患者在治疗减量试验中应当对追求或放弃特定疗法的决策发挥主动作用。本研究的目的是评估参与一个临床试验的患者的纵向患者报告结果 (PROs)，包括决策舒适度和与健康相关的生活质量 (HRQOL)，这个试验评估了在新辅助系统治疗 (NST) 对侵袭性乳腺癌具有极好疗效的情况下，仅采用放射疗法而不进行乳腺手术的临床试验的病人。本研究是一个前瞻性的单组分II临床试验，在7个美国医疗中心进行。于2017年3月6日至2021年11月9日期间，招募了年龄在40岁及以上的无病理证据显示标准NST后剩余疾病的侵袭性cT1-2 N0-1 M0三阴性或人类表皮生长因子受体2(ERBB2)阳性乳腺癌的女性。验证过的PRO测量在基线和放疗后的6、12和36个月进行。数据分析于2023年1月至2月进行。PRO测量包括决策遗憾量表 (DRS)、癌症治疗功能评估-淋巴水肿 (FACT-B+4)和乳腺癌治疗结果量表(BCTOS)。通过时间对PRO测量得分和子分量进行变化分析。在31名患者中，中位数(IQR)年龄为61(56-66)岁，26名(84%)是白人，26名(84%)是非西班牙裔。共有15例(48%)为三阴性疾病，16例(52%)为ERBB2阳性疾病。基线时的决策舒适度较高 (中位数[IQR] DRS评分为10 [0-25]，评分越高表示决策遗憾越高)，并且随时间显著增加 (36个月时中位数[IQR] DRS评分为0 [0-20]; P < .001)。HRQOL基线时相对较高 (中位数[IQR] FACT-B综合评分为121 [111-134]，评分越 high 表示HRQOL越高)，并且随时间显著增加 (36个月时中位数[IQR] FACT-B评分为128 [116-137]; P = .04)。基线时受影响乳房和对侧乳房之间的差异较小 (中位数[IQR] BCTOS评分为1.05 [1.00-1.23]，评分越高表示差异越大)，而随时间增加显著增加 (36个月时中位数[IQR] BCTOS评分为1.36 [1.18-1.64]; P < .001)。放疗后36个月的整体美容子分的评分比基线高0.45分 (95% CI，0.16-0.74; P = .001)，而功能、疼痛和水肿子分评分与基线没有显著差异。在这个非随机分组的phase 2临床试验中，PRO的分析显示了试验参与者的整体积极体验，并且在时间推移中决策舒适度和整体HRQOL得到了持续改善，治疗的副作用持续性不大。ClinicalTrials.gov识别号: NCT02945579.

Patients should have an active role in decisions about pursuing or forgoing specific therapies in treatment de-escalation trials.To evaluate longitudinal patient-reported outcomes (PROs) encompassing decisional comfort and health-related quality of life (HRQOL) among patients who elected to enroll in a clinical trial evaluating radiotherapy alone, without breast surgery, for invasive breast cancers with exceptional response to neoadjuvant systemic therapy (NST).Prospective, single-group, phase 2 clinical trial at 7 US medical centers. Women aged 40 years or older with invasive cT1-2 N0-1 M0 triple-negative or human epidermal growth factor receptor 2 (ERBB2)-positive breast cancer with no pathologic evidence of residual disease following standard NST enrolled from March 6, 2017, to November 9, 2021. Validated PRO measures were administered at baseline and 6, 12, and 36 months post-radiotherapy. Data were analyzed from January to February 2023.PRO measures included the Decision Regret Scale (DRS), Functional Assessment of Cancer Therapy-Lymphedema (FACT-B+4), and Breast Cancer Treatment Outcomes Scale (BCTOS).Changes in PRO measure scores and subscores over time.Among 31 patients, the median (IQR) age was 61 (56-66) years, 26 (84%) were White, and 26 (84%) were non-Hispanic. A total of 15 (48%) had triple-negative disease and 16 (52%) had ERBB2-positive disease. Decisional comfort was high at baseline (median [IQR] DRS score 10 [0-25] on a 0-100 scale, with higher scores indicating higher decisional regret) and significantly increased over time (median [IQR] DRS score at 36 months, 0 [0-20]; P < .001). HRQOL was relatively high at baseline (median [IQR] FACT-B composite score 121 [111-134] on a 0-148 scale, with higher scores indicating higher HRQOL) and significantly increased over time (median [IQR] FACT-B score at 36 months, 128 [116-137]; P = .04). Perceived differences between the affected breast and contralateral breast were minimal at baseline (median [IQR] BCTOS score 1.05 [1.00-1.23] on a 1-4 scale, with higher scores indicating greater differences) and increased significantly over time (median [IQR] BCTOS score at 36 months, 1.36 [1.18-1.64]; P < .001). At 36 months postradiotherapy, the cosmetic subscore was 0.45 points higher than baseline (95% CI, 0.16-0.74; P = .001), whereas function, pain, and edema subscores were not significantly different than baseline.In this nonrandomized phase 2 clinical trial, analysis of PROs demonstrated an overall positive experience for trial participants, with longitudinal improvements in decisional comfort and overall HRQOL over time and minimal lasting adverse effects of therapy.ClinicalTrials.gov Identifier: NCT02945579.