, 100 and 102 patients completed DCEs for adjuvant (Adj-DCE) and metastatic (Met-DCE) settings, respectively. 4-year relapse-free survival (RFS) for Adj-DCE (33%), and 4-year overall survival (OS) for Met-DCE (40%) had the highest relative attribute importance (RI), followed by risk of grade ≥3 adverse events (29% versus 26%, respectively). Patients with advanced melanoma or BRAF mutation had significantly higher RI for RFS and OS. Patients, especially those with advanced melanoma or BRAF mutation, perceive efficacy as the most important factor in determining choice of treatment.
ARTICLE HIGHLIGHTSIn the UK, melanoma is the 5^th most common cancer with around 19,921 newly diagnosed cases in 2022. Fortunately, treatment options for patients with melanoma have expanded significantly over the past few years. Each of these new therapies carries a different benefit-to-risk profile and varies with regard to different aspects of patient convenience.The aim of this study was to understand patient preferences for adjuvant and metastatic treatment attributes in stage II/III and stage IV melanoma in the UK and to determine predictive factors for treatment preferences in different subpopulations.A discrete-choice experiment (DCE) was conducted via an online quantitative treatment preference survey to elicit patients’ relative preferences for treatment attributes, including safety, dosing regimen, and efficacy for adjuvant and metastatic settings, respectively.Analysis of the DCE data using Hierarchical Bayesian (HB) model revealed distinct preferences among the 104 survey respondents.Efficacy, as assessed by 4-year RFS in the adjuvant setting and 4-year OS in the metastatic setting, was the most important attribute, followed by severe adverse events (AEs) and dosing regimen.Immune checkpoint inhibitors (ICI) were consistently ranked as the preferred options across all patient segments. Based on the hypothetical drug profile presented in the DCE, the fixed dose combination of nivolumab and relatlimab was the preferred regimen in the metastatic disease setting while either pembrolizumab or nivolumab were preferred in the adjuvant setting.Our study highlights the importance of considering patients’ prioritization of treatment efficacy as the primary factor when making decisions about melanoma care, both in the adjuvant and metastatic settings. Although efficacy and safety were attributed to have relatively similar importance for treatment preference in the adjuvant setting, patients with more advanced disease or those carrying the BRAF mutation placed heightened emphasis on treatment efficacy. In the UK, melanoma is the 5^th most common cancer with around 19,921 newly diagnosed cases in 2022. Fortunately, treatment options for patients with melanoma have expanded significantly over the past few years. Each of these new therapies carries a different benefit-to-risk profile and varies with regard to different aspects of patient convenience. The aim of this study was to understand patient preferences for adjuvant and metastatic treatment attributes in stage II/III and stage IV melanoma in the UK and to determine predictive factors for treatment preferences in different subpopulations. A discrete-choice experiment (DCE) was conducted via an online quantitative treatment preference survey to elicit patients’ relative preferences for treatment attributes, including safety, dosing regimen, and efficacy for adjuvant and metastatic settings, respectively. Analysis of the DCE data using Hierarchical Bayesian (HB) model revealed distinct preferences among the 104 survey respondents. Efficacy, as assessed by 4-year RFS in the adjuvant setting and 4-year OS in the metastatic setting, was the most important attribute, followed by severe adverse events (AEs) and dosing regimen. Immune checkpoint inhibitors (ICI) were consistently ranked as the preferred options across all patient segments. Based on the hypothetical drug profile presented in the DCE, the fixed dose combination of nivolumab and relatlimab was the preferred regimen in the metastatic disease setting while either pembrolizumab or nivolumab were preferred in the adjuvant setting. Our study highlights the importance of considering patients’ prioritization of treatment efficacy as the primary factor when making decisions about melanoma care, both in the adjuvant and metastatic settings. Although efficacy and safety were attributed to have relatively similar importance for treatment preference in the adjuvant setting, patients with more advanced disease or those carrying the BRAF mutation placed heightened emphasis on treatment efficacy.

