晚期肉瘤治疗的临床试验设计方法

B. Petek, S. Pollack, A. Constantinidou, Robin L. Jones
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引用次数: 0

摘要

肉瘤是影响结缔组织的罕见异质性间充质肿瘤。多年来,以阿霉素为基础的化疗一直是转移性软组织肉瘤患者的主要全身治疗选择。这种“一刀切”的方法带来了边际效益,但在胃肠道间质肿瘤中引入酪氨酸激酶抑制剂已经表明,亚型特异性、分子靶向治疗可以带来重大进展。下一代测序已经发现了许多肉瘤组织学亚型的生物学性质,现在的重点是评估每个亚组的新疗法。然而,由于这些肿瘤的罕见性和异质性,向临床的过渡既具有挑战性又旷日持久。肿瘤学三期试验的高失败率表明,临床试验可能很难进行,尤其是在像肉瘤这样的罕见癌症中。在这篇综述中,作者对肉瘤研究中更好的临床试验设计的潜在方法进行了评估。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Clinical trial design methodologies for advanced sarcoma therapy
Abstract Sarcomas are rare and heterogeneous mesenchymal tumors affecting connective tissue. For many years, doxorubicin-based chemotherapy has been the main systemic therapy option for patients with metastatic soft tissue sarcoma. This ‘one size fits all’ approach has led to marginal benefit, but the introduction of tyrosine kinase inhibitors in gastrointestinal stromal tumors has shown that sub-type-specific, molecularly targeted therapy can lead to significant advances. Next generation sequencing has led to the discovery of the biologic nature of many histological sub-types of sarcomas, and now an emphasis has been placed on assessment of novel therapies for each sub-group. However, the transition into the clinic is both challenging and protracted due to the rarity and heterogeneity of these tumors. The high failure rate of phase III trials in oncology has shown that clinical trials can be difficult to run, especially in rare forms of cancer like sarcomas. In this review, the authors provide an evaluation of the potential approaches toward better clinical trial design in sarcoma studies.
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