Long-term outcome in pulmonary arterial hypertension patients treated with subcutaneous treprostinil
Long-term outcome in pulmonary arterial hypertension patients treated with subcutaneous treprostinil
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DOI：
10.1183/09031936.06.00044406
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发表时间：
2006-12-01
影响因子：
24.3
通讯作者：
Rubin, L. J.
中科院分区：
文献类型：
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作者：
Barst, R. J.;Galie, N.;Rubin, L. J.
肺动脉高压（PAH）如果不治疗是致命的。静脉注射依前列醇可改善PAH患者的运动能力和血液动力学，并增加特发性PAH（IPAH）患者的生存率。为了评价皮下（SC）曲前列环素（一种长效前列环素类似物）的效果，然后在需要时添加其他PAH治疗，对860例接受SC曲前列环素治疗长达4年的PAH患者进行了随访。对于332例具有基线血流动力学的IPAH患者，观察到的生存率也与使用国立卫生研究院公式预测的生存率进行了比较，在860例患者中，199例（23%）因不良事件而停药，136例（16%）死亡，117例（14%）因恶化而停药，29例（3%）撤回同意书，11例（1%）接受移植。总共有97例患者（11%）从SC曲前列尼尔转换为替代前列环素类似物; 105例患者（12%）添加波生坦，25例患者（3%）添加西地那非。总之，所有860例患者的1-4年生存率为87-68%，皮下曲前列尼尔单药治疗的1-4年生存率为88-70%。对于具有基线血流动力学的特发性肺动脉高压亚组（n=332），1-4年的生存率为91-72%。相比之下，1 -4年的预测生存率为69-38%。长期皮下注射曲前列尼尔的安全性特征与先前的短期试验一致，无意外不良事件。
Pulmonary arterial hypertension (PAH) is fatal if untreated. Intravenous epoprostenol improves exercise capacity and haemodynamics in PAH, and increases survival in idiopathic PAH (IPAH). To evaluate the effects of subcutaneous (SC) treprostinil, a longer-acting prostacyclin analogue, followed by the addition of other PAH therapies if needed, 860 PAH patients treated with SC treprostinil for up to 4 yrs were followed.Survival is reported as Kaplan-Meier estimates. For 332 IPAH patients with baseline haemodynamics, observed survival is also compared with predicted survival using the National Institute of Health formula.Out of the 860 patients, 199 (23%) discontinued due to adverse events, 136 (16%) died, 117 (14%) discontinued due to deterioration, 29 (3%) withdrew consent and 11 (1%) underwent transplantation. In total, 97 patients (11%) switched from SC treprostinil to an alternative prostacyclin analogue; bosentan was added in 105 patients (12%) and sildenafil in 25 (3%).In conclusion, survival was 87-68% over 1-4 yrs for all 860 patients and 88-70% over 1-4 yrs with subcutaneous treprostinil monotherapy. For the idiopathic pulmonary arterial hypertension subset with baseline haemodynamics (n=332), survival was 91-72% over 1-4 yrs. In contrast, predicted survival was 69-38% over 1-4 yrs. The safety profile for long-term subcutaneous treprostinil was consistent with previous short-term trials with no unexpected adverse events.