Jansa Pavel, Sadushi-Kolici Roela, Skoro-Sajer Nika, Kopec Grzegorz, Simkova Iveta, Steringer-Mascherbauer Regina, Salobir Barbara, Lindner Jaroslav, Lang Irene M
Clinical Department of Cardiology and Angiology, 2nd Department of Medicine General University Hospital Prague Czech Republic.
Department of Internal Medicine II, Division of Cardiology Medical University of Vienna Vienna Austria.
Pulm Circ. 2025 Apr 27;15(2):e70080. doi: 10.1002/pul2.70080. eCollection 2025 Apr.
The aim of the open label extension (OLE) of CTREPH study was to characterize multimodal treatment in patients with severe inoperable CTEPH, to describe long-term subcutaneous (SC) treprostinil safety and tolerability, and to evaluate change in functional class and exercise capacity over 24 months since completion of the blinded phase of CTREPH. The target population in the OLE consisted of patients who completed 24 weeks of blinded treatment with either high-dose treprostinil of around 30 ng/kg/min (former high-dose group), or low-dose treprostinil of around 3 ng/kg/min (former low-dose group) in the CTREPH study. From the start of OLE, treprostinil dose and any additional therapy were chosen according to the standard of care and physician's discretion. Out of 47 enrolled patients, 20 patients received other PH drugs during OLE and 17 patients underwent at least 1 BPA session. Number of treprostinil-related AEs was substantially higher in the former low-dose group in comparison to the former high-dose group. Related AEs were also more frequent during the first 6 months of the preceding blinded trial than over 24 months of OLE, especially infusion site pain and all local infusion site reactions. No new safety signal was detected. Evaluated clinical outcomes show sustained benefit from long-term treprostinil treatment. Long-term SC treprostinil is a safe and effective component of multimodal treatment for patients with severe CTEPH. Patients who tolerate treprostinil after initiation are likely to continue tolerating it over time, with the clinical benefit maintained over 24 months.
CTREPH研究开放标签扩展（OLE）的目的是明确重度无法手术的慢性血栓栓塞性肺动脉高压（CTEPH）患者的多模式治疗特征，描述皮下注射曲前列尼尔的长期安全性和耐受性，并评估自CTREPH研究双盲阶段结束后24个月内功能分级和运动能力的变化。OLE的目标人群包括在CTREPH研究中接受了24周双盲治疗的患者，这些患者使用的曲前列尼尔剂量为高剂量（约30 ng/kg/min，原高剂量组）或低剂量（约3 ng/kg/min，原低剂量组）。从OLE开始，曲前列尼尔剂量和任何额外治疗均根据医疗标准和医生的判断来选择。在47名入组患者中，20名患者在OLE期间接受了其他肺动脉高压药物治疗，17名患者至少接受了1次球囊肺血管成形术（BPA）治疗疗程。与原高剂量组相比，原低剂量组中与曲前列尼尔相关的不良事件数量显著更高。在前述双盲试验的前6个月中，相关不良事件也比OLE期间的24个月更频繁出现，尤其是输注部位疼痛和所有局部输注部位反应。未检测到新的安全信号。评估的临床结果显示长期使用曲前列尼尔治疗具有持续益处。长期皮下注射曲前列尼尔是重度CTEPH患者多模式治疗的安全有效组成部分。起始后耐受曲前列尼尔的患者可能会长期持续耐受，临床益处可维持24个月。
