PHMR Health Economics, Pricing and Reimbursement, London NW1 8XY, United Kingdom.
Kings Health Partners NET centre, Kings College Hospital London, London SE5 9RS, United Kingdom.
World J Gastroenterol. 2020 Jul 7;26(25):3686-3711. doi: 10.3748/wjg.v26.i25.3686.
Gastroenteropancreatic neuroendocrine tumours (GEP-NETs) are slow-growing cancers that arise from diffuse endocrine cells in the gastrointestinal tract (GI-NETs) or the pancreas (P-NETs). They are relatively uncommon, accounting for 2% of all gastrointestinal malignancies. The usual treatment options in advanced GEP-NET patients with metastatic disease include chemotherapy, biological therapies, and peptide receptor radionuclide therapy. Understanding the impact of treatment on GEP-NET patients is paramount given the nature of the disease. Health-related quality of life (HRQoL) is increasingly important as a concept reflecting the patients' perspective in conjunction with the disease presentation, severity and treatment.
To conduct a systematic literature review to identify literature reporting HRQoL data in patients with GEP-NETs between January 1985 and November 2019.
The PRISMA guiding principles were applied. MEDLINE, Embase and the Cochrane library were searched. Data extracted from the publications included type of study, patient population data (mid-gut/hind-gut/GI-NET/P-NET), sample size, intervention/comparators, HRQoL instruments, average and data spread of overall and sub-scores, and follow-up time for data collection.
Forty-three publications met the inclusion criteria. The heterogeneous nature of the different study populations was evident; the percentage of female participants ranged between 30%-60%, whilst average age ranged from 53.8 to 67.0 years. Eight studies investigated GI-NET patients only, six studies focused exclusively on P-NET patients and the remaining studies involved both patient populations or did not report the location of the primary tumour. The most commonly used instrument was the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-C30 ( = 28) with consistent results across studies; the GI-NET-specific module Quality of Life Questionnaire-GINET21 was used in six of these studies. A number of randomised trials demonstrated no HRQoL changes between active treatment and placebo arms. The Phase III NETTER-1 study provides the best data available for advanced GEP-NET patients; it shows that peptide receptor radionuclide therapy can significantly improve GEP-NET patients' HRQoL.
HRQoL instruments offer a means to monitor patients' general disease condition, disease progression and their physical and mental well-being. Instruments including the commonly used European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-C30 and GINET21 lack, however, validation and a defined minimal clinical important difference specifically for GI-NET and P-NET patients.
胃肠胰神经内分泌肿瘤(GEP-NETs)是一种生长缓慢的癌症,起源于胃肠道(GI-NETs)或胰腺(P-NETs)的弥散内分泌细胞。它们相对少见,占所有胃肠道恶性肿瘤的 2%。晚期 GEP-NET 伴转移疾病患者的常用治疗选择包括化疗、生物疗法和肽受体放射性核素治疗。鉴于疾病的性质,了解治疗对 GEP-NET 患者的影响至关重要。健康相关生活质量(HRQoL)作为一个反映患者观点的概念,与疾病表现、严重程度和治疗相结合,变得越来越重要。
进行系统文献综述,以确定 1985 年 1 月至 2019 年 11 月期间报告 GEP-NET 患者 HRQoL 数据的文献。
应用 PRISMA 指导原则。检索 MEDLINE、Embase 和 Cochrane 图书馆。从出版物中提取的数据包括研究类型、患者人群数据(中肠/后肠/GI-NET/P-NET)、样本量、干预/对照、HRQoL 工具、总体和子评分的平均值和数据分布,以及数据收集的随访时间。
43 篇文献符合纳入标准。不同研究人群的异质性显而易见;女性参与者的百分比在 30%-60%之间,而平均年龄在 53.8 至 67.0 岁之间。八项研究仅调查了 GI-NET 患者,六项研究专门针对 P-NET 患者,其余研究涉及两种患者人群或未报告原发肿瘤的位置。最常用的工具是欧洲癌症研究与治疗组织生活质量问卷-C30(=28),研究结果一致;其中六项研究使用了 GI-NET 特异性模块生活质量问卷-GINET21。一些随机试验表明,活性治疗与安慰剂组之间 HRQoL 无变化。第三阶段 NETTER-1 研究为晚期 GEP-NET 患者提供了最佳数据;它表明肽受体放射性核素治疗可显著改善 GEP-NET 患者的 HRQoL。
HRQoL 工具可用于监测患者的一般疾病状况、疾病进展以及身体和心理健康。然而,常用的欧洲癌症研究与治疗组织生活质量问卷-C30 和 GINET21 等工具缺乏针对 GI-NET 和 P-NET 患者的验证和定义的最小临床重要差异。