Department of Radiology and Nuclear Medicine, University Hospital of Magdeburg, Magdeburg, Germany.
Department of Clinical Oncology, Odense University Hospital, Odense, Denmark.
J Clin Oncol. 2020 Aug 20;38(24):2773-2781. doi: 10.1200/JCO.19.02790. Epub 2020 Jun 25.
We conducted a post hoc analysis of the vandetanib phase III trial involving patients with advanced medullary thyroid cancer (MTC) to assess the efficacy and safety of vandetanib in patients with progressive and symptomatic MTC. The primary objective of the analysis was to determine progression-free survival (PFS) of these patients.
Eligible patients from the ZETA trial were divided into 4 disease severity subgroups: progression and symptoms, symptoms only, progression only, and no progression and no symptoms assessed at baseline. PFS, determined from objective tumor measurements performed by the local investigator, overall survival (OS), time to worsening of pain (TWP), and objective response rate (ORR) were evaluated.
Of the 331 patients in this trial, 184 had symptomatic and progressive disease at baseline. In this subgroup, results were similar in magnitude to those observed in the overall trial for PFS (hazard ratio [HR], 0.43; 95% CI, 0.28 to 0.64; < .0001), OS (HR, 1.08; 95% CI, 0.72 to 1.61; = .71), and TWP (HR, 0.67; 95% CI, 0.43 to 1.04; = .07), and the observed adverse events were consistent with the known safety profile of vandetanib. In this subgroup, the ORR was 37% in the treatment arm versus 2% in the placebo arm.
Vandetanib demonstrated clinical benefit-specifically, increased PFS-in patients with symptomatic and progressive MTC.
我们对涉及晚期甲状腺髓样癌（MTC）患者的凡德他尼 III 期试验进行了事后分析，以评估凡德他尼在进展性和有症状的 MTC 患者中的疗效和安全性。该分析的主要目的是确定这些患者的无进展生存期（PFS）。
ZETA 试验中符合条件的患者被分为 4 个疾病严重程度亚组：基线时具有进展和症状、仅有症状、仅有进展和无进展且无症状。无进展生存期（PFS）是根据当地研究者进行的客观肿瘤测量来确定的，总生存期（OS）、疼痛恶化时间（TWP）和客观缓解率（ORR）也进行了评估。
在这项试验中，331 名患者中有 184 名基线时具有有症状和进展性疾病。在该亚组中，结果与整个试验中的 PFS（风险比 [HR]，0.43；95%置信区间，0.28 至 0.64；<0.0001）、OS（HR，1.08；95%置信区间，0.72 至 1.61；=0.71）和 TWP（HR，0.67；95%置信区间，0.43 至 1.04；=0.07）的观察结果相似，且观察到的不良反应与凡德他尼已知的安全性特征一致。在该亚组中，治疗组的 ORR 为 37%，而安慰剂组为 2%。
凡德他尼在有症状和进展性 MTC 患者中显示出了临床获益——特别是延长了 PFS。
