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特发性肺纤维化治疗药物的最新进展:展望 2023 年及以后。

An update on emerging drugs for the treatment of idiopathic pulmonary fibrosis: a look towards 2023 and beyond.

机构信息

Dipartimento di scienze mediche e chirurgiche, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy.

Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy.

出版信息

Expert Opin Emerg Drugs. 2023 Dec;28(4):283-296. doi: 10.1080/14728214.2023.2281416. Epub 2023 Dec 26.

Abstract

INTRODUCTION

Currently approved drug treatments for idiopathic pulmonary fibrosis (IPF), pirfenidone and nintedanib, have been shown to slow lung function decline and improve clinical outcomes. Since significant advances in the understanding of pathogenetic mechanisms in IPF, novel potential agents are being tested to identify new targeted and better tolerated therapeutic strategies.

AREAS COVERED

This review describes the evidence from IPF phase II and III clinical trials that have been completed or are ongoing in recent years. The literature search was performed using Medline and Clinicaltrials.org databases. Particular attention is paid to the new inhibitor of phosphodiesterase 4B (BI 1015550), being studied in a more advanced research phase. Some emerging critical issues of the pharmacological research are highlighted considering the recent outstanding failures of several phase III trials.

EXPERT OPINION

An exponential number of randomized clinical trials are underway testing promising new molecules to increase treatment choices for patients with IPF and improve patients' quality of life. The next goals should aim at a deeper understanding of the pathogenic pathways of the disease with the challenging goal of being able not only to stabilize but also to reverse the ongoing fibrotic process in patients with IPF.

摘要

简介

目前已批准的特发性肺纤维化 (IPF) 药物治疗药物吡非尼酮和尼达尼布已被证明可减缓肺功能下降并改善临床结局。由于对 IPF 发病机制的理解取得了重大进展,正在测试新型潜在药物以确定新的靶向和更耐受的治疗策略。

涵盖领域

本文综述了近年来已完成或正在进行的 IPF 二期和三期临床试验的证据。文献检索使用了 Medline 和 Clinicaltrials.org 数据库。特别关注在更先进的研究阶段研究的新型磷酸二酯酶 4B 抑制剂 (BI 1015550)。考虑到最近几项三期试验的突出失败,突出了药理学研究中的一些新出现的关键问题。

专家意见

目前正在进行大量的随机临床试验,以测试有前途的新药,为 IPF 患者增加治疗选择并提高患者的生活质量。下一步目标应旨在更深入地了解疾病的发病机制,具有挑战性的目标是不仅能够稳定而且能够逆转 IPF 患者持续的纤维化过程。

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