Jansa Pavel, Kopeć Grzegorz, Torbicki Adam, Sadushi-Kolici Roela, Campean Ioana-Alexandra, Halank Michael, Simkova Iveta, Steringer-Mascherbauer Regina, Salobir Barbara, Klepetko Walter, Lindner Jaroslav, Lang Irene M
Clinical Department of Cardiology and Angiology of the 2nd Department of Medicine General University Hospital Prague Czech Republic.
Department of Cardiac and Vascular Diseases, Pulmonary Circulation Centre Jagiellonian University Medical College and John Paul II Hospital in Krakow Krakow Poland.
Pulm Circ. 2023 Aug 21;13(3):e12274. doi: 10.1002/pul2.12274. eCollection 2023 Jul.
Chronic thromboembolic pulmonary hypertension (CTEPH) is successfully treatable with pulmonary endarterectomy (PEA), balloon pulmonary angioplasty, and medical therapy. Registry to Evaluate Early and Long-Term Pulmonary Arterial Hypertension Disease Management risk score (RRS) is able to predict long-term outcome in inoperable patients or in patients with residual PH after surgery. We performed a post hoc analysis of RRS in patients who were enrolled in the CTREPH study (NCT01416636), a randomized, double-blind clinical trial comparing high-dose and low-dose subcutaneous (SC) treprostinil in patients with severe CTEPH that was classified by an interdisciplinary CTEPH team as nonoperable, or as persistent or recurrent pulmonary hypertension after PEA. Baseline mean RRS was similar in both treatment groups (8.7 in high-dose arm vs. 8.6 in low-dose arm), but mean RRS change from baseline to Week 24 was greater in the high-dose treprostinil group than in the low-dose treprostinil group (-0.88 vs. -0.17). The difference in RRS change from baseline to Week 24 between high dose versus low dose was statistically significant with mean difference of -0.70 (95% confidence interval: -1.36 to -0.05, = 0.0352), and was driven mainly by improvement of World Health Organization functional class and N-terminal pro-brain natriuretic peptide concentration. SC treprostinil therapy administered in standard dose had positive effect on the risk profile measured by RRS in patients with inoperable or persistent/recurrent severe CTEPH. Although our study was limited by the small sample size and post hoc nature, assessment of risk profile is of great importance to this particular patient population with very poor prognosis.
慢性血栓栓塞性肺动脉高压（CTEPH）可通过肺动脉内膜剥脱术（PEA）、球囊肺动脉血管成形术及药物治疗成功治愈。评估肺动脉高压疾病管理风险评分（RRS）的注册研究能够预测无法手术的患者或术后残留肺动脉高压患者的长期预后。我们对参加CTREPH研究（NCT01416636）的患者进行了RRS的事后分析，该研究是一项随机、双盲临床试验，比较高剂量和低剂量皮下注射曲前列尼尔治疗严重CTEPH患者的疗效，这些患者经跨学科CTEPH团队分类为无法手术，或PEA术后持续或复发肺动脉高压。两个治疗组的基线平均RRS相似（高剂量组为8.7，低剂量组为8.6），但从基线到第24周，高剂量曲前列尼尔组的平均RRS变化大于低剂量曲前列尼尔组（-0.88对-0.17）。高剂量组与低剂量组从基线到第24周的RRS变化差异具有统计学意义，平均差异为-0.70（95%置信区间：-1.36至-0.05，P = 0.0352），主要由世界卫生组织功能分级和N末端脑钠肽前体浓度的改善所致。在无法手术或持续/复发严重CTEPH患者中，标准剂量皮下注射曲前列尼尔治疗对通过RRS测量的风险状况有积极影响。尽管我们的研究受到样本量小和事后分析性质的限制，但评估风险状况对这一预后极差的特定患者群体非常重要。
