优化早期试验中的运营效率：儿科试验网络的经验。
Optimizing operational efficiencies in early phase trials: The Pediatric Trials Network experience.
作者信息
England Amanda, Wade Kelly, Smith P Brian, Berezny Katherine, Laughon Matthew
机构信息
Division of Neonatal-Perinatal Medicine, The University of North Carolina at Chapel Hill, N.C. Memorial Hospital, 101 Manning Drive, CB#7596, Chapel Hill, NC 27599-7596, USA.
Division of Neonatology, Children's Hospital of Pennsylvania, CHOP Newborn Care at the Hospital of the University of Pennsylvania, 800 Spruce St., Philadelphia, PA 19107, USA.
出版信息
Contemp Clin Trials. 2016 Mar;47:376-82. doi: 10.1016/j.cct.2016.03.002. Epub 2016 Mar 9.
Abstract
Performing drug trials in pediatrics is challenging. In support of the Best Pharmaceuticals for Children Act, the Eunice Kennedy Shriver National Institute of Child Health and Human Development funded the formation of the Pediatric Trials Network (PTN) in 2010. Since its inception, the PTN has developed strategies to increase both efficiency and safety of pediatric drug trials. Through use of innovative techniques such as sparse and scavenged blood sampling as well as opportunistic study design, participation in trials has grown. The PTN has also strived to improve consistency of adverse event reporting in neonatal drug trials through the development of a standardized adverse event table. We review how the PTN is optimizing operational efficiencies in pediatric drug trials to increase the safety of drugs in children.
摘要
在儿科进行药物试验具有挑战性。为支持《儿童最佳药物法案》，尤妮斯·肯尼迪·施莱佛国家儿童健康与人类发展研究所于2010年资助成立了儿科试验网络（PTN）。自成立以来，PTN已制定策略以提高儿科药物试验的效率和安全性。通过使用稀疏和采集血样以及机会性研究设计等创新技术，参与试验的人数有所增加。PTN还努力通过制定标准化不良事件表来提高新生儿药物试验中不良事件报告的一致性。我们回顾了PTN如何优化儿科药物试验的运营效率以提高儿童用药的安全性。
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本文引用的文献
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Antimicrob Agents Chemother. 2014 May;58(5):2856-65. doi: 10.1128/AAC.02139-13. Epub 2014 Mar 10.
2
Off-label use of drugs in children.儿童药物的标签外使用。
Pediatrics. 2014 Mar;133(3):563-7. doi: 10.1542/peds.2013-4060. Epub 2014 Feb 24.
3
Population pharmacokinetics of intravenous acyclovir in preterm and term infants.静脉注射阿昔洛韦在早产儿和足月儿中的群体药代动力学。
Pediatr Infect Dis J. 2014 Jan;33(1):42-9. doi: 10.1097/01.inf.0000435509.75114.3d.
4
Determining population and developmental pharmacokinetics of metronidazole using plasma and dried blood spot samples from premature infants.使用早产儿的血浆和干血斑样本确定甲硝唑的群体药代动力学和发育药代动力学。
Pediatr Infect Dis J. 2013 Sep;32(9):956-61. doi: 10.1097/INF.0b013e3182947cf8.
5
Risk factors associated with unilateral hearing loss.与单侧听力损失相关的风险因素。
JAMA Otolaryngol Head Neck Surg. 2013 Jan;139(1):59-63. doi: 10.1001/jamaoto.2013.1097.
6
Prognostic value of biochemical liver parameters in neonatal sepsis-associated cholestasis.生化肝脏参数在新生儿败血症相关性胆汁淤积症中的预后价值。
J Paediatr Child Health. 2013 Jan;49(1):E6-11. doi: 10.1111/jpc.12053. Epub 2012 Dec 21.
7
Neonatal seizures and status epilepticus.新生儿惊厥和癫痫持续状态。
J Clin Neurophysiol. 2012 Oct;29(5):441-8. doi: 10.1097/WNP.0b013e31826bd90d.
8
Pharmacokinetics and safety of fluconazole in young infants supported with extracorporeal membrane oxygenation.氟康唑在体外膜肺氧合支持下的婴幼儿药代动力学和安全性。
Pediatr Infect Dis J. 2012 Oct;31(10):1042-7. doi: 10.1097/INF.0b013e31825d3091.
9
Pharmacokinetics and tolerability of single-dose daptomycin in young infants.单剂量达托霉素在婴幼儿中的药代动力学和耐受性。
Pediatr Infect Dis J. 2012 Sep;31(9):935-7. doi: 10.1097/INF.0b013e31825d2fa2.
10
Mortality and neonatal morbidity among infants 501 to 1500 grams from 2000 to 2009.2000 年至 2009 年间，体重 501 至 1500 克的婴儿的死亡率和新生儿发病率。
Pediatrics. 2012 Jun;129(6):1019-26. doi: 10.1542/peds.2011-3028. Epub 2012 May 21.
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