Miranda Aimon C, Cornelio Cyrille K, Tran Bao Anh C, Fernandez Joel
Department of Pharmacotherapeutics and Clinical Research, Taneja College of Pharmacy, University of South Florida Health, Tampa, FL, USA.
Internal Medicine, Morsani College of Medicine, University of South Florida Health, Tampa, FL, USA.
J Pharm Technol. 2025 Feb 22:87551225251317957. doi: 10.1177/87551225251317957.
The objective of the study is to review the characteristics, efficacy, safety, and clinical relevance of sotatercept in pulmonary arterial hypertension (PAH). A literature search containing search terms related to sotatercept and PAH was conducted. Embase via Elsevier, MEDLINE via Ovid, the medRxiv preprint server, Cochrane Library CENTRAL trials registry, and ClinicalTrials.gov were searched from inception through October 31, 2024. The package insert was utilized to obtain drug information and additional data. Phase II-III clinical trials investigating sotatercept for PAH were included. Articles written in English were extracted while animal studies and phase I clinical trials were excluded. In patients with WHO Group 1, functional class II-III PAH, adding sotatercept to background therapy increased 6-minute walk distance in phase II-III trials. Pooled analysis from PULSAR (phase II) and STELLAR (phase III) showed improvements in pulmonary vascular resistance and NT-proBNP. Exploratory data from PULSAR revealed that BMPR2 genetic variant status was not associated with significant differences in treatment effects. SPECTRA (phase IIb) demonstrated improved right ventricular structure and function. Interim analysis from SOTERIA showed that treatment effects persist at 1 year. Sotatercept is a viable add-on therapy for patients with PAH Group 1 and functional class II-III. Additional data are needed to assess long-term outcomes among treatment-naïve patients and those with the most severe symptomatology.
本研究的目的是回顾索他西普在肺动脉高压（PAH）中的特征、疗效、安全性及临床相关性。进行了一项文献检索，检索词包含与索他西普和PAH相关的术语。通过爱思唯尔检索Embase、通过Ovid检索MEDLINE、检索medRxiv预印本服务器、Cochrane图书馆CENTRAL试验注册库以及ClinicalTrials.gov，检索时间从各数据库创建之初至2024年10月31日。利用药品说明书获取药物信息及其他数据。纳入了研究索他西普用于PAH的II - III期临床试验。提取英文撰写的文章，排除动物研究和I期临床试验。在世界卫生组织1组、功能分级为II - III级的PAH患者中，在II - III期试验中，在背景治疗基础上加用索他西普可增加6分钟步行距离。PULSAR（II期）和STELLAR（III期）的汇总分析显示肺血管阻力和N末端B型利钠肽原（NT - proBNP）有所改善。PULSAR的探索性数据显示，骨形态发生蛋白受体2（BMPR2）基因变异状态与治疗效果的显著差异无关。SPECTRA（IIb期）显示右心室结构和功能得到改善。SOTERIA的中期分析表明，治疗效果在1年时持续存在。索他西普是1组、功能分级为II - III级PAH患者可行的附加治疗药物。需要更多数据来评估初治患者和症状最严重患者的长期结局。
