吸入的Treprostinil治疗儿童肺动脉高血压的有效性和安全性
- PMID:22917554
- PMCID:PMC3508003.
- DOI:10.1016 / j.amjcard.2012.07.037
吸入的Treprostinil治疗儿童肺动脉高血压的有效性和安全性
抽象的
前列腺素治疗的引入已经彻底改变了肺动脉高压(PAH)的治疗。然而,前列环素持续静脉输注存在重大风险和挑战,特别是对儿童。吸入曲前列尼已被证明是安全有效的成人。本研究描述了吸入treprostinil治疗PAH儿童的安全性和有效性。回顾性分析29例接受吸入性曲前列尼治疗≥6周的患儿。评估吸入treprostinil对运动能力、功能等级、超声心动图和血流动力学数据的影响。记录不良事件。患者每日4次,3 ~ 9次(6 μg/次)吸入曲前列尼。所有患者均接受肺动脉高压背景治疗;12例曾接受肠外前列腺素治疗。 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.
版权所有©2012 Elsevier Inc.保留所有权利。
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