daCosta DiBonaventura Marco, Copher Ronda, Basurto Enrique, Faria Claudio, Lorenzo Rose
Vice President, Health Outcomes, Health Outcomes Practice, Kantar Health, New York, NY.
Associate Director, Health Economics and Outcomes Research, Eisai Inc, Woodcliff Lake, NJ.
Am Health Drug Benefits. 2014 Oct;7(7):386-96.
Given the various profiles (eg, oral vs intravenous administration, risk of hot flashes vs fatigue) of treatment options (eg, endocrine therapy, chemotherapy) for metastatic breast cancer (mBC), how patients value these attributes of their medications has implications on making treatment decisions and on adherence.
To understand how patients trade off medication side effects with improved effectiveness and/or quality of life, to provide estimates of nonadherence among women with mBC, and to quantify the association of medication nonadherence with health outcomes.
The study was a cross-sectional, Internet-based survey of 181 women diagnosed with mBC who were recruited from cancer-specific online panels (response rate, 7%). Treatment information, demographics, nonadherent behaviors, and quality of life assessed by the Functional Assessment of Cancer Therapy-Breast (FACT-B) were collected in the survey, and each respondent completed a choice-based conjoint exercise to assess patient preferences. The patients' preferences were analyzed using hierarchical Bayesian logistic regression models, and the association between the number of nonadherent behaviors and the health outcomes was analyzed using general linear models.
The mean age of the patient sample was 52.2 years (standard deviation, ±9.1), with 93.9% of participants being non-Hispanic white. Results from the conjoint model indicated that effectiveness (overall survival) was of primary importance to patients, followed by side effects-notably alopecia, fatigue, neutropenia, motor neuropathy, and nausea/vomiting-and finally, dosing regimen. In all, 34.8% of survey respondents either discontinued their treatment or were nonadherent to their treatment regimen. Among those who have ever used oral chemotherapy (N = 95; 52.5%) and those currently using oral chemotherapy (N = 44; 24.3%), the number of nonadherent behaviors was significantly associated with a decrease in functional well-being (b [unstandardized regression coefficient] = -2.01 for patients who had ever used a targeted therapy and b = -3.14 for current users of a targeted therapy), FACT-General total score (b = -4.30 and b = -7.37, respectively), FACT-B total score (b = -3.93 and b = -6.11, respectively), and FACT trial outcome index (b = -5.22 and b = -8.63, respectively; all P <.05).
Patients were willing to accept substantial additional risks from side effects for gains in overall survival. Approximately 33% of women with mBC reported engaging in nonadherent behaviors. Because forgetfulness and adverse events were among the most frequent reasons for nonadherence, these results suggest that less complex treatment regimens, as well as regimens with less toxic profiles, may be associated with improvements in adherence and, subsequently, could correspond to perceptible patient benefits.
鉴于转移性乳腺癌（mBC）治疗方案（如内分泌治疗、化疗）具有多种特征（如口服与静脉给药、潮热风险与疲劳风险），患者如何权衡这些药物属性对治疗决策及依从性具有重要意义。
了解患者如何在药物副作用与疗效改善和/或生活质量提高之间进行权衡，估计mBC女性患者的不依从情况，并量化药物不依从与健康结局之间的关联。
本研究是一项基于互联网的横断面调查，对181名确诊为mBC的女性进行了调查，这些女性是从特定癌症在线小组招募的（应答率为7%）。调查收集了治疗信息、人口统计学资料、不依从行为以及通过癌症治疗功能评估-乳腺癌（FACT-B）评估的生活质量，每位受访者完成了一项基于选择的联合分析以评估患者偏好。使用分层贝叶斯逻辑回归模型分析患者偏好，并使用一般线性模型分析不依从行为数量与健康结局之间的关联。
患者样本的平均年龄为52.2岁（标准差±9.1），93.9%的参与者为非西班牙裔白人。联合模型结果表明，疗效（总生存期）对患者最为重要，其次是副作用——尤其是脱发、疲劳、中性粒细胞减少、运动神经病变以及恶心/呕吐——最后是给药方案。总体而言，34.8%的调查受访者停止了治疗或未遵守治疗方案。在曾经使用过口服化疗的患者（N = 95；52.5%）和目前正在使用口服化疗的患者（N = 44；24.3%）中，不依从行为的数量与功能健康状况下降显著相关（对于曾经使用过靶向治疗的患者，b[未标准化回归系数]= -2.01，对于目前使用靶向治疗的患者，b = -3.14），FACT-一般总分（分别为b = -4.30和b = -7.37），FACT-B总分（分别为b = -3.93和b = -6.11），以及FACT试验结局指数（分别为b = -5.22和b = -8.63；均P <.05）。
患者愿意为了总生存期的获益接受副作用带来的大量额外风险。约33%的mBC女性报告有不依从行为。由于遗忘和不良事件是不依从最常见的原因，这些结果表明，更简单的治疗方案以及毒性较小的方案可能与依从性改善相关，进而可能给患者带来明显益处。
