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前瞻性非干预性SMARTrial

原文发布日期：2023-10-02

英文摘要：

摘要翻译：

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文章：

前瞻性非干预性SMARTrial

the prospective non-interventional SMARTrial

原文发布日期：2023-10-02

英文摘要：

Ex vivo drug response profiling is a powerful tool to study genotype–drug response associations and is being explored as a tool set for precision medicine in cancer. Here we conducted a prospective non-interventional trial to investigate feasibility of ex vivo drug response profiling for treatment guidance in hematologic malignancies (SMARTrial, NCT03488641). The primary endpoint to provide drug response profiling reports within 7 d was met in 91% of all study participants (N = 80). Secondary endpoint analysis revealed that ex vivo resistance to chemotherapeutic drugs predicted chemotherapy treatment failure in vivo. We confirmed the predictive value of ex vivo response to chemotherapy in a validation cohort of 95 individuals with acute myeloid leukemia treated with daunorubicin and cytarabine. Ex vivo drug response profiles improved ELN-22 risk stratification in individuals with adverse risk. We conclude that ex vivo drug response profiling is clinically feasible and has the potential to predict chemotherapy response in individuals with hematologic malignancies beyond clinically established genetic markers.

摘要翻译：

体外药物应答分析是一种强大的工具，用于研究基因型与药物反应之间的关系，并正在探索作为精准医疗工具集合在癌症中的应用。在此，我们开展了一项前瞻性、非干预性试验，旨在评估体外药物应答分析在血液系统恶性肿瘤治疗指导中的可行性（SMARTrial, NCT03488641）。主要终点是7天内提供应答报告，80名研究参与者中有91%的个体达到了这一目标。次要终点分析表明，体外对化疗药物的耐药性预测了体内化疗治疗失败。我们通过一个包含95例接受柔红霉素和卡扎鲁胺治疗的急性髓系白血病患者的验证组 cohort 验证了体外化疗反应的预测价值。体外药物应答资料在不良风险个体中改进了ELN-22风险分层。我们得出结论，体外药物应答分析在临床上是可行的，并有可能预测血液系统恶性肿瘤患者化疗反应，这些患者的遗传标记超出了目前在临床上确认的标准。