Dagher Chebly, Akiki Maria, Swanson Kristin, Carollo Brett, Fiscus Garett, Farber Harrison W, Parikh Raj
Department of Internal Medicine, University of Connecticut, Farmington, CT 06269, USA.
Division of Pulmonary, Critical Care and Sleep, Hartford Hospital, Hartford, CT 06106, USA.
J Clin Med. 2025 Jul 22;14(15):5177. doi: 10.3390/jcm14155177.
: Sotatercept has demonstrated efficacy in pulmonary arterial hypertension (PAH), but its use has not been studied in patients with Group 3 pulmonary hypertension (PH). Additionally, patients with connective tissue disease-associated PAH (CTD-PAH) were underrepresented in the STELLAR trial. Given the limited treatment options for pulmonary hypertension in patients with interstitial lung disease (PH-ILD), this study aimed to evaluate the use of sotatercept in CTD-PAH patients with concomitant ILD. : Eligible patients ( = 7) had a confirmed diagnosis of CTD-PAH with concomitant ILD. The patients were already receiving background PAH therapy. Baseline hemodynamic and clinical measurements were reassessed after 24 weeks of sotatercept therapy. The variables assessed included six-minute walk distance (6MWD), pulmonary vascular resistance (PVR), echocardiographic right ventricular systolic pressure (eRVSP), N-terminal pro-brain natriuretic peptide (NT-proBNP) levels, World Health Organization (WHO) functional class, and supplemental oxygen requirements. : The study included seven patients with a mean age of 57 years (range: 39-73 years). After 24 weeks, the mean 6MWT distance increased from 211 m to 348 m ( < 0.01). Mean PVR decreased from 7.77 WU at baseline to 4.53 WU ( < 0.01). Mean eRVSP decreased from 79.43 mmHg to 54.14 mmHg ( < 0.01). NT-proBNP decreased from 3056.86 pg/mL to 1404.29 pg/mL ( < 0.01). The WHO functional class and supplemental oxygen requirements improved in all patients. : Sotatercept was tolerated in patients with CTD-PAH and ILD, with no evidence of adverse respiratory effects. When added to foundational PAH therapy, sotatercept resulted in significant improvements across multiple parameters. These findings suggest that sotatercept may be a promising therapeutic option as an adjunctive treatment in this patient population.
索他西普已在肺动脉高压（PAH）中显示出疗效，但尚未在3组肺动脉高压（PH）患者中进行研究。此外，在STELLAR试验中，结缔组织病相关性PAH（CTD-PAH）患者的代表性不足。鉴于间质性肺疾病（PH-ILD）患者的肺动脉高压治疗选择有限，本研究旨在评估索他西普在合并ILD的CTD-PAH患者中的应用。符合条件的患者（n = 7）确诊为CTD-PAH合并ILD。这些患者已在接受PAH背景治疗。在索他西普治疗24周后重新评估基线血流动力学和临床指标。评估的变量包括6分钟步行距离（6MWD）、肺血管阻力（PVR）、超声心动图右心室收缩压（eRVSP）、N末端脑钠肽前体（NT-proBNP）水平、世界卫生组织（WHO）功能分级和补充氧气需求。该研究纳入了7名平均年龄为57岁（范围：39 - 73岁）的患者。24周后，平均6MWT距离从211米增加到348米（P < 0.01）。平均PVR从基线时的7.77伍德单位降至4.53伍德单位（P < 0.01）。平均eRVSP从79.43 mmHg降至54.14 mmHg（P < 0.01）。NT-proBNP从3056.86 pg/mL降至1404.29 pg/mL（P < 0.01）。所有患者的WHO功能分级和补充氧气需求均有所改善。CTD-PAH和ILD患者对索他西普耐受性良好，无不良呼吸效应的证据。当添加到基础PAH治疗中时，索他西普在多个参数上有显著改善。这些发现表明，索他西普作为该患者群体的辅助治疗可能是一种有前景的治疗选择。
