Verweij Jaap
Erasmus University Medical Center, Rotterdam, The Netherlands.
Oncologist. 2008;13 Suppl 2:27-31. doi: 10.1634/theoncologist.13-S2-27.
Despite extensive research efforts over the past two decades to identify effective agents for the treatment of soft tissue sarcomas, few agents are available, and with modest utility. There is a high unmet medical need to develop novel therapies for the treatment of patients with soft tissue sarcomas. Clinical trials for soft tissue sarcomas should be optimally designed, and it is crucial that they identify and define the desired clinical outcome. Survival is often the ultimate endpoint; however, physiological and biological markers are often used to predict the potential therapeutic benefit of a new agent. These endpoints can be easily measured, but can lead to false-positive results and do not take into account the complicated nature of soft tissue sarcomas. Alternative endpoints that are currently being evaluated include the progression-free survival rate, time to progression, tumor growth rate, and progression arrest rate. This article discusses some of the limitations of current endpoint criteria and potential endpoint criteria that could be used to evaluate treatment options for patients with soft tissue sarcomas.
尽管在过去二十年里人们付出了大量研究努力来确定治疗软组织肉瘤的有效药物，但可用的药物很少，且效用有限。开发治疗软组织肉瘤患者的新疗法仍存在高度未满足的医疗需求。软组织肉瘤的临床试验应进行优化设计，确定并定义预期的临床结果至关重要。生存通常是最终终点；然而，生理和生物学标志物常被用于预测新药的潜在治疗益处。这些终点易于测量，但可能导致假阳性结果，且未考虑软组织肉瘤的复杂性质。目前正在评估的替代终点包括无进展生存率、疾病进展时间、肿瘤生长率和疾病进展停滞率。本文讨论了当前终点标准的一些局限性以及可用于评估软组织肉瘤患者治疗方案的潜在终点标准。
