Acute Vasodilator Response in Pediatric Pulmonary Arterial Hypertension Current Clinical Practice From the TOPP Registry
Acute Vasodilator Response in Pediatric Pulmonary Arterial Hypertension Current Clinical Practice From the TOPP Registry
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DOI：
10.1016/j.jacc.2016.01.015
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发表时间：
2016-03-22
影响因子：
24
通讯作者：
Berger, Rolf M. F.
中科院分区：
文献类型：
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作者：
Douwes, Johannes M.;Humpl, Tilman;Berger, Rolf M. F.
在肺动脉高压（PAH）中，急性血管扩张剂反应试验（AVT）被认为是识别使用钙通道阻滞剂（CCB）治疗预后良好的成年患者的重要指标。然而，在儿童多环芳烃中，用于识别急性反应者和CCB使用的标准尚未得到充分研究。目的：本研究旨在描述目前儿科PAH的AVT临床实践和随后的治疗决策。方法：2008年1月至2013年5月，529名确诊肺动脉高压的连续儿童在国际注册中心登记。我们分析了可评估的AVT患儿。结果：在382例可评估的AVT患儿中，212例为特发性/家族性PAH (IPAH/FPAH)， 105例为PAH合并先天性心脏病（PAH- chd）。70%的患者采用吸入一氧化氮进行AVT；其余患者使用其他药物。在IPAH/FPAH患者中，78例（37%）患者根据医生的诊断为急性应答，62例（30%）患者根据REVEAL（注册评估-早期和长期PAH疾病管理）-儿科标准，32例（15%）患者根据Sitbon标准。对于PAH-CHD患者，AVT应答者分别为38人（36%）、14人（13%）和7人（7%）。治疗医师判断的AVT应答状态与公布的应答标准之间的相关性很差。此外，在治疗医师判断为急性反应的IPAH/FPAH患者中，只有23%的患者接受了CCB治疗，而没有额外的pah靶向治疗。Sitbon标准选择预后较好且经CCB治疗后预后良好的患者。结论：目前识别AVT应答者和随后CCB治疗的做法与目前的国际指南存在很大差异。此外，在儿童IPAH中，Sitbon标准是确定接受CCB治疗后生存率高的患者的选择标准。(C) 2016年由美国心脏病学会基金会发布。
BACKGROUND In pulmonary arterial hypertension (PAH), acute vasodilator response testing (AVT) is considered important to identify adult patients with favorable prognosis using calcium-channel blocker (CCB) therapy. However, in pediatric PAH, criteria used to identify acute responders and CCB use are insufficiently studied.OBJECTIVES This study sought to describe current clinical practice of AVT and subsequent treatment decisions in pediatric PAH.METHODS From January 2008 to May 2013, 529 consecutive children with confirmed pulmonary hypertension were enrolled in an international registry. We analyzed those children with evaluable AVT.RESULTS Of 382 children with evaluable AVT, 212 had idiopathic/familial PAH (IPAH/FPAH) and 105 had PAH associated with congenital heart disease (PAH-CHD). In 70% of the patients, AVT was performed using inhaled nitric oxide; other agents were used in the remaining patients. In IPAH/FPAH patients, 78 (37%) patients were acute responders according to their physician, 62 (30%) according to REVEAL (Registry-to-Evaluate-Early-And-Long-term PAH disease management)-pediatric criteria, and 32 (15%) according to Sitbon criteria. For PAH-CHD patients, the numbers of AVT responders were 38 (36%), 14 (13%), and 7 (7%) respectively. Correlation between AVT responder status as judged by the treating physician and by published response criteria was poor. Moreover, of the IPAH/FPAH patients judged by the treating physician as acute responders, only 23% were treated with CCB without additional PAH-targeted therapy. The Sitbon criteria selected patients with better prognosis who had excellent outcome when treated with CCB.CONCLUSIONS The current practice of identifying responders to AVT and subsequent treatment with CCB therapy demonstrated large discrepancies with current international guidelines. Also, in pediatric IPAH, the Sitbon criteria are the criteria of choice to identify patients with excellent survival when treated with CCB therapy. (C) 2016 by the American College of Cardiology Foundation.