Clinical features of paediatric pulmonary hypertension: a registry study.
Clinical features of paediatric pulmonary hypertension: a registry study.
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DOI：
10.1016/s0140-6736(11)61621-8
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发表时间：
2012-02-11
期刊：
影响因子：
168.9
通讯作者：
Barst, Robyn J.
中科院分区：
文献类型：
--
作者：
Berger, Rolf M. F.;Beghetti, Maurice;Humpl, Tilman;Raskob, Gary E.;Ivy, D. Dunbar;Jing, Zhi-Cheng;Bonnet, Damien;Schulze-Neick, Ingram;Barst, Robyn J.
儿童肺动脉高压是发病率和死亡率的重要原因，在儿童中的特征不充分。儿童肺动脉高压（TOPP）登记研究的追踪结果和实践是一项全球性前瞻性研究，旨在提供有关儿童肺动脉高压的人口统计学、治疗和结局的信息。从2008年1月31日至2010年2月15日，在19个国家的31个中心招募了诊断为肺动脉高压和肺血管阻力增加的18岁或以下的连续性患者。记录患者和疾病特征，包括诊断和入组时的年龄、性别、种族、临床症状、肺动脉高压分类、共病疾病、病史和家族史、血液动力学指标和功能分级。随访由患者的医生根据个人的保健需要决定。456例连续患者中有362例确诊为肺动脉高压（定义为平均肺动脉压≥25 mm Hg，肺毛细血管楔压≤12 mm Hg，肺血管阻力指数≥3 WU/m32）。317例（88%）患者患有肺动脉高压（PAH），其中182例（57%）为特发性[IPAH]或家族性[FPAH]，135例（43%）与其他疾病相关，其中115例（85%）与先天性心脏病相关。42例患者（12%）患有与呼吸系统疾病或低氧血症相关的肺动脉高压，其中最常见的是支气管肺发育不良。最后，只有3名患者患有慢性血栓栓塞性肺动脉高压或其他原因引起的肺动脉高压。47例（13%）确诊患者报告了染色体异常，主要是21三体。诊断时的中位年龄为7岁（IQR 3-12）; 59%（268/456）为女性。虽然呼吸困难和疲劳是最常见的症状，但晕厥发生在31%（57/182）的IPAH或FPAH患者和18%（8/45）的先天性心脏病修复患者中;未修复的先天性体肺分流的儿童没有晕厥。尽管有严重的肺动脉高压，但362例患者中有230例（64%）的功能分级为I或II级，这与保留的右心功能一致。TOPP确定了儿科肺动脉高压护理的重要临床特征，这引起了对儿科数据的关注，而不是从成人研究中推断。爱可泰隆制药
Paediatric pulmonary hypertension, is an important cause of morbidity and mortality, and is insufficiently characterised in children. The Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension (TOPP) registry is a global, prospective study designed to provide information about demographics, treatment, and outcomes in paediatric pulmonary hypertension. Consecutive patients aged 18 years or younger at diagnosis with pulmonary hypertension and increased pulmonary vascular resistance were enrolled in TOPP at 31 centres in 19 countries from Jan 31, 2008, to Feb 15, 2010. Patient and disease characteristics, including age at diagnosis and at enrolment, sex, ethnicity, presenting symptoms, pulmonary hypertension classification, comorbid disorders, medical and family history, haemodynamic indices, and functional class were recorded. Follow-up was decided by the patients’ physicians according to the individual’s health-care needs. 362 of 456 consecutive patients had confirmed pulmonary hypertension (defined as mean pulmonary artery pressure ≥25 mm Hg, pulmonary capillary wedge pressure ≤12 mm Hg, and pulmonary vascular resistance index ≥3 WU/m32). 317 (88%) patients had pulmonary arterial hypertension (PAH), which was idiopathic [IPAH] or familial [FPAH] in 182 (57%), and associated with other disorders in 135 (43%), of which 115 (85%) cases were associated with congenital heart disease. 42 patients (12%) had pulmonary hypertension associated with respiratory disease or hypoxaemia, with bronchopulmonary dysplasia most frequent. Finally, only three patients had either chronic thromboembolic pulmonary hypertension or miscellaneous causes of pulmonary hypertension. Chromosomal anomalies, mainly trisomy 21, were reported in 47 (13%) of patients with confirmed disease. Median age at diagnosis was 7 years (IQR 3–12); 59% (268 of 456) were female. Although dyspnoea and fatigue were the most frequent symptoms, syncope occurred in 31% (57 of 182) of patients with IPAH or FPAH and in 18% (eight of 45) of those with repaired congenital heart disease; no children with unrepaired congenital systemic-to-pulmonary shunts had syncope. Despite severe pulmonary hypertension, functional class was I or II in 230 of 362 (64%) patients, which is consistent with preserved right-heart function. TOPP identifies important clinical features specific to the care of paediatric pulmonary hypertension, which draw attention to the need for paediatric data rather than extrapolation from adult studies. Actelion Pharmaceuticals.