Blot Koenraad, Duchateau Luc, Lescrauwaet Benedicte, Liyanage Nilani, Ray David, Mirakhur Beloo, Vinik Aaron I
Alacrita Consulting Ltd, London, UK.
Department of Comparative Physiology and Biometrics, Faculty of Veterinary Medicine, Ghent University, Ghent, Belgium.
Patient Relat Outcome Meas. 2019 Oct 29;10:335-343. doi: 10.2147/PROM.S219982. eCollection 2019.
The purpose of this analysis of patient-reported outcomes from the ELECT (Evaluation of Lanreotide Depot/Autogel Efficacy and Safety as a Carcinoid Syndrome Treatment) trial (NCT00774930) was to explore the effect of lanreotide on symptoms of carcinoid syndrome. Specifically, this post hoc analysis was designed to identify the most important patient-reported outcomes for patients in ELECT.
The post hoc analysis of ELECT, a placebo-controlled study of lanreotide in patients with neuroendocrine tumors, evaluated patient-reported outcomes during the double-blind phase of the trial, specifically daily diarrhea and flushing symptoms, octreotide rescue use, and the EORTC QLQ-C30 and QLQ-GINET21 questionnaires at baseline and week 12. Principal component (PC) analysis was applied on baseline data to identify independent variable clusters and clinically meaningful summary measures that highly correlated to these PCs. From those, the minimum clinical important differences were derived so to perform a responder analysis.
The three largest PCs captured 42.9% of the variation among baseline variables. The C30 summary score (C30-SS), diarrhea burden, and flushing burden were highly correlated with PC1, PC2, and PC3, respectively. Lanreotide patients were more likely to experience an improvement on the C30-SS (risk ratio [RR] 2.42; =0.023), diarrhea burden (RR 2.85; =0.005), and flushing burden (RR 1.39; =0.31) compared to placebo patients. Lanreotide-treated patients have a higher probability of being a responder on at least one of the three domains of C30-SS, diarrhea burden, or flushing burden compared to placebo patients (RR 1.48; =0.06).
The higher response rates in the diarrhea burden are consistent with the previously reported effects of lanreotide on octreotide rescue medication use, while the findings of a greater efficacy of lanreotide vs placebo in the quality-of-life domains represent a novel aspect in the benefits of lanreotide.
ClinicalTrials.gov identifier: NCT00774930.
本分析旨在探讨兰瑞肽对类癌综合征症状的影响，该分析基于ELECT（长效兰瑞肽注射剂/自凝胶治疗类癌综合征的疗效和安全性评估）试验（NCT00774930）中患者报告的结局。具体而言，这项事后分析旨在确定ELECT试验中对患者最重要的患者报告结局。
ELECT试验是一项针对神经内分泌肿瘤患者的兰瑞肽安慰剂对照研究，其事后分析评估了试验双盲阶段患者报告的结局，具体包括每日腹泻和潮红症状、奥曲肽解救用药情况，以及基线和第12周时的欧洲癌症研究与治疗组织核心问卷（EORTC QLQ-C30）和神经内分泌肿瘤问卷（QLQ-GINET21）。对基线数据进行主成分（PC）分析，以识别自变量聚类以及与这些主成分高度相关的具有临床意义的汇总指标。据此得出最小临床重要差异，以便进行反应者分析。
最大的三个主成分占基线变量变异的42.9%。C30汇总评分（C30-SS）、腹泻负担和潮红负担分别与主成分1、主成分2和主成分3高度相关。与安慰剂组患者相比，接受兰瑞肽治疗的患者在C30-SS（风险比[RR] 2.42；P = 0.023）、腹泻负担（RR 2.85；P = 0.005）和潮红负担（RR 1.39；P = 0.31）方面更有可能出现改善。与安慰剂组患者相比，接受兰瑞肽治疗的患者在C30-SS、腹泻负担或潮红负担这三个领域中至少有一个领域成为反应者的概率更高（RR 1.48；P = 0.06）。
腹泻负担方面较高的反应率与先前报道的兰瑞肽对奥曲肽解救用药的影响一致，而兰瑞肽在生活质量领域比安慰剂更有效的研究结果代表了兰瑞肽益处的一个新方面。
ClinicalTrials.gov标识符：NCT00774930。
