University of Colorado Denver Health Sciences Center, Children's Hospital, USA.
Am J Cardiol. 2010 Nov 1;106(9):1332-8. doi: 10.1016/j.amjcard.2010.06.064.
Treatment algorithms in pediatric pulmonary arterial hypertension (PAH) are derived from clinical trials in adult populations and from clinical practice, but experience in children is limited. In this retrospective cohort study, we analyzed outcomes in a previously identified cohort of 86 consecutive children with PAH treated with bosentan as part of their treatment regimen. All children with idiopathic PAH or heritable PAH and PAH associated with congenital heart disease or connective tissue disease who started bosentan treatment from May 2001 to April 2003 in 2 tertiary pediatric referral centers were followed, with data collection ending August 2006. Eighty-six children (37 male, 49 female) 11 ± 5 years of age with idiopathic/heritable PAH (n = 36), PAH associated with congenital heart disease (n = 48), or PAH associated with connective tissue disease (n = 2) received bosentan as monotherapy (n = 42) or as an add-on to pre-existing continuous intravenous epoprostenol or subcutaneous treprostinil (n = 44). Median observation period was 39 months (range 2 to 60). Thirty-four patients (40%) received ≥1 additional PAH-specific therapy during follow-up. At end of data collection, 25 patients (29%) remained on bosentan, 43 (50%) had stopped bosentan, 11 (13%) had died while on bosentan, and 7 were lost to follow-up. At 4 years, the Kaplan-Meier estimate of disease progression in patients while on bosentan was 54% (7 patients at risk) with a survival estimate of 82% (16 patients at risk). Risk factors significantly associated with survival were World Health Organization functional class and indexed pulmonary vascular resistance. In conclusion, outcome in children with PAH managed with current treatment regimens appears favorable. However, despite current therapy options, disease progression remains a concern.
儿童肺动脉高压 (PAH) 的治疗方案源自成人人群的临床试验和临床实践，但儿童的经验有限。在这项回顾性队列研究中，我们分析了在之前确定的 86 例接受波生坦治疗的 PAH 儿童队列中的结局，这些儿童均接受波生坦治疗作为其治疗方案的一部分。所有患有特发性 PAH 或遗传性 PAH 以及与先天性心脏病或结缔组织疾病相关的 PAH 的儿童，如果在 2001 年 5 月至 2003 年 4 月期间在 3 个儿科转诊中心开始接受波生坦治疗，则纳入本研究，数据收集于 2006 年 8 月结束。86 例（男 37 例，女 49 例）年龄为 11 ± 5 岁，患有特发性/遗传性 PAH（n = 36）、与先天性心脏病相关的 PAH（n = 48）或与结缔组织疾病相关的 PAH（n = 2），接受波生坦单药治疗（n = 42）或作为现有持续静脉内依前列醇或皮下曲前列尼尔的附加治疗（n = 44）。中位观察期为 39 个月（范围 2 至 60）。34 例（40%）在随访期间接受了≥1 种额外的 PAH 特异性治疗。在数据收集结束时，25 例（29%）继续接受波生坦治疗，43 例（50%）停止了波生坦治疗，11 例（13%）在接受波生坦治疗时死亡，7 例失访。4 年时，接受波生坦治疗的患者疾病进展的 Kaplan-Meier 估计值为 54%（7 例处于风险中），生存率估计值为 82%（16 例处于风险中）。与生存显著相关的危险因素是世界卫生组织功能分级和肺血管阻力指数。总之，采用目前的治疗方案管理儿童 PAH 的结局似乎较好。然而，尽管有了目前的治疗选择，疾病进展仍然是一个问题。
