The Children's Hospital Heart Institute, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, Colorado, USA.
Curr Opin Cardiol. 2012 Mar;27(2):70-81. doi: 10.1097/HCO.0b013e32835018cd.
Pulmonary arterial hypertension (PAH) is an important cause of morbidity and mortality in children. Approved medications for the treatment of adult PAH have been used to treat children, but evidence-based treatment algorithms for children are lacking.
Pediatric PAH registries have begun to define the incidence and prevalence of idiopathic PAH and PAH associated with congenital heart disease. A pediatric-specific classification of pulmonary hypertensive vascular disease has been proposed. Furthermore, the first randomized placebo-controlled trial of type-5 phosphodiesterase therapy in treatment-naïve children with PAH has been completed and reported. This trial highlights the importance of the difficulties of performing clinical trials in children with targeted PAH therapy as well as the importance of long-term follow-up of adverse events.
Classification, clinical trials, and therapy for children with PAH must take into account the unique aspects of PAH in children.
肺动脉高压（PAH）是儿童发病率和死亡率的重要原因。已批准用于治疗成人 PAH 的药物已被用于治疗儿童，但缺乏针对儿童的循证治疗方案。
儿科 PAH 登记处开始定义特发性 PAH 和与先天性心脏病相关的 PAH 的发病率和患病率。已经提出了一种儿科特有的肺动脉高压血管疾病分类。此外，已经完成并报告了首个针对治疗初治儿童 PAH 的 5 型磷酸二酯酶治疗的随机安慰剂对照试验。该试验强调了在靶向 PAH 治疗中对儿童进行临床试验的困难以及对不良事件进行长期随访的重要性。
PAH 患儿的分类、临床试验和治疗必须考虑到 PAH 在儿童中的独特方面。
