Department of Internal Medicine II, Division of Cardiology, Vienna General Hospital, Medical University of Vienna, Vienna, Austria.
György Gottsegen, Hungarian Institute of Cardiology, Budapest, Hungary.
Heart. 2018 Jul;104(14):1195-1199. doi: 10.1136/heartjnl-2017-312143. Epub 2018 Feb 7.
To assess the efficacy and safety of subcutaneous treprostinil in adult patients with congenital heart disease (CHD)-associated pulmonary arterial hypertension (PAH) after 12 months of treatment.
Consecutive adult patients with CHD-PAH received subcutaneous treprostinil to maximum tolerated doses in an observational study.
Advanced CHD-PAH patients with WHO class III or IV disease (n=32, age 40±10 years, 20 females) received treprostinil for suboptimal response to bosentan (n=12), WHO functional class IV disease (FC, n=7) or prior to bosentan approval (n=13). In the multivariate mixed model, mean increase in 6 min walk distance (6-MWD) from baseline to 12 months was 114 m (76; 152) (P<0.001). WHO FC improved significantly (P=0.001) and B-type brain natriuretic peptide decreased from 1259 (375; 2368) pg/mL to 380 (144; 1468) pg/mL (P=0.02). In those 14 patients who had haemodynamic data before and after initiation of treprostinil, pulmonary vascular resistance decreased significantly (from 18.4±11.1 to 12.6±7.9 Wood units, P=0.003). The most common adverse events were infusion-site erythema and pain. One patient stopped treatment because of intolerable infusion-site pain after 8 months of treatment. No other major treatment-related complications were observed. Five patients died during early follow-up, having experienced a decrease in their 6-MWD prior.
Subcutaneous treprostinil therapy is generally safe and effective for at least 12 months and may be used in CHD-related PAH class III and IV.
评估皮下曲前列尼尔治疗 12 个月后成人先天性心脏病（CHD）相关肺动脉高压（PAH）患者的疗效和安全性。
连续的 CHD-PAH 成年患者在观察性研究中接受皮下曲前列尼尔治疗，直至达到最大耐受剂量。
32 例（年龄 40±10 岁，20 例女性）WHO 功能分级 III 或 IV 级疾病（n=32）、对波生坦治疗反应不佳（n=12）、WHO 功能分级 IV 级疾病（n=7）或在波生坦获得批准前（n=13）的晚期 CHD-PAH 患者接受了曲前列尼尔治疗。在多变量混合模型中，从基线到 12 个月时 6 分钟步行距离（6-MWD）的平均增加量为 114m（76；152）（P<0.001）。WHO FC 显著改善（P=0.001），B 型脑钠肽从 1259（375；2368）pg/ml 降至 380（144；1468）pg/ml（P=0.02）。在开始曲前列尼尔治疗前后有血流动力学数据的 14 例患者中，肺血管阻力显著降低（从 18.4±11.1 伍德单位降至 12.6±7.9 伍德单位，P=0.003）。最常见的不良事件是输注部位红斑和疼痛。1 例患者在治疗 8 个月后因难以忍受的输注部位疼痛而停止治疗。未观察到其他与治疗相关的严重并发症。5 例患者在早期随访期间死亡，他们在死亡前经历了 6-MWD 的下降。
皮下曲前列尼尔治疗至少 12 个月通常是安全有效的，可用于 CHD 相关的 PAH III 级和 IV 级患者。
