Professor of Pediatrics and Pediatric Cardiology, PhD in medical physiology, Former Head of Pediatrics and Pediatric Cardiology in Gazi University Faculty of Medicine. Founder of Pediatric Cardiology and PAH center in Gazi University. Former President of Turkish Association of Pediatric Cardiology and Surgery, Gazi University , Ankara, Turkiye.
Expert Opin Pharmacother. 2020 Jul;21(10):1253-1268. doi: 10.1080/14656566.2020.1757071. Epub 2020 May 13.
Pulmonary hypertension (PH) is a heterogeneous disease that mainly affects the pulmonary arterioles, leading to significant morbidity and mortality. Pulmonary hypertension in children from birth to adolescence presents important differences from that of adults. The majority of pediatric pulmonary arterial hypertension (PAH) cases are idiopathic or associated with congenital heart disease. However, the management of pediatric PAH mainly depends on the results of evidence-based adult studies and the clinical experiences of pediatric experts.
This article briefly reviews the recent updates on the definition, classification, and diagnostic evaluation of pediatric PAH and their impact on treatment strategies. The main purpose of this review is to discuss the current pediatric therapies, as well as the prospective therapies, in terms of therapeutic targets, actions, side effects, and dosages.
Although there is no cure for PAH, recent advances in the form of new treatment options have improved the quality of life and survival rates of PAH patients. PAH-targeted drugs and treatment strategies for adult PAH have not been sufficiently studied in children. However, the growing scientific activity in that field will surely change the treatment option recommendations in pediatric PH from experience-based to evidence-based in the near future.
肺动脉高压（PH）是一种异质性疾病，主要影响肺小动脉，导致发病率和死亡率显著增加。从出生到青春期的儿童肺动脉高压与成人有很大的不同。大多数儿科肺动脉高压（PAH）病例是特发性的或与先天性心脏病有关。然而，儿科 PAH 的治疗主要取决于基于证据的成人研究结果和儿科专家的临床经验。
本文简要回顾了儿科 PAH 的定义、分类和诊断评估的最新进展，以及它们对治疗策略的影响。本文的主要目的是讨论目前儿科治疗方法，以及针对治疗靶点、作用、副作用和剂量的潜在治疗方法。
虽然 PAH 无法治愈，但新的治疗选择形式的最新进展改善了 PAH 患者的生活质量和生存率。针对成人 PAH 的 PAH 靶向药物和治疗策略在儿童中研究不足。然而，该领域日益增加的科学活动肯定会在不久的将来将儿科 PH 的治疗选择建议从经验基础转变为基于证据。
