Das Bibhuti B, Moskowitz William B, Butler Javed
Heart Center, Department of Pediatrics, Mississippi Children's Hospital, University of Mississippi Medical Center, Jackson, MS 39212, USA.
Department of Medicine, University of Mississippi Medical Center, Jackson, MS 39212, USA.
Children (Basel). 2021 Apr 22;8(5):322. doi: 10.3390/children8050322.
This review discusses the potential drug and device therapies for pediatric heart failure (HF) due to reduced systolic function. It is important to realize that most drugs that are used in pediatric HF are extrapolated from adult cardiology practices or consensus guidelines based on expert opinion rather than on evidence from controlled clinical trials. It is difficult to conclude whether the drugs that are well established in adult HF trials are also beneficial for children because of tremendous heterogeneity in the mechanism of HF in children and variations in the pharmacokinetics and pharmacodynamics of drugs from birth to adolescence. The lessons learned from adult trials can guide pediatric cardiologists to design clinical trials of the newer drugs that are in the pipeline to study their efficacy and safety in children with HF. This papers focus is that the reader should specifically think through the pathophysiological mechanism of HF and the mode of action of drugs for the selection of appropriate pharmacotherapy. We review the drug and device trials in adults with HF to highlight the knowledge gap that exists in the pediatric HF population.
本综述讨论了因收缩功能降低所致小儿心力衰竭(HF)的潜在药物和器械治疗方法。必须认识到,小儿HF所使用的大多数药物是从成人心脏病学实践或基于专家意见的共识指南推断而来,而非来自对照临床试验的证据。由于小儿HF机制存在巨大异质性,且从出生到青春期药物的药代动力学和药效学存在差异,因此很难得出在成人HF试验中已确立的药物对儿童是否也有益的结论。从成人试验中吸取的经验教训可指导小儿心脏病专家设计正在研发的新药的临床试验,以研究其在小儿HF患者中的疗效和安全性。本文的重点是,读者应具体思考HF的病理生理机制以及药物的作用方式,以便选择合适的药物治疗。我们回顾了成人HF的药物和器械试验,以突出小儿HF人群中存在的知识空白。