Introduction: Somatostatin analogues (SSA) prolong progression-free survival (PFS) in patients with well-differentiated gastroenteropancreatic neuroendocrine tumors (GEP-NETs). However, the eligibility criteria in randomized clinical trials (RCTs) have been restricted, which contrasts with the vast heterogeneity found in NETs. Methods: We identified patients with well-differentiated (Ki67% ≤20%), metastatic GEP-NETs treated in first-line with SSA monotherapy from the Spanish R-GETNE registry. The therapeutic effect was evaluated using a Bayesian Cox model. The objective was to compare survival-based outcomes from real world clinical practice versus RCTs. Results: The dataset contained 535 patients with a median age of 62 years (range: 26-89). The median Ki67% was 4 (range: 0-20). The most common primary tumor sites were: midgut, 46%; pancreas, 34%; unknown primary, 10%; and colorectal, 10%. Half of the patients received octreotide LAR (n=266) and half, lanreotide autogel (n=269). The median PFS was 28.0 months (95% CI, 22.1-32.0) for octreotide vs 30.1 months (95% CI, 23.1-38.0) for lanreotide. The overall hazard ratio for lanreotide vs octreotide was 0.90 (95% credible interval, 0.71-1.12). The probability of effect sizes >30% with lanreotide vs octreotide was 2% and 6% for midgut and foregut NENs, respectively. Conclusion: Our study evaluated the external validity of RCTs examining SSAs in the real world, as well as the main effect-modifying factors (progression status, symptoms, tumor site, specific metastases, and analytical data).. Our results indicate that both octreotide LAR and lanreotide autogel had a similar effect on PFS. Consequently, both represent valid alternatives in patients with well-differentiated, metastatic GEP-NENs.


中文翻译：

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生长抑素类似物试验在晚期神经内分泌肿瘤中的外部有效性：GETNE-TRASGU 研究

简介：生长抑素类似物 (SSA) 可延长高分化胃肠胰神经内分泌肿瘤 (GEP-NETs) 患者的无进展生存期 (PFS)。然而，随机临床试验 (RCT) 的资格标准受到限制，这与 NET 中发现的巨大异质性形成鲜明对比。方法：我们从西班牙 R-GETNE 登记处确定了在一线接受 SSA 单药治疗的高分化（Ki67% ≤20%）转移性 GEP-NET 患者。使用贝叶斯 Cox 模型评估治疗效果。目的是比较现实世界临床实践与 RCT 的基于生存的结果。结果：该数据集包含 535 名患者，中位年龄为 62 岁（范围：26-89）。Ki67% 的中位数为 4（范围：0-20）。最常见的原发肿瘤部位是：中肠，46%；胰腺，34%；未知初级，10%；和结直肠癌，10%。一半的患者接受奥曲肽 LAR (n=266) 和一半的兰瑞肽 autogel (n=269)。奥曲肽的中位 PFS 为 28.0 个月（95% CI，22.1-32.0），而兰瑞肽为 30.1 个月（95% CI，23.1-38.0）。兰瑞肽与奥曲肽的总体风险比为 0.90（95% 可信区间，0.71-1.12）。对于中肠和前肠 NEN，兰瑞肽与奥曲肽的效应大小 > 30% 的概率分别为 2% 和 6%。结论：我们的研究评估了在现实世界中检查 SSA 的 RCT 的外部有效性，以及主要的影响调节因素（进展状态、症状、肿瘤部位、特异性转移和分析数据）。我们的结果表明， LAR 和兰瑞肽 autogel 对 PFS 有相似的影响。所以，