肺动脉高压的临床试验设计、终点和新兴疗法。

IF 16.6 1区 医学 Q1 RESPIRATORY SYSTEM
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI:10.1183/13993003.01205-2024
Jason Weatherald, Thomas R Fleming, Martin R Wilkins, Thomas M Cascino, Mitchell A Psotka, Roham Zamanian, Werner Seeger, Nazzareno Galiè, Mardi Gomberg-Maitland
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引用次数: 0

摘要

肺动脉高压(PAH)临床试验已使几种能改善症状、运动能力和临床疗效的有效治疗方法获得批准。在三期临床试验中,主要终点必须反映患者的 "感觉、功能或存活 "情况。对于 PAH 这样的罕见疾病,随着治疗方案和候选疗法的不断增加,未来的临床试验面临着样本量要求、效率以及在接受多种药物背景治疗的患者中证明传统终点的增量获益等方面的挑战。新的临床试验终点、创新的试验设计和统计方法以及新技术可能是应对未来 PAH 试验所面临挑战的潜在解决方案,但这些方案必须为患者和监管机构所接受,同时又要保持方法的严谨性。在这篇世界肺动脉高压研讨会特别工作组的文章中,我们讨论了新出现的试验终点和设计、生物标记物和替代终点验证、疾病改变的概念、解决多样性和代表性问题的挑战和机遇,以及人工智能等新技术在 PAH 临床试验中的应用。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Clinical trial design, end-points, and emerging therapies in pulmonary arterial hypertension.

Clinical trials in pulmonary arterial hypertension (PAH) have led to the approval of several effective treatments that improve symptoms, exercise capacity and clinical outcomes. In phase 3 clinical trials, primary end-points must reflect how a patient "feels, functions or survives". In a rare disease like PAH, with an ever-growing number of treatment options and numerous candidate therapies being studied, future clinical trials are now faced with challenges related to sample size requirements, efficiency and demonstration of incremental benefit on traditional end-points in patients receiving background therapy with multiple drugs. Novel clinical trial end-points, innovative trial designs and statistical approaches and new technologies may be potential solutions to tackle the challenges facing future PAH trials, but these must be acceptable to patients and regulatory bodies while preserving methodological rigour. In this World Symposium on Pulmonary Hypertension task force article, we address emerging trial end-points and designs, biomarkers and surrogate end-point validation, the concept of disease modification, challenges and opportunities to address diversity and representativeness, and the use of new technologies such as artificial intelligence in PAH clinical trials.

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来源期刊
European Respiratory Journal
European Respiratory Journal 医学-呼吸系统
CiteScore
27.50
自引率
3.30%
发文量
345
审稿时长
2-4 weeks
期刊介绍: The European Respiratory Journal (ERJ) is the flagship journal of the European Respiratory Society. It has a current impact factor of 24.9. The journal covers various aspects of adult and paediatric respiratory medicine, including cell biology, epidemiology, immunology, oncology, pathophysiology, imaging, occupational medicine, intensive care, sleep medicine, and thoracic surgery. In addition to original research material, the ERJ publishes editorial commentaries, reviews, short research letters, and correspondence to the editor. The articles are published continuously and collected into 12 monthly issues in two volumes per year.
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