离体药物反应分析是研究基因型-药物反应关联的强大工具，并且正在探索作为癌症精准医学的工具集。在这里，我们进行了一项前瞻性非干预性试验，以研究体外药物反应分析用于指导血液恶性肿瘤治疗的可行性（SMARTrial，NCT03488641）。 91% 的研究参与者达到了在 7 天内提供药物反应分析报告的主要终点 (N = 80)。次要终点分析显示，体外对化疗药物的耐药性预示着体内化疗治疗的失败。我们在由 95 名接受柔红霉素和阿糖胞苷治疗的急性髓系白血病患者组成的验证队列中证实了离体化疗反应的预测价值。体外药物反应概况改善了具有不良风险的个体的 ELN-22 风险分层。我们的结论是，离体药物反应分析在临床上是可行的，并且有可能预测患有超出临床确定的遗传标记的血液恶性肿瘤个体的化疗反应。© 2023。作者。

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.© 2023. The Author(s).