一项评估咖啡因用于治疗缺氧缺血性脑病婴儿的安全性的 I 期临床试验。
A phase I trial of caffeine to evaluate safety in infants with hypoxic-ischemic encephalopathy.
机构信息
Department of Pediatrics, School of Medicine, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
Division of Pharmacotherapy and Experimental Therapeutics, UNC Eshelman School of Pharmacy, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
出版信息
J Perinatol. 2024 Apr;44(4):508-512. doi: 10.1038/s41372-023-01752-y. Epub 2023 Aug 16.
OBJECTIVE
Caffeine provides neuroprotection following hypoxic-ischemic injury in animals. We characterized the safety of escalating doses of caffeine in infants with hypoxic-ischemic encephalopathy (HIE) receiving therapeutic hypothermia.
STUDY DESIGN
Phase I trial of infants undergoing therapeutic hypothermia for HIE receiving IV caffeine 20 mg/kg followed by up to two daily doses of 5 mg/kg (n = 9) or 10 mg/kg (n = 8). Safety was evaluated based on adverse events and frequency of pre-specified outcomes compared to data from the Whole-Body Hypothermia for HIE trial (Shankaran, 2005).
RESULTS
Twelve of 17 (71%) infants had ≥1 adverse event during the study period. The frequency of clinical outcomes related to HIE were not statistically different from outcomes in infants receiving hypothermia in the Whole-Body Hypothermia for HIE trial.
CONCLUSION
Caffeine administration was well tolerated. A larger study is required to determine the optimal dose and evaluate drug safety and efficacy.
CLINICAL TRIAL
ClinicalTrials.gov Identifier: NCT03913221.
目的
咖啡因可在动物缺氧缺血性损伤后提供神经保护。我们对接受治疗性低温的缺氧缺血性脑病（HIE）婴儿进行递增剂量的咖啡因治疗的安全性进行了评估。
研究设计
在接受 HIE 治疗性低温治疗的婴儿中进行的一项 I 期试验，给予静脉内 20mg/kg 的咖啡因，随后每日给予 5mg/kg（n=9）或 10mg/kg（n=8）的最多两个剂量。安全性评估基于不良事件以及与全身低温治疗 HIE 试验（Shankaran，2005 年）数据相比的特定结果的频率。
结果
17 名婴儿中有 12 名（71%）在研究期间出现≥1 次不良事件。与接受全身低温治疗的 HIE 试验中的婴儿相比，与 HIE 相关的临床结局的发生频率没有统计学差异。
结论
咖啡因给药耐受性良好。需要进行更大的研究来确定最佳剂量并评估药物的安全性和疗效。
临床试验
ClinicalTrials.gov 标识符：NCT03913221。
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