Hollander Seth A, Ogawa Michelle T, Hopper Rachel K, Liu Esther, Chen Sharon, Rosenthal David N, Feinstein Jeffrey A
Department of Pediatrics (Cardiology), Stanford University, Palo Alto, California, USA.
Pediatr Transplant. 2020 Aug;24(5):e13742. doi: 10.1111/petr.13742. Epub 2020 May 19.
Treprostinil, a prostacyclin analog, is a safe and effective therapy for children with PAH; however, the use of this agent in children with mild PVR elevations related to HF, including those with SV congenital heart disease awaiting HT, is understudied. We describe the hemodynamic and symptomatic changes in pediatric patients awaiting HT treated with treprostinil.
Single-center retrospective review of all patients was listed for HT who received treprostinil during the listing period. Changes in hemodynamic and functional indices between the baseline catheterization (prior to drug initiation), and prior to HT, and patient outcomes were analyzed.
Among 16/17 (94%) who survived to HT, 8 (50%) were female, and 10 (63%) had SV physiology. The median age at drug initiation was 9 (IQR: 1, 14) years. The median duration of therapy prior to HT was 253 (IQR: 148, 504) days. Treprostinil significantly decreased PVR (3.8 vs 3.1 WU, P = .03), while mLA or mPCW pressure did not change (11 vs 13 mm Hg, P = .9). HF symptoms improved in 9/15 (60%) patients without VAD support prior to drug initiation, including 4/10 (40%) who did not receive a VAD any point while awaiting HT.
Treprostinil may be used safely in patients with mild PAH awaiting HT, including those with SV disease. PVR falls without substantial increases in mLA/mPCW pressure. HF symptoms improve in some patients.
曲前列尼尔是一种前列环素类似物，是治疗儿童肺动脉高压（PAH）的安全有效药物；然而，对于与心力衰竭（HF）相关的轻度肺血管阻力（PVR）升高的儿童，包括那些等待心脏移植（HT）的单心室（SV）先天性心脏病患儿，该药物的使用研究较少。我们描述了接受曲前列尼尔治疗的等待HT的儿科患者的血流动力学和症状变化。
对在等待期接受曲前列尼尔治疗且被列入HT名单的所有患者进行单中心回顾性研究。分析了基线导管插入术（药物开始前）、HT前血流动力学和功能指标的变化以及患者预后。
在16/17例（94%）存活至HT的患者中，8例（50%）为女性，10例（63%）具有SV生理特征。药物开始时的中位年龄为9岁（四分位间距：1，14）。HT前的中位治疗持续时间为253天（四分位间距：148，504）。曲前列尼尔显著降低了PVR（3.8 vs 3.1 Wood单位，P = 0.03），而平均左房（mLA）或平均肺毛细血管楔压（mPCW）未改变（11 vs 13 mmHg，P = 0.9）。在药物开始前无心室辅助装置（VAD）支持的9/15例（60%）患者中，HF症状得到改善，包括4/10例（40%）在等待HT期间未在任何时间接受VAD的患者。
曲前列尼尔可安全用于等待HT的轻度PAH患者，包括患有SV疾病的患者。PVR下降，而mLA/mPCW压力无大幅升高。部分患者的HF症状得到改善。
