基胺在患有状细胞贫血的非常年幼儿童中:多中心,随机,受控试验 (BABY HUG)
Winfred C Wang1, Russell E Ware, Scott T Miller
1St Jude Children's Research Hospital, Memphis, TN 38105, USA. winfred.wang@stjude.org
Lancet (London, England)
|May 17, 2011
概括
在患有状细胞贫血的非常年幼儿童中,氧碳胺治疗显著减少了疼痛和牙炎. 虽然脏和功能的主要终点没有显示出显著差异,但该药物
科学领域:
- 儿科血液学 儿科血液学
- 药理学 药理学是指药理学的学科.
- 临床试验 临床试验
背景情况:
- 状细胞贫血会导致婴儿的显著发病率,包括器官功能障碍和急性并发症.
- 已知氧碳胺可减少成年人状细胞贫血的并发症.
- 这项研究研究了氧化碳胺在患有状细胞贫血的非常年幼儿童中的作用.
研究的目的:
- 评估基胺对患有状细胞贫血的婴儿器官功能障碍和临床并发症的影响.
- 检查实验室发现和氧化碳胺在这个年龄组的潜在毒性作用.
主要方法:
- 一项随机,双盲,安慰剂对照试验,涉及193名患有状细胞贫血 (HbSS或HbSβ) 的婴儿 (9-18个月).
- 参与者接受了液态氧化碳胺 (20 mg/kg/天) 或安慰剂两年.
- 主要终点包括脏和功能;次要终点包括临床事件,血液计数和安全性.
主要成果:
- 与安慰剂相比,氧化碳胺显著减少了疼痛事件 (p=0.002) 和指甲炎 (p<0.0001).
- 在主要终点上没有发现显著差异:脏功能 (p=0.21) 和膜过率 (p=0.84).
- 氧化碳胺增加了胎儿的血红蛋白,减少了白细胞数量,毒性有限 (轻度至中度的中性质疏松症).
结论:
- 基胺证明了在减少状细胞贫血的非常年幼儿童的疼痛事件和牙炎的安全性和有效性.
- 这些发现支持在所有被诊断患有状细胞贫血的非常年幼儿童中考虑氧化碳胺.
- 进一步的研究可能会探索这种儿科患者群体对器官功能的长期影响.
相关概念视频
Drug Dosing: Infants and Children
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...
Pharmacokinetics in Pediatric Patients: Drug Metabolism
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 a challenge in...
Pharmacokinetics in Pediatric Patients: Drug Excretion
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...
Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption
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...
Bioavailability Study Design: Healthy Subjects Versus Patients
Bioavailability studies are essential for evaluating a drug's therapeutic efficacy and understanding its absorption patterns under various physiological conditions. Conducting such studies on target patient populations provides more relevant data by simulating real-world disease states. However, practical challenges often necessitate the use of young, healthy adult volunteers as study subjects.Patients may exhibit altered drug absorption patterns due to the effects of the disease itself,...
Pharmacokinetics in Pediatric Patients: Drug Distribution
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, compared...

