针对患有慢性免疫血小板缺血症 (PETIT2) 的儿童的Eltrombopag:一个随机,多中心,安慰剂对照试验
John D Grainger1, Franco Locatelli2, Thirachit Chotsampancharoen3
1Department of Haematology, Royal Manchester Children's Hospital, University of Manchester, Manchester, UK; Central Manchester Hospitals NHS Foundation Trust, NIHR/Wellcome Trust Manchester CRF, Royal Manchester Children's Hospital, University of Manchester, Manchester, UK.
Lancet (London, England)
|August 2, 2015
概括
在患有慢性免疫血小板缺血的儿童中,Eltrombopag有效地增加了血小板数量,这表明它是一种安全且合适的治疗选择. 该研究没有发现新的安全问题,由于不良事件而停止治疗的情况很少.
科学领域:
- 儿童血液学
- 药理学
- 临床试验
背景情况:
- 慢性免疫血小板减少 (ITP) 影响儿童的血小板数量.
- 埃尔特罗姆波巴 (Eltrombopag) 是一种血栓形成素受体激活剂,已被批准用于成人ITP治疗.
- 儿童ITP治疗的疗效和安全性需要进一步研究.
研究的目的:
- 评估eltrombopag在患有慢性ITP的儿童中的安全性和有效性.
- 评估eltrombopag在儿童患者中达到并维持适当血小板数量的能力.
主要方法:
- 这是一项随机,双盲,安慰剂控制的多中心研究 (PETIT2),涉及92名儿科患者 (1至17岁).
- 患者接受了13周的eltrombopag或安慰剂治疗,随后是开放的eltrombopag治疗期.
- 主要结局:在第5至第12周之间,患者的血小板数量达到≥50×10^9/ L,持续6周.
主要成果:
- 40% 的eltrombopag受试者达到主要结果,而安慰剂受试者只有 3% (OR 18. 0, p=0. 0004).
- 血小板反应在不同年龄段 (12-17岁,6-11岁,1-5岁) 一致.
- 在eltrombopag组的出血率较低;没有死亡,恶性瘤或血栓发生.
结论:
- 对于导致持续血小板反应的慢性症状ITP的儿童,Eltrombopag是一种合适的治疗选择.
- 该药在儿科患者中表现出良好的安全性,没有发现新的安全问题.
相关概念视频
01:23Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption
762
Understanding the physiological differences in the pediatric population is crucial for effective pharmacotherapy. Neonates, infants, and children exhibit significant variations in gastric pH, gastric emptying time, intestinal transit time, and biliary function. These variations profoundly affect oral drug absorption, necessitating a nuanced approach to pediatric dosing.Neonates present with a unique physiological profile, having a gastric pH greater than 4 and faster and more irregular gastric...
762
01:24Pharmacokinetics in Pediatric Patients: Drug Metabolism
341
In pediatric care, understanding the nuances of hepatic drug metabolism is crucial, as it significantly differs from that of adults. This divergence is primarily due to the developmental stage of drug-metabolizing enzymes, which affects how medications are processed in the body. In neonates, for instance, the activity of Phase I enzymes—critical for the initial breakdown of drugs—is markedly reduced, functioning at just 20–40% of the levels seen in adults. This reduction poses...
341
01:17Pharmacokinetics in Pediatric Patients: Drug Distribution
484
Drug distribution in the pediatric population exhibits unique challenges and considerations due to the physiological differences between children, particularly neonates and infants, and adults. A crucial aspect of pediatric pharmacology is understanding how these differences impact the pharmacokinetics of various drugs, necessitating age-specific dosing strategies to ensure efficacy and safety.Neonates and infants have a higher total body water content, ~75%–90% of their body weight,...
484
01:26Pharmacokinetics in Pediatric Patients: Drug Excretion
378
In pediatric medicine, understanding the renal function and drug elimination nuances is crucial for administering safe and effective treatments. Newborns, in particular, display markedly slower renal functions than adults, profoundly affecting how drugs are cleared from their bodies. This slower drug clearance requires clinicians to extend the dosing intervals for many medications to prevent drug accumulation and toxicity while ensuring therapeutic efficacy.One key area where these adjustments...
378
01:29Drug Dosing: Infants and Children
804
Pediatric patient dosages diverge from adults due to disparities in body surface area, total body water, and extracellular fluid per kilogram of body weight. The dosing regimen considers the variations in pharmacokinetics and pharmacology across distinct age groups, encompassing preterm newborns, infants, young children, older children, and adolescents. Calculation of pediatric patient doses is predicated on determining body surface area, which exhibits a superior correlation with the child's...
804
01:24Disorders of Hemostasis
2.8K
Hemostasis, the process that stops bleeding after a blood vessel injury, is crucial for maintaining the integrity of the circulatory system. However, disorders of hemostasis can disrupt this delicate balance, leading to either excessive clotting or bleeding. These disorders can be broadly classified into thromboembolic disorders and bleeding disorders.
Thromboembolic Disorders
Two factors primarily cause thromboembolic conditions.
Thromboembolic Disorders
Two factors primarily cause thromboembolic conditions.
2.8K