Department of Medical Oncology and Hematology, Princess Margaret Cancer Centre, Toronto, Ontario, Canada.
Ontario Cancer Institute Biostatistics, Princess Margaret Cancer Centre, Toronto, Ontario, Canada.
Oncologist. 2019 Jul;24(7):e501-e509. doi: 10.1634/theoncologist.2018-0544. Epub 2019 Apr 5.
In lung cancer, brain metastases (BM) and their treatment are associated with high economic burden and inferior health-related quality of life. In the era of targeted therapy, real world evidence through health utility scores (HUS) is critical for economic analyses.
In a prospective observational cohort study (2014-2016), outpatients with stage IV lung cancer completed demographic and EQ-5D-3L surveys (to derive HUS). Health states and clinicopathologic variables were obtained from chart abstraction. Patients were categorized by the presence or absence of BM; regression analyses identified factors that were associated with HUS. A subset of patients prospectively completed neurocognitive function (NCF) tests and/or the FACT-brain (FACT-Br) questionnaire, which were then correlated with HUS (Spearman coefficients; regression analyses).
Of 519 patients with 1,686 EQ-5D-3L-derived HUS, 94 (18%) completed NCF tests and 107 (21%) completed FACT-Br; 301 (58%) never developed BM, 24 (5%) developed first BM during study period, and 194 (37%) had BM at study entry. The sample was enriched (46%) for mutations (m) and -rearrangements (r). There were no HUS differences by BM status overall and in subsets by demographics. In multivariable analyses, superior HUS was associated with having m/r ( < .0001), no prior radiation for extracranial disease ( < .001), and both intracranial ( = .002) and extracranial disease control ( < .01). HUS correlated with multiple elements of the FACT-Br and tests of NCF.
Having BM in lung cancer is not associated with inferior HUS in a population enriched for m and r. Patients exhibiting disease control and those with oncogene-addicted tumors have superior HUS.
In the setting of mutations or rearrangement non-small cell lung cancer (NSCLC), a diagnosis of brain metastases no longer consigns the patient to an inferior health state suggesting that new economic analyses in NSCLC are needed in the era of targeted therapies. Additionally, the EQ-5D questionnaire is associated with measures of health-related quality of life and neurocognitive scores suggesting this tool should be further explored in prospective clinical studies.
在肺癌中，脑转移（BM）及其治疗与高昂的经济负担和较差的健康相关生活质量有关。在靶向治疗时代，通过健康效用评分（HUS）获得的真实世界证据对于经济分析至关重要。
在一项前瞻性观察队列研究（2014-2016 年）中，IV 期肺癌门诊患者完成了人口统计学和 EQ-5D-3L 调查（以得出 HUS）。从图表摘录中获得健康状况和临床病理变量。根据是否存在 BM 对患者进行分类；回归分析确定了与 HUS 相关的因素。一组患者前瞻性地完成了神经认知功能（NCF）测试和/或 FACT-brain（FACT-Br）问卷，然后对其进行了 HUS 的相关性分析（Spearman 系数；回归分析）。
在 519 名患者的 1686 个 EQ-5D-3L 衍生的 HUS 中，94 名（18%）完成了 NCF 测试，107 名（21%）完成了 FACT-Br；301 名（58%）从未发生过 BM，24 名（5%）在研究期间首次发生 BM，194 名（37%）在研究开始时就有 BM。该样本（46%）为 突变（m）和 -重排（r）阳性。整体上 BM 状态和根据人口统计学分组的 HUS 无差异。多变量分析显示，更好的 HUS 与 m/r 相关（<.0001），无颅外疾病的既往放疗（<.001），以及颅内（=.002）和颅外疾病控制（<.01）相关。HUS 与 FACT-Br 的多个部分和 NCF 测试相关。
在富含 m 和 r 的人群中，肺癌患者的 BM 与较差的 HUS 无关。表现出疾病控制的患者和携带致癌基因依赖性肿瘤的患者具有更好的 HUS。
在 突变或 重排非小细胞肺癌（NSCLC）的情况下，脑转移的诊断不再意味着患者处于较差的健康状态，这表明在靶向治疗时代，需要对 NSCLC 进行新的经济分析。此外，EQ-5D 问卷与健康相关生活质量的衡量标准和神经认知评分相关，这表明该工具应该在未来的临床研究中进一步探索。
