Department of Medical Oncology, Institut Roi Albert II, Cliniques Universitaires Saint-Luc, Belgium; Institute for Clinical and Experimental Research (POLE MIRO), Université Catholique de Louvain, Brussels, Belgium.
Department of Drug Development and Innovation, Institut Curie, Paris & Saint-Cloud, Paris, France; INSERM U900 Research Unit, Saint-Cloud, France; Versailles-Saint-Quentin-en-Yvelines University, Montigny-le-Bretonneux, France.
Ann Oncol. 2018 Dec 1;29(12):2313-2327. doi: 10.1093/annonc/mdy452.
The molecular landscape of squamous cell carcinoma of the head and the neck (SCCHN) has been characterized and actionable or targetable genomic alterations have been identified. However, targeted therapies have very limited activity in unselected SCCHN, and the current treatment strategy is still based on tumor location and disease stage and not on tumor biology. Trying to select upfront the patients who will benefit from a specific treatment might be a way to improve patients' outcome. With the objective of optimizing the activity of targeted therapies and immunotherapy, we have designed an umbrella biomarker-driven study dedicated to recurrent and/or metastatic SCCHN patients (EORTC-1559-HNCG, NCT03088059). In this article, we review not only the different trial designs for biomarker-driven studies with their respective advantages and opportunities but also the potential pitfalls that led to the design of the EORTC-1559-HNCG protocol. We also discuss the scientific and logistic challenges of biomarker-driven trials.
头颈部鳞状细胞癌 (SCCHN) 的分子图谱已经得到了描述，并且已经确定了可操作或可靶向的基因组改变。然而，在未选择的 SCCHN 中，靶向治疗的活性非常有限，目前的治疗策略仍然基于肿瘤位置和疾病阶段，而不是肿瘤生物学。尝试在初始阶段选择将从特定治疗中受益的患者可能是改善患者预后的一种方法。为了优化靶向治疗和免疫治疗的活性，我们设计了一项伞式生物标志物驱动的研究，专门针对复发性和/或转移性 SCCHN 患者（EORTC-1559-HNCG，NCT03088059）。在本文中，我们不仅回顾了不同的试验设计用于生物标志物驱动的研究及其各自的优势和机会，还讨论了导致 EORTC-1559-HNCG 方案设计的潜在陷阱。我们还讨论了生物标志物驱动试验的科学和后勤挑战。
