Department of Clinical Epidemiology, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands.
Imaging Division, University Medical Center Utrecht, Utrecht, The Netherlands.
Breast Cancer Res Treat. 2017 Jul;164(2):445-450. doi: 10.1007/s10549-017-4242-4. Epub 2017 Apr 25.
In oncology, RCTs are often beset by slow recruitment, limited generalizability, and strong preferences for interventions by patients and physicians. The cohort multiple randomized controlled trial (cmRCT) is an innovative design with the potential to overcome those challenges. In cmRCT, a prospective cohort serves as an infrastructure for multiple RCTs. We implemented cmRCT in a clinical breast cancer setting by creating UMBRELLA-a large prospective cohort of breast cancer and DCIS patients/survivors.
For all participants, clinical data and patient-reported outcomes (PROs, i.e., quality of life, fatigue, anxiety and depression, physical activity, work ability, and cosmetic satisfaction) are being collected at regular time-intervals for a period of 10 years. These data are being used both for observational and randomized studies. For each intervention to be tested against standard care, a subcohort of eligible patients is identified within UMBRELLA. From this subcohort, a random sample of patients is offered the intervention. Their outcomes are compared to the outcomes of patients receiving standard care.
So far, between October 2013 and July 2016, we have recruited 1308 participants. In this period, 1308/1486 (88%) patients who were invited for participation in UMBRELLA consented to cohort participation. Of these patients, 1138 (87%) gave broad consent for randomization to future interventions. Return rate for PROs at baseline were 80%, and varied from 67 to 74% during follow-up. Several observational studies-and the first randomized intervention study-are currently ongoing.
Results from UMBRELLA show that this novel study design is feasible and acceptable to patients in a clinical breast cancer setting. We invite researchers who are interested in conducting randomized or observational studies within the UMBRELLA cohort to contact the UMBRELLA scientific advisory board.
在肿瘤学中，随机对照试验常常面临招募缓慢、普遍性有限以及患者和医生对干预措施的强烈偏好等问题。队列多项随机对照试验（cmRCT）是一种具有克服这些挑战潜力的创新设计。在 cmRCT 中，前瞻性队列为多项 RCT 提供了基础。我们通过创建 UMBRELLA（一个大型乳腺癌和 DCIS 患者/幸存者的前瞻性队列），在临床乳腺癌环境中实施了 cmRCT。
对于所有参与者，临床数据和患者报告的结果（PROs，即生活质量、疲劳、焦虑和抑郁、身体活动、工作能力和美容满意度）在 10 年内定期收集。这些数据既用于观察性研究，也用于随机研究。对于要与标准护理进行比较的每一项干预措施，都会在 UMBRELLA 中确定符合条件的患者亚组。从这个亚组中，随机抽取一部分患者接受干预措施。将他们的结果与接受标准护理的患者的结果进行比较。
截至 2013 年 10 月至 2016 年 7 月，我们共招募了 1308 名参与者。在此期间，邀请参加 UMBRELLA 的 1486 名患者中有 1308 名（88%）同意参与队列研究。其中，1138 名（87%）患者同意广泛随机化接受未来的干预措施。PROs 的基线回复率为 80%，随访期间从 67%到 74%不等。目前正在进行几项观察性研究和第一项随机干预研究。
UMBRELLA 的结果表明，这种新的研究设计在临床乳腺癌环境中对患者是可行且可接受的。我们邀请有兴趣在 UMBRELLA 队列中进行随机或观察性研究的研究人员联系 UMBRELLA 科学顾问委员会。
