Ugalde Anna, Tothill Richard W, Quinn Stephen, Wong Hui-Li, Prall Owen, Mitchell Catherine, Wickramasinghe Nilmini, Fedele Clare, Richards Natalie, Todio Elizabeth, Bryant Cindy, Collins Louisa G, McLean Sarah, Ko Hyun Soo, Akhurst Tim, Steer Christopher, Gao Bo, Wong Mark, Georgiou Chloe, Karanth Narayan, Kuchel Anna, Nott Louise, Padinharakam Shamsudheen, Shackleton Mark, Collins Ian M, Singh Madhu, Wong Rachel, Wong Zee Wan, Butler Alexis, Sivakumaran Tharani, Schofield Penelope, Mileshkin Linda
Institute for Health Transformation, Faculty of Health, Deakin University, 221 Burwood Highway, Burwood, Geelong, Victoria, 3125, Australia.
Peter MacCallum Cancer Centre, Parkville, Victoria, Australia.
BMC Cancer. 2025 Jan 29;25(1):171. doi: 10.1186/s12885-025-13506-4.
People with malignancy of undefined primary origin (MUO) have a poor prognosis and may undergo a protracted diagnostic workup causing patient distress and high cancer related costs. Not having a primary diagnosis limits timely site-specific treatment and access to precision medicine. There is a need to improve the diagnostic process, and healthcare delivery and support for these patients. This trial aims to implement and evaluate an optimal model of care for people presenting with MUO to reduce time to diagnosis, improve patient experiences and reduce healthcare costs.
METHODS/DESIGN: This is a pragmatic stepped-wedge cluster randomised trial comparing a control phase of standard practice with an intervention phase. Patient inclusion criteria are: 1) age 18 years or older, 2) presenting with suspected metastatic malignancy without an obvious primary site on imaging, 3) clinically appropriate to undergo diagnostic work-up and 4) able to provide written or verbal consent. The intervention is a new model of care comprising four key components: standardised diagnostic workup, dedicated cancer care coordinators, virtual multidisciplinary meetings and a website resource for patients, carers and clinicians. The primary endpoint is the time to completion of minimum diagnostic workup. Secondary outcomes are whether the type of tumour is diagnosed, clinical trial participation, referral to palliative care, patient-reported physical, social and mental health, patient-reported understanding and uncertainty. Implementation outcomes include acceptability, feasibility, fidelity and adoption and health care use and costs. Intervention implementation will be supported using clinical leadership, education and reinforcement. Patients who consent to having their data collected will receive the model of care active at the site at the time of recruitment. Patients will complete a patient-reported outcomes questionnaire four months after study enrolment. A health economic analysis will be included. Across 15 hospitals, a total sample size of 240 is planned.
There is a lack of intervention research for people presenting with MUO. The stepped-wedge design seeks to mitigate the potential challenge of enrolling people with a poor prognosis and high symptom burden in trials. This research will generate important evidence with scalability for future research at trial completion.
ACTRN12622001504707.
原发灶不明的恶性肿瘤（MUO）患者预后较差，可能需要长时间的诊断检查，这会给患者带来痛苦，并导致高昂的癌症相关费用。没有明确的原发诊断会限制及时的针对特定部位的治疗以及精准医疗的应用。因此，需要改进这些患者的诊断流程、医疗服务和支持。本试验旨在实施并评估一种针对MUO患者的最佳照护模式，以缩短诊断时间、改善患者体验并降低医疗成本。
方法/设计：这是一项实用的阶梯楔形整群随机试验，将标准实践的对照阶段与干预阶段进行比较。患者纳入标准为：1）年龄18岁及以上；2）影像学检查显示疑似转移性恶性肿瘤且无明显原发部位；3）临床上适合进行诊断检查；4）能够提供书面或口头同意。干预措施是一种新的照护模式，包括四个关键组成部分：标准化诊断检查、专门的癌症护理协调员、虚拟多学科会议以及面向患者、护理人员和临床医生的网站资源。主要终点是完成最低限度诊断检查的时间。次要结局包括是否诊断出肿瘤类型、参与临床试验情况、转介至姑息治疗情况、患者报告的身体、社会和心理健康状况、患者报告的理解程度和不确定性。实施结局包括可接受性、可行性、保真度、采用情况以及医疗保健使用和成本。将通过临床领导力、教育和强化措施来支持干预措施的实施。同意收集其数据的患者将在招募时接受所在机构当时实施的照护模式。患者将在研究入组四个月后完成一份患者报告结局问卷。将进行卫生经济学分析。计划在15家医院共纳入240例样本。
目前针对MUO患者缺乏干预研究。阶梯楔形设计旨在减轻在试验中纳入预后不良和症状负担重的患者所面临的潜在挑战。本研究将在试验完成时产生具有可扩展性的重要证据，以供未来研究参考。
ACTRN12622001504707。
