Real-World Effectiveness of Palbociclib Versus Clinical Trial Results in Patients With Advanced/Metastatic Breast Cancer That Progressed on Previous Endocrine Therapy
Real-World Effectiveness of Palbociclib Versus Clinical Trial Results in Patients With Advanced/Metastatic Breast Cancer That Progressed on Previous Endocrine Therapy
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DOI：
10.1177/1178223418823238
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发表时间：
2019-01-10
影响因子：
2.9
通讯作者：
van de Garde, Ewoudt M. W.
中科院分区：
文献类型：
--
作者：
Bui, Tam Binh V.;Burgers, Desiree M. T.;van de Garde, Ewoudt M. W.
目的：本研究的目的是评估帕博西利联合内分泌治疗激素受体阳性(HR阳性)、人表皮生长因子受体2阴性(HER2阴性)、晚期/转移性乳腺癌的真实疗效和耐受性，并将这些结果与Paloma-3临床试验的结果进行比较。方法：本研究是一项回顾性观察队列研究，包括圣安东尼奥医院2016年9月1日至2018年4月1日期间开始使用帕博西利治疗HR阳性的所有患者，HER2阴性的晚期/转移性乳腺癌，在以前的内分泌治疗中进展。个别患者的数据是从电子病历中收集的。主要研究结果是无进展生存期(PFS)和由于不良事件(AEs)导致疾病进展前永久停止治疗的数量。次要结果是所有(严重)不良反应的发生频率、剂量减少的频率和原因、中断和周期延迟。结果：共有46例患者进行了中位数13.0个月的随访。总体而言，现实世界临床实践中的中位PFS为10.0个月(95%可信区间为4.9-15.1)，而Paloma-3的中位PFS为9.5个月(95%可信区间为9.2-11.0)。2例因不良反应停止治疗。中性粒细胞减少是最常见的3-4级AE，但没有发热的中性粒细胞减少事件。大多数AEs采用改良的帕波西利剂量治疗。在这些改进方面，与Paloma-3相比，临床实践中出现了更多的周期延迟、更少的剂量减少和更少的剂量中断(分别为59vs36%、22vs34%和9vs54%)。不符合Paloma-3研究资格标准的患者(n=16)的中位PFS较低，为5.5个月(95%CI 4.7-6.4)。结论：Palbociclib在实际临床实践中的有效性和耐受性与Paloma-3临床试验中获得的结果很好地一致。尽管剂量调整存在差异，但这项研究表明，在这一患者群体中没有疗效-有效性差距。]
OBJECTIVE: The aim of this study was to assess the real-world effectiveness and tolerability of palbociclib combined with endocrine therapy for the treatment of hormone receptor positive (HR-positive), human epidermal growth factor receptor 2 negative (HER2-negative), advanced/metastatic breast cancer that progressed on previous endocrine therapy, and to compare these results with the outcomes of the PALOMA-3 clinical trial.METHODS: This study was a retrospective observational cohort study including all patients who started with palbociclib in the St. Antonius Hospital between September 1, 2016 and April 1, 2018 for the treatment of HR-positive, HER2-negative advanced/metastatic breast cancer that progressed on previous endocrine therapy. Individual patient data were collected from electronic medical records. Primary study outcomes were progression-free survival (PFS) and the number of permanent treatment discontinuations before disease progression due to adverse events (AEs). Secondary outcomes were the frequency of all (serious) AEs and the frequency of and reasons for dose reductions, -interruptions and cycle delays.RESULTS: A total of 46 patients were studied with a median follow-up of 13.0 months. Overall, the median PFS in real-world clinical practice was 10.0 months (95% confidence interval (CI) 4.9-15.1), compared with 9.5 months in PALOMA-3 (95% CI 9.2-11.0). Two patients discontinued treatment because of AEs. Neutropenia was the most frequent grade 3-4 AE, but with no febrile neutropenia events. Most AEs were managed with palbociclib dose modifications. Regarding these modifications, more cycle delays, less dose reductions, and less dose interruptions occurred in clinical practice compared with PALOMA-3 (59 vs 36%, 22 vs 34%, and 9 vs 54%, respectively). Patients who did not meet the PALOMA-3 study eligibility criteria (n = 16) showed a lower median PFS of 5.5 months (95% CI 4.7-6.4).CONCLUSION: The effectiveness and tolerability of palbociclib in real-world clinical practice corresponded well with the results obtaineds in the PALOMA-3 clinical trial. Despite the differences in dose modifications, this study suggests that there is no efficacy-effectiveness gap in this patient population.]