MD NET: Pediatric

美国国立卫生研究院(NCI)癌症治疗与诊断部(DCTD)在其全国临床试验网络(NCTN)中启动了一项战略倡议，以定义和发现急性髓系白血病/骨髓增生异常综合征(AML/MDS)患者的新疗法。正如医学研究所最近的一份报告所描述的那样，过去40年来，对这些患者的治疗没有实质性的改变，这表明在这一领域亟需取得进展。 这项计划的主要目标是优化成人、年轻人和青少年急性髓系白血病和骨髓增生异常综合征(AML/MDS)患者的治疗策略。 这包括： ·一种主要的筛查方案，评估分配给临床试验的患者。 ·数据支持结构，允许纵向分析白血病的敏感性和耐药性生物标记物，以发现新的分子靶点 ·破译微小残留病中的克隆选择 ·了解抵抗机制和对治疗的反应 这项研究的长期目标是： ·确定有可能优化AML/MDS治疗选择的生物标记物。 ·定义预测AML/MDS中不同反应类别的分子签名。 ·确定急性髓系白血病/多药耐药反应/耐药的表型/基因型特征，这些特征将对具有不同作用机制的不同或特定类别的药物有效。 ·确定微小残留病(MRD)的目标，包括复发的目标，并确定根除MRD的新方法。

The Division of Cancer Treatment and Diagnosis, NCI (DCTD) has embarked on a strategic initiative in its National Clinical Trials Network (NCTN) to define and discover new treatments for patients with Acute Myelogenous Leukemia/Myelodysplastic Syndrome (AML/MDS). As described in a recent report from the Institute of Medicine ‘treatment for these patients has not changed in a substantive way in the last 40 years’ demonstrating that advances in this area are critically needed. The primary goal of this initiative is to optimize therapeutic strategies for adult, young adult, and adolescent patients with Acute Myeloid Leukemia and Myelodysplastic Syndromes (AML/MDS). This includes: • A master screening protocol that evaluates patients for assignment to clinical trials. • A data support structure that allows longitudinal analysis of sensitivity and resistance biomarkers in leukemia for discovery of new molecular targets • Deciphering clonal selection in minimal residual disease • Understanding mechanisms of resistance and response to therapy The long-term goals of the study are to: • Identify biomarkers with the potential to optimize therapy selection in AML/MDS. • Define molecular signatures that predict different response categories in AML/MDS. • Define phenotype/genotype characteristics of response/resistance in AML/MDS that will be valid for diverse or specific classes of drugs with different mechanisms of action. • To identify targets in minimal residual disease (MRD), including targets in relapse and to identify new ways to eradicate MRD.