Hannouf Malek B, Winquist Eric, Mahmud Salaheddin M, Brackstone Muriel, Sarma Sisira, Rodrigues George, Rogan Peter K, Hoch Jeffrey S, Zaric Gregory S
Department of Epidemiology and Biostatistics, Schulich School of Medicine and Dentistry, Western University, 1255 Western Road, London, ON, N6G 0N1, Canada.
Ivey Business School, Western University, London, ON, Canada.
Pharmacoecon Open. 2018 Sep;2(3):255-270. doi: 10.1007/s41669-017-0051-2.
Several genomic tests have recently been developed to identify the primary tumour in cancer of unknown primary tumour (CUP). However, the value of identifying the primary tumour in clinical practice for CUP patients remains questionable and difficult to prove in randomized trials.
We aimed to assess the clinical and economic value of primary tumour identification in CUP using a retrospective matched cohort study.
We used the Manitoba Cancer Registry to identify all patients initially diagnosed with metastatic cancer between 2002 and 2011. We defined patients as having CUP if their primary tumour was found 6 months or more after initial diagnosis or never found during the course of disease. Otherwise, we considered patients to have metastatic cancer from a known primary tumour (CKP). We linked all patients with Manitoba Health databases to estimate their direct healthcare costs using a phase-of-care approach. We used the propensity score matching technique to match each CUP patient with a CKP patient on clinicopathologic characteristics. We compared treatment patterns, overall survival (OS) and phase-specific healthcare costs between the two patient groups and assessed association with OS using Cox regression adjustment.
Of 5839 patients diagnosed with metastatic cancer, 395 had CUP (6.8%); 1:1 matching created a matched group of 395 CKP patients. CUP patients were less likely to receive surgery, radiation, hormonal and targeted therapy and more likely to receive cytotoxic empiric chemotherapeutic agents. Having CUP was associated with reduced OS (hazard ratio [HR] 1.31; 95% confidence interval 1.1-1.58), but this lost statistical significance with adjustment for treatment differences. CUP patients had a significant increase in the mean net cost of initial diagnostic workup before diagnosis and a significant reduction in the mean net cost of continuing cancer care.
Identifying the primary tumour in CUP patients might enable the use of more effective therapies, improve OS and allow more efficient allocation of healthcare resources.
最近已开发出几种基因组检测方法，以确定原发性不明肿瘤（CUP）患者的原发肿瘤。然而，在临床实践中识别CUP患者原发肿瘤的价值仍存在疑问，且难以在随机试验中得到证实。
我们旨在通过一项回顾性匹配队列研究，评估识别CUP患者原发肿瘤的临床和经济价值。
我们利用曼尼托巴癌症登记处，确定2002年至2011年间最初被诊断为转移性癌症的所有患者。如果患者的原发肿瘤在初次诊断后6个月或更长时间才被发现，或者在疾病过程中从未被发现，我们将其定义为患有CUP。否则，我们认为患者患有已知原发肿瘤的转移性癌症（CKP）。我们将所有患者与曼尼托巴省卫生数据库进行关联，采用护理阶段方法估计其直接医疗费用。我们使用倾向得分匹配技术，根据临床病理特征将每位CUP患者与一名CKP患者进行匹配。我们比较了两组患者的治疗模式、总生存期（OS）和特定阶段的医疗费用，并使用Cox回归调整评估与OS的关联。
在5839例被诊断为转移性癌症的患者中，395例患有CUP（6.8%）；1:1匹配产生了一个由395例CKP患者组成的匹配组。CUP患者接受手术、放疗、激素和靶向治疗的可能性较小，而接受细胞毒性经验性化疗药物的可能性较大。患有CUP与OS降低相关（风险比[HR]1.31；95%置信区间1.1 - 1.58），但在对治疗差异进行调整后，这一差异失去了统计学意义。CUP患者在诊断前初始诊断检查的平均净成本显著增加，而持续癌症护理的平均净成本显著降低。
识别CUP患者的原发肿瘤可能有助于使用更有效的治疗方法，改善OS，并实现医疗资源更有效的分配。
