Rosenzweig Erika Berman, Ivy D Dunbar, Widlitz Allison, Doran Aimee, Claussen Lori R, Yung Delphine, Abman Steven H, Morganti Adele, Nguyen Ngoc, Barst Robyn J
Division of Pediatric Cardiology, New York Presbyterian Hospital, New York, New York 10032, USA.
J Am Coll Cardiol. 2005 Aug 16;46(4):697-704. doi: 10.1016/j.jacc.2005.01.066.
This study investigated the long-term outcome of children with pulmonary arterial hypertension (PAH) treated with bosentan therapy, with or without concomitant prostanoid therapy.
Bosentan, an oral endothelin ET(A)/ET(B) receptor antagonist, improves hemodynamics and exercise capacity in adults with PAH; however, limited data are available on its long-term effects in children.
In this retrospective study, 86 children with PAH (idiopathic, associated with congenital heart or connective tissue disease) started bosentan with or without concomitant intravenous epoprostenol or subcutaneous treprostinil therapy. Hemodynamics, World Health Organization (WHO) functional class, and safety data were collected.
At the cutoff date, 68 patients (79%) were still treated with bosentan, 13 (15%) were discontinued, and 5 (6%) had died. Median exposure to bosentan was 14 months. In 90% of the patients (n = 78), WHO functional class improved (46%) or was unchanged (44%) with bosentan treatment. Mean pulmonary artery pressure and pulmonary vascular resistance decreased (64 +/- 3 mm Hg to 57 +/- 3 mm Hg, p = 0.005 and 20 +/- 2 U x m2 to 15 +/- 2 U x m2, p = 0.01, respectively; n = 49). Kaplan-Meier survival estimates at one and two years were 98% and 91%, respectively. The risk for worsening PAH was lower in patients in WHO functional class I/II at bosentan initiation than in patients in WHO class III/IV at bosentan initiation.
These data suggest that bosentan, an oral endothelin ET(A)/ET(B) receptor antagonist, with or without concomitant prostanoid therapy, is safe and efficacious for the treatment of PAH in children.
本研究调查了接受波生坦治疗（无论是否联合使用前列环素类药物）的儿童肺动脉高压（PAH）患者的长期预后。
波生坦是一种口服内皮素ET(A)/ET(B)受体拮抗剂，可改善成年PAH患者的血流动力学和运动能力；然而，关于其对儿童长期影响的数据有限。
在这项回顾性研究中，86例PAH患儿（特发性、与先天性心脏病或结缔组织病相关）开始接受波生坦治疗，无论是否联合静脉注射依前列醇或皮下注射曲前列尼尔治疗。收集血流动力学、世界卫生组织（WHO）功能分级和安全性数据。
截止日期时，68例患者（79%）仍在接受波生坦治疗，13例（15%）停药，5例（6%）死亡。波生坦的中位暴露时间为14个月。90%的患者（n = 78）接受波生坦治疗后WHO功能分级改善（46%）或不变（44%）。平均肺动脉压和肺血管阻力下降（分别从64±3 mmHg降至57±3 mmHg，p = 0.005；从20±2 U x m2降至15±2 U x m2，p = 0.01；n = 49）。1年和2年的Kaplan-Meier生存率估计分别为98%和91%。波生坦起始治疗时WHO功能分级为I/II级的患者PAH恶化风险低于波生坦起始治疗时WHO分级为III/IV级的患者。
这些数据表明，口服内皮素ET(A)/ET(B)受体拮抗剂波生坦，无论是否联合前列环素类药物，对儿童PAH的治疗都是安全有效的。
