Division of Medical Oncology and Hematology, Princess Margaret Cancer Centre, University of Toronto, Toronto, ON, Canada.
Faculty of Medicine, University of Toronto, Toronto, ON, Canada.
Cancer Med. 2019 Dec;8(18):7542-7555. doi: 10.1002/cam4.2603. Epub 2019 Oct 24.
As the treatment landscape in patients with non-small cell lung cancer (NSCLC) harboring mutations in the epidermal growth factor receptor (EGFRm) continues to evolve, real-world health utility scores (HUS) become increasingly important for economic analyses.
In an observational cohort study, questionnaires were completed in EGFRm NSCLC outpatients, to include demographics, EQ-5D-based HUS and patient-reported toxicity and symptoms. Clinical and radiologic characteristics together with outcomes were extracted from chart review. The impact of health states, treatment type, toxicities, and clinical variables on HUS were evaluated.
Between 2014 and 2018, a total of 260 patients completed 994 encounters. Across treatment groups, patients with disease progression had lower HUS compared to controlled disease (0.771 vs 0.803; P = .01). Patients predominantly received gefitinib as the first-line EGFR tyrosine kinase inhibitor (TKI) (n = 157, mean-HUS = 0.798), whereas osimertinib (n = 62, mean-HUS = 0.806) and chemotherapy (n = 38, mean-HUS = 0.721) were more likely used in subsequent treatment lines. In longitudinal analysis, TKIs retained high HUS (>0.78) compared to chemotherapy (HUS < 0.74). There were no differences between the frequency or severity of toxicity scores in patients receiving gefitinib compared to osimertinib; however, TKI therapy resulted in fewer toxicities than chemotherapy (P < .05), with the exception of worse diarrhea and skin rash (P < .001). Severity in toxicities inversely correlated with HUS (P < .001). Clinico-demographic factors significantly affecting HUS included age, Eastern Cooperative Oncology Group Performance Score (ECOG PS), disease state, treatment group, and metastatic burden.
In a real-world EGFRm population, patients treated with gefitinib or osimertinib had similar HUS and toxicities, scores which were superior to chemotherapy. Health utility scores inversely correlated with patient-reported toxicity scores. In the era of targeted therapies, future economic analyses should incorporate real-world HUS.
随着表皮生长因子受体（EGFR）突变的非小细胞肺癌（NSCLC）患者的治疗格局不断发展，真实世界健康效用评分（HUS）对于经济分析变得越来越重要。
在一项观察性队列研究中，对 EGFRm NSCLC 门诊患者完成了问卷调查，内容包括人口统计学、基于 EQ-5D 的 HUS 以及患者报告的毒性和症状。从图表审查中提取了临床和影像学特征以及结局。评估了健康状况、治疗类型、毒性和临床变量对 HUS 的影响。
在 2014 年至 2018 年间，共有 260 名患者完成了 994 次就诊。在各个治疗组中，疾病进展患者的 HUS 低于疾病控制患者（0.771 与 0.803；P=0.01）。患者主要接受吉非替尼作为一线 EGFR 酪氨酸激酶抑制剂（TKI）（n=157，平均 HUS=0.798），而奥希替尼（n=62，平均 HUS=0.806）和化疗（n=38，平均 HUS=0.721）更可能在后续治疗线中使用。在纵向分析中，TKI 保持了较高的 HUS（>0.78），而化疗则较低（HUS<0.74）。接受吉非替尼治疗的患者与接受奥希替尼治疗的患者之间的毒性评分频率或严重程度没有差异；然而，TKI 治疗比化疗导致更少的毒性（P<.05），除了更严重的腹泻和皮疹（P<.001）。毒性的严重程度与 HUS 呈负相关（P<.001）。显著影响 HUS 的临床和人口统计学因素包括年龄、东部合作肿瘤学组表现评分（ECOG PS）、疾病状态、治疗组和转移性负担。
在真实世界的 EGFRm 人群中，接受吉非替尼或奥希替尼治疗的患者具有相似的 HUS 和毒性，这些评分优于化疗。健康效用评分与患者报告的毒性评分呈负相关。在靶向治疗时代，未来的经济分析应纳入真实世界的 HUS。