本研究旨在了解英国辅助治疗与转移性黑色素瘤患者对治疗相关属性的偏好。2022年11月至2023年2月期间，Ⅱ~Ⅳ期黑色素瘤患者完成了一项在线调查，该调查包含两项离散选择实验（Discrete Choice Experiment, DCE），分别探索疗效、安全性与给药方案。104名受访者中，分别有100名和102名患者完成了辅助治疗（Adj-DCE）与转移性疾病（Met-DCE）场景下的DCE。辅助治疗场景下的4年无复发生存期（relapse-free survival, RFS）（33%）与转移性疾病场景下的4年总生存期（overall survival, OS）（40%）具有最高的相对属性重要性（relative attribute importance, RI），其次为3级及以上不良事件发生风险（分别为29%与26%）。伴有晚期黑色素瘤或BRAF突变的患者，其RFS与OS的相对属性重要性显著更高。此类患者，尤其是晚期黑色素瘤或BRAF突变人群，均将疗效视为决定治疗选择的首要因素。

研究亮点：
在英国，黑色素瘤是第5大常见癌症，2022年新增确诊病例约19921例。幸运的是，过去数年间黑色素瘤患者的治疗选择已显著拓展。各类新型疗法的获益-风险特征各不相同，且在患者便利性的多个维度存在差异。
本研究旨在了解英国Ⅱ/Ⅲ期及Ⅳ期黑色素瘤患者对辅助治疗与转移性疾病治疗属性的偏好，并明确不同亚群患者治疗偏好的预测因素。
本研究通过在线量化治疗偏好调查开展离散选择实验（Discrete Choice Experiment, DCE），分别获取患者对辅助治疗与转移性疾病场景下的疗效、安全性及给药方案等治疗属性的相对偏好。
采用分层贝叶斯（Hierarchical Bayesian, HB）模型对DCE数据进行分析，结果显示104名受访者的治疗偏好存在显著差异。
疗效——以辅助治疗场景下的4年无复发生存期（RFS）与转移性疾病场景下的4年总生存期（OS）进行评估——为最关键的治疗属性，其次为严重不良事件（adverse events, AEs）与给药方案。
免疫检查点抑制剂（Immune Checkpoint Inhibitors, ICI）在所有患者亚群中均被列为首选治疗方案。
基于DCE中呈现的假想药物特征，纳武利尤单抗（nivolumab）与雷利单抗（relatlimab）的固定剂量联合疗法为转移性疾病场景下的首选给药方案，而帕博利珠单抗（pembrolizumab）或纳武利尤单抗（nivolumab）为辅助治疗场景下的首选方案。
本研究强调，在制定黑色素瘤诊疗决策时，无论辅助治疗还是转移性疾病场景，均需将患者对治疗疗效的优先考量作为核心因素。尽管在辅助治疗场景中，疗效与安全性对治疗偏好的相对重要性较为接近，但伴有更晚期疾病或携带BRAF突变的患者，会更加强调治疗疗效的重要性。

在英国，黑色素瘤是第5大常见癌症，2022年新增确诊病例约19921例。幸运的是，过去数年间黑色素瘤患者的治疗选择已显著拓展。各类新型疗法的获益-风险特征各不相同，且在患者便利性的多个维度存在差异。
本研究旨在了解英国Ⅱ/Ⅲ期及Ⅳ期黑色素瘤患者对辅助治疗与转移性疾病治疗属性的偏好，并明确不同亚群患者治疗偏好的预测因素。
通过在线量化治疗偏好调查开展离散选择实验（DCE），分别获取患者对辅助治疗与转移性疾病场景下的疗效、安全性及给药方案等治疗属性的相对偏好。
采用分层贝叶斯（HB）模型对DCE数据进行分析，结果显示104名受访者的治疗偏好存在显著差异。
疗效——以辅助治疗场景下的4年RFS与转移性疾病场景下的4年OS进行评估——为最关键的治疗属性，其次为严重不良事件（AEs）与给药方案。
免疫检查点抑制剂（ICI）在所有患者亚群中均被列为首选治疗方案。
基于DCE中呈现的假想药物特征，纳武利尤单抗与雷利单抗的固定剂量联合疗法为转移性疾病场景下的首选给药方案，而帕博利珠单抗或纳武利尤单抗为辅助治疗场景下的首选方案。
本研究强调，在制定黑色素瘤诊疗决策时，无论辅助治疗还是转移性疾病场景，均需将患者对治疗疗效的优先考量作为核心因素。尽管在辅助治疗场景中，疗效与安全性对治疗偏好的相对重要性较为接近，但伴有更晚期疾病或携带BRAF突变的患者，会更加强调治疗疗效的重要性。