肺肉样瘤病的临床试验；需要什么、正在发生什么以及下一步是什么？

IF 2.8 3区医学 Q2 RESPIRATORY SYSTEM

Current Opinion in Pulmonary Medicine Pub Date : 2024-09-01 Epub Date: 2024-07-04 DOI:10.1097/MCP.0000000000001098

Marcel Veltkamp

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引用次数: 0

摘要

审查目的：改善肺肉样瘤病患者治疗的临床需求尚未得到满足。由于肉样瘤病患者的表现和病程各不相同，回顾性和前瞻性药物试验都受到了阻碍。本综述概述了肺肉样瘤病的各种药物试验，重点是不同的主要终点和与之相关的问题：近年来，不同的工作组/研究机构利用肉样瘤病患者提供的大量信息，试图开发出一套最重要的核心结果，以便在今后的肉样瘤病治疗研究中加以衡量。此外，目前正在对治疗肺肉样瘤病的新药进行三项大型临床试验。总结：在制定一套核心疗效测量指标方面已取得进展，这些指标可用于制定未来肉样瘤病药物试验的综合主要终点。

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Clinical trials in pulmonary sarcoidosis; what is needed, what is happening and what is next?

Purpose of review: There is a clinical unmet need to improve treatment for patients with pulmonary sarcoidosis. Both retrospective and prospective drug trials are hampered by the fact that patients with sarcoidosis are characterized by a heterogeneous presentation and disease course. In this review, an overview is given of different drug trials in pulmonary sarcoidosis with an emphasis on different primary endpoints and the problems related to them.

Recent findings: In recent years, using significant input from patients with sarcoidosis, different task-forces/studies tried to develop a core set of most important outcomes to measure in future studies on treatment of sarcoidosis. Furthermore, at present, three major clinical trials are being conducted on new drugs for treatment of pulmonary sarcoidosis.

Summary: Progress has been made to develop a core set of outcomes measures that can be used in making a combined primary endpoint in future drug trials in sarcoidosis.

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来源期刊

Current Opinion in Pulmonary Medicine 医学-呼吸系统

CiteScore

6.20

自引率

0.00%

发文量

109

审稿时长

6-12 weeks

期刊介绍： Current Opinion in Pulmonary Medicine is a highly regarded journal offering insightful editorials and on-the-mark invited reviews, covering key subjects such as asthma; cystic fibrosis; infectious diseases; diseases of the pleura; and sleep and respiratory neurobiology. Published bimonthly, each issue of Current Opinion in Pulmonary Medicine introduces world renowned guest editors and internationally recognized academics within the pulmonary field, delivering a widespread selection of expert assessments on the latest developments from the most recent literature.