Division of Pediatric Cardiology, Columbia University College of Physicians and Surgeons, New York, New York, USA.
Am J Cardiol. 2010 May 15;105(10):1485-9. doi: 10.1016/j.amjcard.2009.12.075. Epub 2010 Apr 8.
Before 2001, intravenous epoprostenol was the only approved drug for patients with idiopathic pulmonary arterial hypertension (IPAH) or familial pulmonary arterial hypertension (FPAH) who were nonresponsive to high-dose calcium channel blockade. The investigators report transitioning select pediatric patients with IPAH or FPAH from intravenous epoprostenol to oral and/or inhaled agents for pulmonary arterial hypertension. A retrospective review was performed on all pediatric patients with IPAH or FPAH treated at Columbia University (1987 to 2008) who transitioned off epoprostenol to oral or inhaled drugs. Criteria for transition included functional class I or II, mean pulmonary arterial pressure <35 mm Hg, normal cardiac index, and age >6 years. Hemodynamic and clinical data were obtained at baseline (before epoprostenol), at peak epoprostenol dose, and after epoprostenol discontinuation. Fourteen of 104 pediatric patients with IPAH or FPAH transitioned off epoprostenol to oral or inhaled drugs from April 2003 to July 2008. Of the 14 subjects, 13 transitioned off epoprostenol successfully to oral or inhaled drugs. No significant changes in functional class, hemodynamics, or exercise data were seen after epoprostenol (mean follow-up duration 7.0 +/- 5.8 months) compared to peak epoprostenol, but further improvement was reported in World Health Organization functional class (p <0.005) after epoprostenol discontinuation. After successful epoprostenol discontinuation, 77% of patients were treated with endothelin receptor antagonists, 69% with phosphodiesterase-5 inhibitors, 38% with calcium channel blockers, and 8% inhaled iloprost. At the cut-off date (May 2009), there was 100% survival and 93% transition success. In conclusion, in carefully selected children with IPAH or FPAH initiated on intravenous epoprostenol before the availability of nonparenteral therapy, transition to oral or inhaled therapy for pulmonary arterial hypertension appears safe, with efficacy maintained when performed with close follow-up at a pulmonary hypertension specialty center.
在 2001 年之前，对于对大剂量钙通道阻滞剂无反应的特发性肺动脉高压（IPAH）或家族性肺动脉高压（FPAH）患者，静脉内依前列醇是唯一批准的药物。研究人员报告了将特发性肺动脉高压或家族性肺动脉高压的部分儿科患者从静脉内依前列醇转为口服和/或吸入肺动脉高压药物。对 2008 年以前在哥伦比亚大学接受治疗的所有特发性肺动脉高压或家族性肺动脉高压儿科患者（1987 年至 2008 年）进行了回顾性分析，这些患者已从静脉内依前列醇转为口服或吸入药物。转换的标准包括功能分类 I 或 II，平均肺动脉压<35mmHg，正常心指数和年龄>6 岁。在开始使用依前列醇之前（基线），在依前列醇峰值剂量时和停用依前列醇后获得血流动力学和临床数据。2003 年 4 月至 2008 年 7 月期间，104 名特发性肺动脉高压或家族性肺动脉高压儿科患者中有 14 名从静脉内依前列醇转为口服或吸入药物。在 14 名患者中，有 13 名成功转为口服或吸入药物。与依前列醇峰值相比，停用依前列醇后（平均随访时间为 7.0+/-5.8 个月），功能分类，血流动力学或运动数据无明显变化，但世界卫生组织功能分类进一步改善（p<0.005）。依前列醇停用后，77%的患者接受内皮素受体拮抗剂治疗，69%接受磷酸二酯酶-5 抑制剂治疗，38%接受钙通道阻滞剂治疗，8%接受吸入伊洛前列素治疗。在截止日期（2009 年 5 月），100%的患者存活，93%的患者成功过渡。总之，在开始接受非肠外治疗之前已接受静脉内依前列醇治疗的特发性肺动脉高压或家族性肺动脉高压的精心选择的儿童中，转为口服或吸入肺动脉高压药物治疗似乎是安全的，并且在肺动脉高压专业中心进行密切随访时可以维持疗效。
