Kula Serdar, Canbeyli Fatma, Atasayan Vildan, Tunaoğlu Fatma Sedef, Oğuz Ayşe Deniz
Department of Pediatric Cardiology, Faculty of Medicine, Gazi University; Ankara-Turkey.
Anatol J Cardiol. 2018 Jul;20(1):41-47. doi: 10.14744/AnatolJCardiol.2018.78370.
The aim of this study was to evaluate children with pulmonary arterial hypertension (PAH) regarding epidemiological characteristics, clinical status with respect to the WHO functional class (WHO-FC), prognostic factors, and efficacy of medical treatment.
A retrospective evaluation of 41 patients with PAH was made in the Pediatric Cardiology Unit, Gazi University Medical Faculty, between February 2006 and October 2015.
Of the 41 patients included in this study, 51.2% were female. The median age was 60 months at first evaluation. The median follow-up was 60 months. At the start of the treatment, 43.9% patients were receiving combined drug therapy, and this rate increased to 60.9% by the last evaluation. The median time of adding a new medication to the therapy was 20 months. The 1- and 5-year survival rates were 94% and 86%, respectively. At the time of diagnosis, only pro-brain natriuretic peptide (proBNP) levels were associated with mortality (p=0.004), but at the last evaluation, 6-min walking test, proBNP and uric acid levels, and WHO-FC were also associated with survival (p=0.02, p=0.001, p=0.002, and p=0.05, respectively).
With current treatment choices in experienced centers, positive results are obtained with respect to the functional status and survival rates of patients with PAH. At the time of diagnosis, only proBNP had a prognostic value, whereas at the last evaluation, WHO-FC, 6-min walking test, proBNP, and uric acid were reported prognostic factors. For preventing rapid progression, determination of factors that have an effect on prognosis, in particular, is extremely important.
本研究旨在评估肺动脉高压（PAH）患儿的流行病学特征、世界卫生组织功能分级（WHO-FC）相关的临床状况、预后因素及药物治疗效果。
对2006年2月至2015年10月期间在加齐大学医学院儿科心脏病科就诊的41例PAH患者进行回顾性评估。
本研究纳入的41例患者中，51.2%为女性。首次评估时的中位年龄为60个月。中位随访时间为60个月。治疗开始时，43.9%的患者接受联合药物治疗，到最后一次评估时，这一比例增至60.9%。添加新药物至治疗方案的中位时间为20个月。1年和5年生存率分别为94%和86%。诊断时，仅前脑钠肽（proBNP）水平与死亡率相关（p=0.004），但在最后一次评估时，6分钟步行试验、proBNP和尿酸水平以及WHO-FC也与生存率相关（分别为p=0.02、p=0.001、p=0.002和p=0.05）。
在经验丰富的中心采用当前的治疗选择，PAH患者的功能状态和生存率取得了积极结果。诊断时，只有proBNP具有预后价值，而在最后一次评估时，WHO-FC、6分钟步行试验、proBNP和尿酸被报告为预后因素。特别是为防止疾病快速进展，确定影响预后的因素极为重要。
