Outcome of Pediatric Patients With Pulmonary Arterial Hypertension in the Era of New Medical Therapies
Outcome of Pediatric Patients With Pulmonary Arterial Hypertension in the Era of New Medical Therapies
复制标题
DOI：
10.1016/j.amjcard.2010.02.023
复制
发表时间：
2010-07-01
影响因子：
2.8
通讯作者：
Berger, Rolf M. F.
中科院分区：
文献类型：
--
作者：
van Loon, Rosa Laura E.;Roofthooft, Marcus T. R.;Berger, Rolf M. F.
关于“第二代药物”（前列腺素类、内皮素受体拮抗剂、5-磷酸二酯酶抑制剂）在儿童肺动脉高压（PAH）中的作用知之甚少。本研究描述了在这些药物可用的时代，PAH儿童的全国队列的结果。从1993年至2008年，连续52例特发性PAH（n = 29）或体肺分流相关PAH（n = 23）儿童接受了基线和随访评估。根据功能分级、急性肺血管反应性反应和药物可用性开始治疗。根据诊断时间与第二代药物可用性的关系评价观察到的生存率，随后与计算的预测生存率进行比较。第二代药物治疗的儿童的生存率比预测的生存率有所提高（1年、3年和5年生存率分别为93%、83%和66% vs 79%、61%和50%）。然而，这种生存率的改善仅在病程期间使用第二代药物的患者中观察到。在诊断时已经有药物可用的患者中，没有观察到生存率的改善。与生存率降低相关的基线变量包括较高的功能分级、较高的肺动脉与体动脉压比值、较低的心脏指数和较高的血清N末端脑钠肽前体和尿酸水平。开始使用第二代药物后，功能分级、6分钟步行距离和N末端脑钠肽前体改善，但在较长时间随访后逐渐下降。总之，自引入第二代药物以来，儿童PAH的生存率似乎仅在选定的患者中得到改善，这些患者在其病程中可获得这些药物。第二代药物的开始最初引起临床改善，但这些影响在较长的随访后下降。(C)2010年爱思唯尔公司All rights reserved. (Am J Cardiol 2010;106：117-124）
Little is known about the effects of "second-generation drugs" (prostanoids, endothelin receptor antagonists, 5-phosphodiesterase inhibitors) in children with pulmonary arterial hypertension (PAH). This study describes the outcome of a national cohort of children with PAH in an era when these drugs became available. From 1993 to 2008, 52 consecutive children with idiopathic PAH (n = 29) or systemic-to-pulmonary shunt-associated PAH (n = 23) underwent baseline and follow-up assessments. Treatment was initiated depending on functional class, acute pulmonary vasoreactivity response, and drug availability. Observed survival was evaluated depending on time of diagnosis in relation to second-generation drug availability and subsequently compared to calculated predicted survival. Children for whom second-generation drugs were available had improved survival compared to their predicted survival (1-, 3-, and 5-year survival rates 93%, 83%, and 66% vs 79%, 61%, and 50%, respectively). However, this improved survival was observed only in patients for whom second-generation drugs became available during their disease course. No improved survival was observed in patients for whom drugs were available already at diagnosis. Baseline variables associated with decreased survival included higher functional class, higher pulmonary-to-systemic arterial pressure ratio, lower cardiac index, and higher serum levels of N-terminal pro brain natriuretic peptide and uric acid. After start of second-generation drugs, functional class, 6-minute walking distance, and N-terminal pro brain natriuretic peptide improved but gradually decreased after longer follow-up. In conclusion, survival of pediatric PAH seemed improved since the introduction of second-generation drugs only in selected patients for whom these drugs became available during their disease course. Start of second-generation drugs initially induced clinical improvements, but these effects decreased after longer follow-up. (C) 2010 Elsevier Inc. All rights reserved. (Am J Cardiol 2010;106:117-124)