Pediatric Heart Center, University Children's Hospital, Feulgenstrasse 12, D-35392 Giessen, Germany.
Department of Biomedical Engineering, University of Maastricht, Universiteitssingel 50, 6229 ER Maastricht, Netherlands.
Nat Rev Cardiol. 2015 Apr;12(4):244-54. doi: 10.1038/nrcardio.2015.6. Epub 2015 Feb 3.
Pulmonary arterial hypertension (PAH) is an important cause of morbidity and mortality in children. Improved understanding of the pathophysiological mechanisms of the underlying diseases has resulted in the development of effective, but not yet curative, therapies. Currently, drugs from three main pharmacological groups targeting specific aberrant pathways (endothelin, nitric oxide, and prostacyclin) and four routes of administration (inhaled, intravenous, oral, and subcutaneous) have been approved for adult patients with PAH. However, only a minority of these drugs has been officially approved for children, mainly because randomized controlled studies are limited by the small number of paediatric patients with PAH worldwide. In children with progressive, severe PAH and an inadequate response to drug therapy, advances in interventional and surgical approaches have provided promising new strategies to avoid right ventricular deterioration. These techniques can delay or even avoid the need for lung transplantation. In this Review, we present an update on developments in drug therapy for patients with PAH and highlight the current status of these treatments in children. We also describe the new surgical, interventional, and hybrid procedures, as well as their practical application in children with severe PAH.
肺动脉高压（PAH）是儿童发病率和死亡率的重要原因。对基础疾病病理生理机制的深入了解，促成了有效但尚未能根治的治疗方法的发展。目前，针对特定异常途径（内皮素、一氧化氮和前列环素）和四种给药途径（吸入、静脉、口服和皮下）的三种主要药理学药物已被批准用于成人 PAH 患者。然而，这些药物中只有少数被正式批准用于儿童，主要是因为随机对照研究受到全球 PAH 患儿数量较少的限制。对于进展性、严重 PAH 且对药物治疗反应不佳的儿童，介入和手术方法的进步为避免右心室恶化提供了有前途的新策略。这些技术可以延迟甚至避免肺移植的需要。在这篇综述中，我们介绍了 PAH 患者药物治疗的最新进展，并强调了这些治疗方法在儿童中的现状。我们还描述了新的手术、介入和混合程序，以及它们在严重 PAH 儿童中的实际应用。
