Effectiveness and Safety of Inhaled Treprostinil for the Treatment of Pulmonary Arterial Hypertension in Children
Effectiveness and Safety of Inhaled Treprostinil for the Treatment of Pulmonary Arterial Hypertension in Children
复制标题
DOI：
10.1016/j.amjcard.2012.07.037
复制
发表时间：
2012-12-01
影响因子：
2.8
通讯作者：
Rosenzweig, Erika B.
中科院分区：
文献类型：
--
作者：
Krishnan, Usha;Takatsuki, Shinichi;Rosenzweig, Erika B.
前列腺素疗法的引入彻底改变了肺动脉高压（PAH）的治疗。然而，持续静脉输注前列环素具有显著的风险和挑战，特别是在儿童中。吸入性曲前列替尼已被证明对成人安全有效。本研究描述了吸入曲前列汀治疗多环芳烃患儿的安全性和有效性。回顾性分析29例患儿吸入曲前列替尼治疗bb0 = 6周。评估吸入曲前列替尼对运动能力、功能分级、超声心动图和血流动力学数据的影响。记录了不良事件。患者接受3 ~ 9次呼吸（6 μ g/次呼吸），4次/天。所有患者均接受多环芳烃背景治疗；其中12人曾接受过前列腺素外注射。4例患者因咳嗽、支气管痉挛（n = 3）和PAH进展（n = 1）等症状停用吸入性曲前列替尼。轻微的副作用包括咳嗽（n = 9）和喉咙痛（n = 6），不需要停药。19年世界卫生组织功能等级有所提高，10年没有变化；运动能力随步行距离的增加而显著提高，随访时从455.7 +/- 71.5 m提高到498 +/- 70 m (p = 0.01)，峰值耗氧量从25.5 +/- 10.2增加到27.4 +/- 10 （p = 0.04）。综上所述，在儿童中加入背景靶向多环芳烃治疗时，吸入曲前列替尼与运动能力和世界卫生组织功能等级的改善有关，并且具有可接受的安全性。基于这些早期数据，进一步研究吸入曲前列地尼在小儿PAH患者中的应用似乎是有必要的。(c) 2012 Elsevier Inc.版权所有。[Am J Cardiol 2012;110:1704-1709]
The introduction of prostanoid therapy has revolutionized the treatment of pulmonary arterial hypertension (PAH). However, continuous intravenous prostacyclin infusion poses significant risks and challenges, particularly in children. Inhaled treprostinil has been shown to be safe and efficacious in adults. This study describes the safety and efficacy of inhaled treprostinil in children with PAH. A retrospective analysis of 29 children treated with inhaled treprostinil for >= 6 weeks was performed. Effects of inhaled treprostinil on exercise capacity, functional class, and echocardiographic and hemodynamic data were evaluated. Adverse events were documented. Patients received 3 to 9 breaths (6 mu g/breath) of inhaled treprostinil 4 times/day. All were receiving background PAH therapy; 12 had previously received parenteral prostanoid. Inhaled treprostinil was discontinued in 4 patients because of symptoms including cough and bronchospasm (n = 3) and progression of PAH (n = 1). Mild side effects including cough (n = 9) and sore throat (n = 6) did not require discontinuation of therapy. World Health Organization functional class improved in 19 and was unchanged in 10; exercise capacity significantly improved with the 6-minute walk distance, improving on follow-up from 455.7 +/- 71.5 to 498 +/- 70 m (p = 0.01) and peak oxygen consumption increasing from 25.5 +/- 10.2 to 27.4 +/- 10 (p = 0.04). In conclusion, inhaled treprostinil was associated with improvement in exercise capacity and World Health Organization functional class when added to background targeted PAH therapy in children and had an acceptable safety profile. Based on these early data, further study of inhaled treprostinil appears warranted in pediatric patients with PAH. (c) 2012 Elsevier Inc. All rights reserved. (Am J Cardiol 2012;110:1704-1709)