Department of Medicine (Hematology/Oncology), University of California San Francisco Comprehensive Cancer Center, San Francisco, USA.
Department of Medical Oncology, Institut Curie, Paris, France.
Ann Oncol. 2018 Apr 1;29(4):888-894. doi: 10.1093/annonc/mdy012.
Patient-reported outcomes are integral in benefit-risk assessments of new treatment regimens. The PALOMA-2 study provides the largest body of evidence for patient-reported health-related quality of life (QOL) for patients with metastatic breast cancer (MBC) receiving first-line endocrine-based therapy (palbociclib plus letrozole and letrozole alone).
Treatment-naïve postmenopausal women with estrogen receptor-positive (ER+)/human epidermal growth factor receptor 2-negative (HER2-) MBC were randomized 2 : 1 to palbociclib plus letrozole (n = 444) or placebo plus letrozole (n = 222). Patient-reported outcomes were assessed at baseline, day 1 of cycles 2 and 3, and day 1 of every other cycle from cycle 5 using the Functional Assessment of Cancer Therapy (FACT)-Breast and EuroQOL 5 dimensions (EQ-5D) questionnaires.
As of 26 February 2016, the median duration of follow-up was 23 months. Baseline scores were comparable between the two treatment arms. No significant between-arm differences were observed in change from baseline in FACT-Breast Total, FACT-General Total, or EQ-5D scores. Significantly greater improvement in pain scores was observed in the palbociclib plus letrozole arm (-0.256 versus -0.098; P = 0.0183). In both arms, deterioration of FACT-Breast Total score was significantly delayed in patients without progression versus those with progression and patients with partial or complete response versus those without. No significant difference was observed in FACT-Breast and EQ-5D index scores in patients with and without neutropenia.
Overall, women with MBC receiving first-line endocrine therapy have a good QOL. The addition of palbociclib to letrozole maintains health-related QOL and improves pain scores in treatment-naïve postmenopausal patients with ER+/HER2- MBC compared with letrozole alone. Significantly greater delay in deterioration of health-related QOL was observed in patients without progression versus those who progressed and in patients with an objective response versus non-responders. ClinicalTrials.gov: NCT01740427 (https://clinicaltrials.gov/ct2/show/NCT01740427).
患者报告的结局是新治疗方案获益-风险评估的重要组成部分。PALOMA-2 研究为转移性乳腺癌（MBC）患者接受一线内分泌治疗（哌柏西利联合来曲唑和来曲唑单药）的患者报告健康相关生活质量（HRQOL）提供了最大的证据。
未经治疗的绝经后妇女，雌激素受体阳性（ER+）/人表皮生长因子受体 2 阴性（HER2-）MBC 患者以 2:1 的比例随机分为哌柏西利联合来曲唑（n=444）或安慰剂联合来曲唑（n=222）。使用功能性评估癌症治疗（FACT）-乳房和欧洲五维健康量表（EQ-5D）问卷在基线、第 2 和第 3 个周期的第 1 天以及第 5 个周期后的每个周期的第 1 天评估患者报告的结局。
截至 2016 年 2 月 26 日，中位随访时间为 23 个月。两个治疗组的基线评分相当。从基线到 FACT-乳房总分、FACT-一般总分或 EQ-5D 评分的变化，两组间均无显著差异。哌柏西利联合来曲唑组疼痛评分的改善明显更大（-0.256 与-0.098；P=0.0183）。在两个治疗组中，与进展患者和部分或完全缓解患者相比，无进展患者的 FACT-乳房总分恶化显著延迟。中性粒细胞减少症患者和无中性粒细胞减少症患者的 FACT-乳房和 EQ-5D 指数评分无显著差异。
总体而言，接受一线内分泌治疗的 MBC 女性具有良好的 QOL。与来曲唑单药治疗相比，哌柏西利联合来曲唑可维持 HRQOL，并改善未经治疗的绝经后 ER+/HER2-MBC 患者的疼痛评分。与进展患者和无反应患者相比，无进展患者的健康相关 QOL 恶化显著延迟，客观缓解患者也显著延迟。ClinicalTrials.gov：NCT01740427（https://clinicaltrials.gov/ct2/show/NCT01740427）。
