Douwes Johannes M, Berger Rolf M F
Department of Pediatric Cardiology, Center for Congenital Heart Diseases, Beatrix Children's Hospital, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.
Curr Opin Pulm Med. 2017 Sep;23(5):398-403. doi: 10.1097/MCP.0000000000000406.
Current recommendations for diagnosis and treatment of pulmonary arterial hypertension (PAH) during childhood are expert opinion based, because of lacking pediatric data. In recent years, however, important pediatric data have emerged on PAH.
PAH in children shows similarities as well as differences compared to adults. Neonates and children know specific clinical presentations and a hemodynamic profile that differs from adults with PAH. Children identified as acute vasodilator responders according to the criteria proposed for adults rather than the pediatric criteria have better outcome when treated with calcium channel blockers. For nonresponders, combination PAH-targeted therapy leads to improved outcome compared to monotherapy. In pediatric PAH, WHO functional class, N-terminal pro-brain natriuretic peptide and tricuspid annular plane systolic excursion were identified as surrogates for survival and therefore qualify to be treatment goals in a goal-oriented treatment strategy.
In order to refine current pediatric treatment guidelines, data on efficacy of specific treatment regiments and strategies are needed. The recently validated composite endpoint of clinical worsening allows for trials that will provide these data. For the first time, evidence-based treatment goals have been identified that will allow for a goal-oriented treatment strategy. Furthermore, various prognostic predictors have been identified that may prove treatment goals in future.
由于缺乏儿科数据，目前关于儿童肺动脉高压（PAH）诊断和治疗的建议基于专家意见。然而，近年来出现了关于PAH的重要儿科数据。
儿童PAH与成人相比既有相似之处也有不同之处。新生儿和儿童有特定的临床表现以及与成人PAH不同的血流动力学特征。根据成人而非儿科标准被确定为急性血管扩张剂反应者的儿童，在接受钙通道阻滞剂治疗时预后更好。对于无反应者，与单一疗法相比，联合PAH靶向治疗可改善预后。在儿科PAH中，世界卫生组织功能分级、N末端脑钠肽前体和三尖瓣环平面收缩期位移被确定为生存替代指标，因此有资格成为以目标为导向的治疗策略中的治疗目标。
为了完善当前的儿科治疗指南，需要有关特定治疗方案和策略疗效的数据。最近验证的临床恶化综合终点使得能够开展提供这些数据的试验。首次确定了基于证据的治疗目标，这将有助于实施以目标为导向的治疗策略。此外，还确定了各种预后预测因素，未来可能会证明其为治疗目标。
