Departments of Pathology and Oncology, General Hospital of Treviso, Piazza Ospedale 1, Treviso, Italy.
J Pathol. 2011 Apr;223(5):565-6. doi: 10.1002/path.2847.
'Chordoma' represents orphan sarcoma subtypes in which systemic treatments have so far proved ineffective. Two original papers in the Journal of Pathology have recently elucidated important steps of its molecular oncogenesis. The demonstration of gains of copy number of both T (brachyury homologue) and EGFR (epidermal growth factor receptor) genes in chordoma, as well as of their role in promoting cell proliferation, offers a strong preclinical rationale for exploring the inhibition of their products as a credible therapeutic strategy. When dealing with rare cancers, clinical trials based on a frequency-based statistical methodology are hardly feasible. To override the limitations of rarity, the clinical community is suggesting the adoption of a Bayesian methodology. The success of innovative clinical trials strongly depends upon the availability of preclinical evidences of efficacy. The papers discussed herein represent good examples of what is actually needed to set up a solid prior probability.
“脊索瘤”是一种孤儿肉瘤亚型，迄今为止，全身治疗对其均无效。《病理学杂志》最近发表的两篇原始论文阐明了其分子癌变过程中的重要步骤。脊索瘤中 T（短尾同源物）和 EGFR（表皮生长因子受体）基因拷贝数增加及其在促进细胞增殖中的作用的证明，为探索抑制其产物作为可信治疗策略提供了强有力的临床前依据。在处理罕见癌症时，基于频率的统计方法的临床试验几乎是不可行的。为了克服罕见性的局限性，临床界建议采用贝叶斯方法。创新性临床试验的成功在很大程度上取决于是否有有效的临床前证据。本文讨论的论文很好地说明了建立可靠先验概率所需要的实际内容。
