Pediatric Pharmacogenomics and Personalized Medicine

儿科药物基因组学和个性化医疗

• 批准号: 8532008
• 负责人: JAMES STEVEN LEEDER
• 金额: $ 0.6万
• 依托单位: CHILDREN'S MERCY HOSP (KANSAS CITY, MO)
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-04-01 至 2014-06-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8532008
• 关键词: Acute Lymphocytic Leukemia; Address; Adolescence; Adolescent; Adult; Age; Ally; Applied Research; Area; Birth; Buspirone; Child; Childhood; Cities; Clinical; Clinical Pharmacology; Clinical Trials; Clinics and Hospitals; Collaborations; Development; Developmental Process; Diagnostic; Disease; Dose; Drug Exposure; Drug Targeting; Enzymes; Ethics; Frequencies; Genetic Variation; Genomics; Goals; Growth; Healthcare; Human Biology; Individual; Infant; Investigation; Kansas; Knowledge; Laws; Lead; Legal; Measures; Medical; Medicine; Newborn Infant; Outcome; Patients; Pattern; Pediatrics; Pharmaceutical Preparations; Pharmacogenetics; Pharmacogenomics; Pharmacologic Substance; Pharmacotherapy; Reaction; Regulation; Relative (related person); Research; Research Personnel; Research Priority; Risk; Safety; Science; Scientist; Series; Signal Transduction Pathway; Staging; Therapeutic; Toxic effect; U-Series Cooperative Agreements; Uncertainty; age related; base; clinical care; disorder prevention; dissemination research; drug efficacy; experience; improved; innovation; meetings; programs; response; symposium

DESCRIPTION (provided by applicant): A major challenge for clinicians, pharmaceutical companies and regulatory agencies is to better understand the relative contributions of ontogeny and genetic variation to observed variability in drug disposition and response across the pediatric age spectrum from pre-term and term newborns, to infants, children and adolescents. Knowledge of the contribution of genetic variation to variability in drug disposition and response in adults has increased substantially over the past 25 years, but the application of pharmacogenetic and pharmacogenomic principles to pediatric drug therapy has lagged well behind. Extrapolation of adult experience with pharmacogenomics and personalized medicine to pediatric patients of different ages and developmental stages is fraught with many challenges. Some pediatric diseases have no adult correlate or are more prevalent in children compared to adults. Likewise, several adverse drug reactions are unique to children or occur at a higher frequency in children. Compared to adults, pediatric pharmacogenetics and pharmacogenomics involves an added measure of complexity as variability due to developmental processes,or ontogeny, is superimposed upon genetic variation. In the context of drug disposition and response, the additional complexity that ontogeny contributes manifests as unanticipated differences in dosing requirements, drug efficacy or risk of toxicity relative to what is expected based on experience in adults. The primary objective of this proposal is to continue an ongoing series of annual conferences on "Pediatric Pharmacogenomics and Personalized Medicine" organized by the Center for Personalized Medicine and Therapeutic Innovation and Division of Clinical Pharmacology and Medical Therapeutics at Children's Mercy Hospitals and Clinics in Kansas City, MO. The goals of the meeting are 1) To bring together clinicians, basic and translational scientists and allied healthcare practitioners, and engage in multi- and cross-disciplinary dialogue aimed at implementing personalized medicine in pediatric settings; 2) To provide a forum for the presentation and dissemination of research related to the application of pharmacogenomic strategies to investigations of variability of drug disposition and response in children; 3) To explore the ethical, legal and societal implications of pharmacogenomics and personalized medicine that are unique to children; and 4) To create networking opportunities for stimulating discussion, cooperation and collaboration to devise strategies to address research needs identified. Ultimately, the goal of this program is to improve the safety and efficacy of medications in children of all ages.

描述（由申请人提供）：对临床医生，制药公司和监管机构的一个主要挑战是更好地了解本体发育和遗传变异对观察到的对药物处于药物处置的可变性和对新生儿的儿童年龄频谱的可变性的相对贡献，对婴儿，儿童和青少年，对新生儿和年龄的差异。在过去25年中，遗传变异对成人药物处置和反应变异性的贡献的知识大大增加，但是药物遗传学和药物基因组原理在小儿药物治疗中的应用已落后。对不同年龄和发育阶段的儿科患者进行药物基因组学和个性化医学经验的推断经验充满了许多挑战。与成年人相比，某些儿科疾病在儿童中没有成人相关或更普遍。同样，几种不良药物反应是儿童独有的，或者在儿童中发生较高的频率。与成人相比，小儿药物遗传学和药物基因组学涉及复杂性的附加度量，因为由于发育过程或个体发育而导致的变异性叠加在遗传变异上。在药物处置和反应的背景下，个体发育的额外复杂性表现为剂量需求，药物疗效或毒性风险的意外差异，相对于基于成人经验的预期而言。该提案的主要目的是继续在密苏里州堪萨斯州堪萨斯城的儿童Mercy's Mercy Hospitals and Clinics和Mercy's Mercy Hospitals and Clinics in Chirpenal Pharmagication和Medical Therapeics中，继续进行一系列关于“小儿药物基因组学和个性化医学”的年度会议。会议的目标是1）将临床医生，基础和转化科学家和盟友医疗保健从业人员汇集在一起​​，并进行多个旨在在儿科环境中实施个性化医学的多学科对话； 2）提供一个论坛，以介绍和传播与药物分配策略在儿童药物处置和反应变异性研究中的应用有关的研究； 3）探索儿童独有的药物基因组学和个性化医学的道德，法律和社会意义； 4）为刺激讨论，合作与协作创造网络机会，以制定策略来满足所确定的研究需求。最终，该计划的目的是提高各个年龄段儿童药物的安全性和功效。