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Embracing the challenges of neonatal and paediatric pulmonary hypertension
European Respiratory Journal ( IF 16.6 ) Pub Date : 2024-10-31
Ivy, D., Rosenzweig, E. B., Abman, S. H., Beghetti, M., Bonnet, D., Douwes, J. M., Manes, A., Berger, R. M. F.

Paediatric pulmonary arterial hypertension (PAH) shares common features with adult disease, but is associated with several additional disorders and challenges that require unique approaches. This article discusses recent advances, ongoing challenges and distinct approaches for caring for infants and children with PAH, as presented by the paediatric task force of the 7th World Symposium on Pulmonary Hypertension. We provide updates on diagnosing, classifying, risk-stratifying and treating paediatric pulmonary hypertension (PH) and identify critical knowledge gaps. An updated risk stratification tool and treatment algorithm is provided, now also including strategies for patients with associated cardiopulmonary conditions. Treatment of paediatric PH continues to be hindered by the lack of randomised controlled clinical trials. The challenging management of children failing targeted PAH therapy is discussed, including balloon atrial septostomy, lung transplantation and pulmonary-to-systemic shunt (Potts). A novel strategy using a multimodal approach for the management of PAH associated with congenital heart diseases with borderline pulmonary vascular resistance is included. Advances in diagnosing neonatal PH, especially signs and interpretation of PH by echocardiography, are highlighted. A team approach to the rapidly changing physiology of neonatal PH is emphasised. Challenges in drug approval are discussed, particularly the challenges of designing accurate paediatric clinical trials with age-appropriate end-points and adequate enrolment.



中文翻译:


迎接新生儿和儿童肺动脉高压的挑战



小儿肺动脉高压 (PAH) 与成人疾病具有共同特征,但与需要独特方法的几种其他疾病和挑战有关。本文讨论了第 7 届世界肺动脉高压研讨会儿科工作组提出的护理 PAH 婴儿和儿童的最新进展、持续挑战和不同方法。我们提供有关诊断、分类、风险分层和治疗儿科肺动脉高压 (PH) 的最新信息,并确定关键知识差距。提供了更新的风险分层工具和治疗流程,现在还包括针对相关心肺疾病患者的策略。由于缺乏随机对照临床试验,儿科 PH 的治疗继续受到阻碍。讨论了靶向 PAH 治疗失败儿童的挑战性管理,包括球囊房间隔造口术、肺移植和肺体分流术 (Potts)。包括一种使用多模式方法管理与先天性心脏病相关的 PAH 的新策略,伴临界性肺血管抵抗。本文重点介绍了诊断新生儿 PH 的进展,特别是超声心动图对 PH 的体征和解读。强调对新生儿 PH 快速变化的生理学采取团队方法。讨论了药物批准的挑战,特别是设计具有适合年龄的终点和足够入组的准确儿科临床试验的挑战。

更新日期:2024-10-31
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