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肺动脉高压的未来展望:新型在研治疗方法与适应症综述

Future Perspectives of Pulmonary Arterial Hypertension: A Review of Novel Pipeline Treatments and Indications.

作者信息

Novara Maria Eugenia, Di Martino Enrica, Stephens Brandon, Nayrouz Mary, Vitulo Patrizio, Carollo Anna, Provenzani Alessio

机构信息

Clinical Pharmacy Service, Mediterranean Institute for Transplantation and Advanced Specialized Therapies (ISMETT), Palermo, Italy.

School of Pharmacy, University of Pittsburgh, Pittsburgh, PA, 15261, USA.

出版信息

Drugs R D. 2024 Mar;24(1):13-28. doi: 10.1007/s40268-024-00453-x. Epub 2024 Mar 22.

Abstract

Pulmonary arterial hypertension is characterized by elevated blood pressure and pathological changes in the pulmonary arterioles, leading to the development of right-heart failure and potentially fatal outcomes if left untreated. This review aims to provide an overview of novel drugs or formulations and new drug indications for pulmonary arterial hypertension that are currently in phases II-III of randomized controlled trials, and describe the rationale for the use of these targeted therapies, as well as their efficacy, safety profile, and impact on quality of life and survival. The literature research was conducted using data from ClinicalTrials.gov for the period between 1 January 2016 up to 31 December 2022. The population of interest includes individuals aged ≥ 18 years who have been diagnosed with pulmonary arterial hypertension. The review selection criteria included trials with recruiting, enrolling by invitation, active, terminated or completed status in 2022 and 2023. A total of 24 studies were selected for evaluation based on the inclusion and exclusion criteria. This review summarizes the updated information from randomized clinical trials involving novel therapies for pulmonary arterial hypertension. However, larger clinical trials are required to validate their clinical safety and effects. In the future, clinicians should choose therapies based on the patient's individual situation and requirements when developing treatment strategies.

摘要

肺动脉高压的特征是血压升高和肺小动脉的病理改变,如果不治疗,会导致右心衰竭并可能产生致命后果。本综述旨在概述目前处于随机对照试验II-III期的肺动脉高压新药或制剂以及新的药物适应症,并描述这些靶向治疗的使用原理、疗效、安全性概况以及对生活质量和生存率的影响。文献研究使用了ClinicalTrials.gov在2016年1月1日至2022年12月31日期间的数据。感兴趣的人群包括年龄≥18岁且已被诊断为肺动脉高压的个体。综述的选择标准包括在2022年和2023年处于招募、受邀入组、活跃、终止或完成状态的试验。根据纳入和排除标准,共选择了24项研究进行评估。本综述总结了涉及肺动脉高压新疗法的随机临床试验的最新信息。然而,需要更大规模的临床试验来验证其临床安全性和效果。未来,临床医生在制定治疗策略时应根据患者的个体情况和需求选择治疗方法。

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本文引用的文献

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Phase 3 Trial of Sotatercept for Treatment of Pulmonary Arterial Hypertension.索他洛尔治疗肺动脉高压的3期试验
N Engl J Med. 2023 Apr 20;388(16):1478-1490. doi: 10.1056/NEJMoa2213558. Epub 2023 Mar 6.
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Novel Mechanisms Targeted by Drug Trials in Pulmonary Arterial Hypertension.肺动脉高压药物试验的新靶点。
Chest. 2022 Apr;161(4):1060-1072. doi: 10.1016/j.chest.2021.10.010. Epub 2021 Oct 13.

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