MD NET: Pediatric

MD NET：儿科

• 批准号: 10048432
• 负责人: RALPH PARCHMENT
• 金额: $ 475.51万
• 依托单位: LEIDOS BIOMEDICAL RESEARCH, INC.
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-30 至 2024-09-29
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10048432
• 关键词: Acute Myelocytic Leukemia; Adult; Area; Authorization documentation; Biological Assay; Biological Markers; Categories; Characteristics; Childhood; Clinical; Clinical Data; Clinical Protocols; Clinical Trials; Contracts; Data; Data Files; Diagnostic; Division of Cancer Treatment and Diagnosis; Dysmyelopoietic Syndromes; Eligibility Determination; Enrollment; Future; Generations; Genotype; Goals; Immune; Institute of Medicine (U.S.); Laboratories; Molecular Profiling; Molecular Target; National Clinical Trials Network; Oncogenic; Pathway interactions; Patients; Pharmaceutical Preparations; Phenotype; Physicians; Protocols documentation; Publications; Regulation; Relapse; Reporting; Reproducibility; Residual Neoplasm; Residual state; Resistance; Selection for Treatments; Site; Specimen; Structure; Testing; Therapeutic; Vendor; Work; adolescent patient; base; biobank; data access; data warehouse; leukemia; longitudinal analysis; predictive signature; profiles in patients; resistance mechanism; response; sample collection; treatment optimization; young adult

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.

NCI 癌症治疗和诊断部门 (DCTD) 已在其国家临床试验网络 (NCTN) 中启动一项战略举措，为急性髓性白血病/骨髓增生异常综合征 (AML/MDS) 患者定义和发现新的治疗方法。医学研究所最近的一份报告“在过去 40 年里，对这些患者的治疗没有发生实质性改变”，表明迫切需要在这一领域取得进展。 该计划的主要目标是优化成人、青年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： • 评估患者分配临床试验的主筛选方案。 • 数据支持结构，允许对白血病的敏感性和耐药性生物标志物进行纵向分析，以发现新的分子靶标 • 解读微小残留病中的克隆选择 • 了解治疗的耐药机制和反应 该研究的长期目标是： • 识别具有优化AML/MDS 治疗选择潜力的生物标志物。 • 定义预测AML/MDS 中不同反应类别的分子特征。 • 定义AML/MDS 中反应/耐药的表型/基因型特征，这对于具有不同作用机制的多种或特定类别的药物有效。 • 确定残留微小病变(MRD) 的目标，包括复发的目标，并确定根除MRD 的新方法。