Patient-reported health state utilities in metastatic gastroenteropancreatic neuroendocrine tumours - an analysis based on the CLARINET study
Patient-reported health state utilities in metastatic gastroenteropancreatic neuroendocrine tumours - an analysis based on the CLARINET study
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DOI：
10.1186/s12955-017-0711-z
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发表时间：
2017-06-29
影响因子：
3.6
通讯作者：
Dinet, Jerome
中科院分区：
文献类型：
--
作者：
Meng, Yang;McCarthy, Grant;Dinet, Jerome
背景：胃肠胰神经内分泌肿瘤（GEP-NET）是一种罕见的癌症，最常见于胃肠系统或胰腺。然而，基于临床试验的患者报告的健康状态效用此前在该疾病领域尚未有报道。 方法：CLARINET 研究收集了稳定和进展疾病状态患者的 EORTC QLQ-C30 数据，尽管后者的数据由于试验设计而仅在疾病进展的早期阶段可用。使用已发布的算法，将数据映射到 EQ-5D 效用值。使用随机效应广义最小二乘模型来研究进展状态、肿瘤部位和其他患者特征对映射效用值的影响。结果：总共，使用 McKenzie 映射算法将 204 名患者的 1053 个观察结果映射到 EQ-5D 效用值。最终的随机效应模型包括年龄、性别、基线效用和进展状态作为协变量；由于 CLARINET 研究中的死亡人数有限，调查死亡时间效用是不可行的。肿瘤位置（中肠与胰腺）似乎并不影响效用。然而，在基本案例分析中，基于进展状态的效用差异具有统计显着性（p < 0.05），稳定和进展疾病的估计效用分别为 0.776 和 0.726。此外，情景分析显示，疾病进展的效用在数值上低于疾病稳定的效用，但这在使用替代 Longworth 映射算法的情况下可能不具有统计显着性。 结论：根据来自 CLARINET 研究的患者报告的健康相关生活质量 (HRQL) 数据，与疾病稳定状态相比，GEP-NET 患者在疾病进展状态下的效用值更差。进展性疾病状态下效用的下降可能被低估，因为进展性 HRQL 数据仅在试验中进展事件发生后不久收集。基于试验的估计效用可用于未来 GEP-NET 治疗的经济评估，并为医生提供有关 GEP-NET 中患者报告的生活质量结果的更多见解。
Background: Gastroenteropancreatic neuroendocrine tumours (GEP-NETs) are rare cancers most often found in the gastrointestinal system or the pancreas. However, patient-reported health state utilities based on clinical trials have not been previously reported in this disease area.Methods: The CLARINET study collected EORTC QLQ-C30 data from patients in both stable and progressive disease states, although data for the latter were only available during the early stage of progression due to trial design. Using published algorithms, data were mapped to EQ-5D utility values. Random-effects generalised least squares models were used to investigate the impacts of progression status, tumour site and other patient characteristics on mapped utility values.Results: In total, 1053 observations from 204 patients were mapped to EQ-5D utilities using the McKenzie mapping algorithm. The final random-effects model included age, gender, baseline utility and progression status as covariates; it was not feasible to investigate time-to-death utility due to a limit number of deaths in the CLARINET study. Tumour location (midgut vs pancreas) does not seem to affect utility. However, the difference in utilities based on progression status is statistically significant (p < 0.05) in the base case analysis, and the estimated utilities for stable and progressive disease are 0.776 and 0.726, respectively. Furthermore, scenario analyses showed that utility for progressive disease is numerically lower than for stable disease, but this may not be statistically significant in scenarios where alternative Longworth mapping algorithm was used.Conclusions: Patients with GEP-NETs experience worse utility values in the progressive disease state compared to the stable disease state, based on patient-reported health-related quality of life (HRQL) data from the CLARINET study. The decline of utility in the progressive disease state may be underestimated because progressive HRQL data were only collected shortly after the progression event in the trial. The estimated trial-based utilities can be used in future economic evaluations for GEP-NET treatments and to provide more insights to physicians on patient-reported quality of life outcomes in GEP-NETs.