Background: 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.
Materials and methods: 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).
Results: 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 EGFR mutations (EGFRm) and ALK-rearrangements (ALKr). There were no HUS differences by BM status overall and in subsets by demographics. In multivariable analyses, superior HUS was associated with having EGFRm/ALKr (p < .0001), no prior radiation for extracranial disease (p < .001), and both intracranial (p = .002) and extracranial disease control (p < .01). HUS correlated with multiple elements of the FACT-Br and tests of NCF.
Conclusion: Having BM in lung cancer is not associated with inferior HUS in a population enriched for EGFRm and ALKr. Patients exhibiting disease control and those with oncogene-addicted tumors have superior HUS.
Implications for practice: In the setting of EGFR mutations or ALK 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.
摘要
背景。在肺癌患者中，脑转移(BM)及其治疗会带来沉重的经济负担，生活质量也会随之变差。在倡导靶向治疗的时代，通过健康效用评分 (HUS) 取得的真实世界证据对经济分析至关重要。
材料和方法。在一项前瞻性观察队列研究 (2014‐2016) 中，门诊 IV 期肺癌患者完成了人口统计调查和 EQ‐5D‐3L 调查(以取得 HUS)。根据图表得出健康状况和临床病理变量。患者按有无 BM 进行分组；采用回归分析确定 HUS 的相关因素。一部分患者预期接受并完成了神经认知功能 (NCF) 测试和/或 FACT‐brain (FACT‐Br) 问卷，随后与HUS(Spearman 系数；回归分析)进行相关性分析。
结果。在 519 例 1 686 EQ‐5D‐3L 衍生性 HUS 患者中，94 例 (18%) 完成了 NCF 测试，107 例 (21%) 完成了 FACT‐Br 问卷调查；301 例 (58%) 未出现过 BM，24 例 (5%) 在研究期间首次出现 BM，194 例 (37%) 在入组研究时便出现了 BM。(46%)样本存在 EGFR 突变 (EGFRm) 和 ALK 重排(ALKr)。总体 BM 状态和人口统计学亚组人群 HUS 无显著性差异。多变量分析结果显示，较好的 HUS 与 EGFRm/ALKr (p < 0.000 1)、既往无颅外放疗 (p < 0.001)、颅内 (p = 0.002) 和颅外疾病控制 (p < 0.01) 相关。HUS 与 FACT‐B 和 NCF 测试的多个元素相关。
结论。在 EGFRm 和 ALKr 富集的人群中，肺癌 BM 与 HUS 差无关。表现出疾病控制的患者和癌基因依赖性肿瘤患者的 HUS 更优。
实践意义:在 EGFR 突变或 ALK 重排非小细胞肺癌 (NSCLC) 的背景下，脑转移诊断不再被认为患者具较差的健康状态，这表明在靶向治疗时代，需要重新对 NSCLC 进行经济分析。此外，EQ‐5D 问卷与健康相关的生活质量和神经认知评分相关，这表明，未来的临床研究应对 EQ‐5D 展开进一步探索。
Keywords: Brain metastases; EQ‐5D; Health utility scores; Metastatic lung cancer.
© AlphaMed Press 2019.