NC ProCESS: a Stakeholder-Driven, Population-Based Prospective Cohort Study

描述(由申请人提供)：根据医学研究所和卫生保健研究和质量机构的说法，早期(局部)前列腺癌治疗方案的比较有效性研究是一个“最优先”的主题。目前，患者和医生缺乏关于现代治疗方案(包括主动监测)的潜在益处和危害的必要信息，无法做出明智的决定，而在研究证据表明与旧疗法相比，新的和更昂贵的治疗技术的益处和危害之前，新的和更昂贵的治疗技术被迅速采用。之前的一些研究甚至表明，较新的手术和放射治疗实际上可能会比较旧的治疗引起更多的副作用。因此，迫切需要对现代治疗方案进行研究，以帮助患者和医生做出治疗决策，并向政策制定者通报本地化前列腺癌治疗方案的比较优势和危害。北卡罗来纳州前列腺癌比较有效性和存活率研究(NC PROCESS)是一项基于人群的队列研究，患者在确诊后几周内入选，并进行纵向跟踪，以评估患者报告的结果(PRO)和不同治疗后的癌症控制。最初为期3年的资助使2011-2013年间成功招募了这一庞大而多样化的队列，使用独特的NC癌症登记系统，使所有患者能够在治疗前登记，这对最大限度地减少登记和治疗中的潜在偏见很重要 数据收集(尤其是PRO)。这项目前的提案将在5-6年的随访中评估患者的结果，为患者、医生和政策制定者提供及时的和目前无法获得的信息。目前的研究对于从这个丰富的队列中产生有意义的结果，实现具体的目标(如下)和测试关于不同治疗方案之间的比较有效性的假设是必不可少的。NC流程是与包括患者在内的利益相关者合作设计的，他们将在整个研究过程中继续为研究人员提供建议，并将帮助解释研究结果和传播。具体目标包括：目标1：通过积极监测、开放根治性前列腺癌切除术、微创根治性前列腺癌切除术、调强放射治疗和近距离放射治疗，直接比较患者报告的前列腺癌患者的具体和全球生活质量(QOL)、对前列腺癌的焦虑和5年内的决定悔恨。我们将探索与立体定向放射治疗和质子治疗的比较。目的2：直接比较不同治疗方案在随访6年内的无癌复发(FFCR)。多学科研究团队在前列腺癌研究方面拥有丰富的经验，这项研究的结果将极大地推进前列腺癌本地化比较有效性研究领域，并改善对这一常见疾病患者的护理。一个全国性的利益相关者小组将帮助向美国各地的患者、医生和政策制定者传播重要的研究结果。

DESCRIPTION (provided by applicant): Comparative effectiveness research in early (localized) prostate cancer treatment options is a "highest priority" topic according to the Institute of Medicine and the Agency for Healthcare Research and Quality. Currently, patients and physicians lack the necessary information on potential benefits and harms of modern treatment options (including active surveillance) to make informed decisions, while newer and more expensive treatment technologies become rapidly adopted before research evidence demonstrates their benefit and harms compared to older treatments. Some prior studies have even suggested that newer surgical and radiation treatments could actually cause more side effects than older treatments. Therefore, there is an urgent need for research on modern treatment options to help patients and physicians make treatment decisions, and inform policymakers about the comparative benefits and harms of localized prostate cancer treatment options. The North Carolina Prostate cancer Comparative Effectiveness & Survivorship Study (NC ProCESS) is a population-based cohort of patients who were enrolled within weeks of diagnosis, and followed longitudinally to assess patient-reported outcomes (PRO) and cancer control after different treatments. An initial 3-year funding allowed successful recruitment of thi large and diverse cohort from 2011-2013, using a unique NC Cancer Registry system enabling all patient enrollment prior to treatment, important to minimize potential biases in enrollment and data collection (especially PRO). This current proposal will assess patient outcomes at 5-6 years' follow-up, providing timely and currently unavailable information to patients, physicians, and policymakers. This current study is essential to yield meaningful results from this rich cohort, achieve the specific aims (below) and test hypotheses on the comparative effectiveness among different treatment options. NC ProCESS was designed in collaboration with stakeholders including patients, who will continue to advise the investigators throughout the study and will help with interpreting the study results and dissemination. The Specific Aims include: Aim 1: To directly compare patient-reported prostate-cancer specific and global quality of life (QOL), anxiety regarding prostate cancer and decisional regret within 5 years of follow-up in a cohort of men with localized prostate cancer managed by active surveillance, open radical prostatectomy, minimally-invasive radical prostatectomy, intensity-modulated radiation therapy, and brachytherapy. We will explore comparisons with stereotactic radiation therapy and proton therapy. Aim 2: To directly compare freedom-from cancer recurrence (FFCR) within 6 years of follow-up among different options. The multidisciplinary team of investigators has extensive experience in prostate cancer research, and results from this study will significantly advance the field of localized prostate cancer comparative effectiveness research, and improve the care of patients with this common disease. A national group of stakeholders will help disseminate significant study findings to patients throughout the US, physicians, and policymakers.