Kędzierski Piotr, Banaszkiewicz Marta, Florczyk Michał, Piłka Michał, Mańczak Rafał, Wieteska-Miłek Maria, Szwed Piotr, Kasperowicz Krzysztof, Wrona Katarzyna, Darocha Szymon, Torbicki Adam, Kurzyna Marcin
Chair and Department of Pulmonary Circulation, Thromboembolic Diseases and Cardiology, Centre of Postgraduate Medical Education, European Health Centre, ERN-LUNG Member, 05-400 Otwock, Poland.
Department of Vascular, Endovascular Surgery, Angiology and Phlebology, Poznan University of Medical Science, 61-701 Poznan, Poland.
Biomedicines. 2025 Jan 13;13(1):172. doi: 10.3390/biomedicines13010172.
: Treprostinil, which is administered via continuous subcutaneous or intravenous infusion, is a medication applied in the treatment of pulmonary arterial hypertension (PAH). The dose of treprostinil is adjusted on an individual basis for each patient. A number of factors determine how well patients respond to treatment. : The aim of this study was to identify factors that may influence the clinical response to the dose of treprostinil at 3 months after the start of therapy. : The factors influencing treatment response were analyzed in consecutive PAH patients who started receiving treprostinil treatment. The treatment efficacy was assessed as improvement in 6 min walk distance (6MWD) and WHO functional class (WHO FC), a reduction in N-terminal prohormone of brain natriuretic peptide (NTproBNP), and the percentage of patients achieving low-risk status after 12 months of treatment. : A total of 83 patients were included in this analysis. Classification of patients according to the tertiles of treprostinil dose achieved at 3 months after drug inclusion shows that after 12 months of follow-up, the median WHO FC in the highest dose group was lower than that in the intermediate dose group (WHO FC II vs. WHO FC III, = 0.005), the median NTproBNP was lower (922 pg/mL, vs. 1686 pg/mL, = 0.036) and 6MWD was longer (300 m vs. 510 m, = 0.015). The French Noninvasive Criteria (NIFC) scale score was higher (2 vs. 0, = 0.008), and the Reveal scale score was lower (5.0 vs. 8.5, = 0.034). In the group of patients who exceeded a dose of 19.8 ng/kg/min within 3 months, an improvement in 6MWD was observed significantly more often after one year of therapy, and they were more likely to show an increase in NIFC scale scores after one year of therapy than the group of patients who received the lower dose (65% vs. 30%, = 0.02). In the group of patients younger than 50 years of age, a statistically significant correlation was observed between the dose of treprostinil achieved after three months of treatment and the parameters assessed after 12 months of treatment, including WHO FC, 6MWD, and NIFC prognostic scale scores (all < 0.05). : The clinical effect of treatment is critically dependent on the rapid escalation of the treprostinil dose during the first three months of treatment.
前列环素通过持续皮下或静脉输注给药，是一种用于治疗肺动脉高压（PAH）的药物。前列环素的剂量针对每位患者进行个体化调整。有许多因素决定患者对治疗的反应程度。本研究的目的是确定在治疗开始3个月后可能影响对前列环素剂量临床反应的因素。对开始接受前列环素治疗的连续性PAH患者分析影响治疗反应的因素。治疗效果通过6分钟步行距离（6MWD）改善、世界卫生组织功能分级（WHO FC）、脑钠肽N末端前体激素（NTproBNP）降低以及治疗12个月后达到低风险状态的患者百分比来评估。本分析共纳入83例患者。根据药物纳入后3个月达到的前列环素剂量三分位数对患者进行分类，结果显示，随访12个月后，最高剂量组的WHO FC中位数低于中间剂量组（WHO FC II级对WHO FC III级，P = 0.005），NTproBNP中位数更低（922 pg/mL对1686 pg/mL，P = 0.036），6MWD更长（300 m对510 m，P = 0.015）。法国无创标准（NIFC）量表评分更高（2分对0分，P = 0.008），而Reveal量表评分更低（5.0分对8.5分，P = 0.034）。在3个月内超过19.8 ng/kg/min剂量的患者组中，治疗1年后6MWD改善更为常见，且与接受较低剂量的患者组相比，治疗1年后他们NIFC量表评分增加的可能性更大（65%对30%，P = 0.02）。在年龄小于50岁的患者组中，观察到治疗3个月后达到的前列环素剂量与治疗12个月后评估的参数之间存在统计学显著相关性，包括WHO FC、6MWD和NIFC预后量表评分（均P < 0.05）。治疗的临床效果严重依赖于治疗前三个月前列环素剂量的快速递增。
