磷酸二酯酶-4抑制作为与风湿性疾病相关的间质性肺疾病的治疗策略的基本原理

IF 5.1 2区 医学 Q1 RHEUMATOLOGY RMD Open Pub Date : 2024-12-23 DOI:10.1136/rmdopen-2024-004704
Martin Aringer, Oliver Distler, Anna-Maria Hoffmann-Vold, Masataka Kuwana, Helmut Prosch, Elizabeth R Volkmann
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引用次数: 0

摘要

与类风湿关节炎或结缔组织疾病(如系统性硬化症)相关的间质性肺病(ILD)可统称为系统性自身免疫性风湿性疾病相关ILD (sard -ILD)或风湿性肌肉骨骼疾病相关ILD。SARD-ILD导致大量的发病率和死亡率,因此对SARD-ILD中针对纤维化和炎症途径的有效治疗有很高的医学需求。磷酸二酯酶4 (PDE4)水解环AMP,其调节炎症过程中的多种途径。PDE4在炎性疾病患者外周血单核细胞中过表达。然而,在纤维化条件下泛pde4抑制的临床数据缺乏。PDE4B亚型在脑、肺、心脏、骨骼肌和免疫细胞中高度表达。因此,抑制PDE4B可能是治疗特发性肺纤维化(IPF)和SARD-ILD等纤维化性ild的一种新方法。PDE4B抑制的临床前数据提供了抗炎和抗纤维化活性的初步证据,与泛pde4抑制剂相比,其胃肠道毒性潜在降低。在IPF患者的概念验证II期试验中,nerandomilast (BI 1015550)是目前临床开发的唯一PDE4B抑制剂,与安慰剂相比,在12周内可防止肺功能下降。PDE4B抑制的潜在临床益处目前正在III期研究中,有两项试验评估nerandomilast对IPF (FIBRONEER-IPF)或进行性肺纤维化(非IPF (FIBRONEER-ILD))患者的治疗效果。在这里,我们回顾了临床前和临床数据,这些数据为PDE4B抑制作为SARD-ILD患者的治疗策略提供了依据。
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Rationale for phosphodiesterase-4 inhibition as a treatment strategy for interstitial lung diseases associated with rheumatic diseases.

Interstitial lung disease (ILD) associated with rheumatoid arthritis or with connective tissue diseases such as systemic sclerosis can be collectively named systemic autoimmune rheumatic disease-associated ILDs (SARD-ILDs) or rheumatic musculoskeletal disorder-associated ILDs. SARD-ILDs result in substantial morbidity and mortality, and there is a high medical need for effective therapies that target both fibrotic and inflammatory pathways in SARD-ILD. Phosphodiesterase 4 (PDE4) hydrolyses cyclic AMP, which regulates multiple pathways involved in inflammatory processes. PDE4 is overexpressed in peripheral blood monocytes from patients with inflammatory diseases. However, clinical data on pan-PDE4 inhibition in fibrotic conditions are lacking. The PDE4B subtype is highly expressed in the brain, lungs, heart, skeletal muscle and immune cells. As such, inhibition of PDE4B may be a novel approach for fibrosing ILDs such as idiopathic pulmonary fibrosis (IPF) and SARD-ILD. Preclinical data for PDE4B inhibition have provided initial evidence of both anti-inflammatory and antifibrotic activity, with reduced potential for gastrointestinal toxicity compared with pan-PDE4 inhibitors. In a proof-of-concept phase II trial in patients with IPF, nerandomilast (BI 1015550), the only PDE4B inhibitor currently in clinical development, prevented a decline in lung function over 12 weeks compared with placebo. The potential clinical benefit of PDE4B inhibition is now being investigated in the phase III setting, with two trials evaluating nerandomilast in patients with IPF (FIBRONEER-IPF) or with progressive pulmonary fibrosis other than IPF (FIBRONEER-ILD). Here, we review the preclinical and clinical data that provide rationale for PDE4B inhibition as a treatment strategy in patients with SARD-ILD.

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来源期刊
RMD Open
RMD Open RHEUMATOLOGY-
CiteScore
7.30
自引率
6.50%
发文量
205
审稿时长
14 weeks
期刊介绍: RMD Open publishes high quality peer-reviewed original research covering the full spectrum of musculoskeletal disorders, rheumatism and connective tissue diseases, including osteoporosis, spine and rehabilitation. Clinical and epidemiological research, basic and translational medicine, interesting clinical cases, and smaller studies that add to the literature are all considered.
期刊最新文献
Correction: Efficacy and safety of filgotinib in patients with rheumatoid arthritis: week 156 interim results from a long- term extension study. Correction: Efficacy and safety of jaktinib hydrochloride tablets in active axial spondyloarthritis: a multicentre, randomised, double-blind, placebo-controlled phase II clinical trial. Distinct clinical outcomes based on multiple serum cytokine and chemokine profiles rather than autoantibody profiles and ultrasound findings in rheumatoid arthritis: a prospective ultrasound cohort study. Trends in work participation among patients with inflammatory rheumatic musculoskeletal diseases (iRMDs): Data from the German National Database (2010-2022). Efficacy and safety of pharmacological treatments in inclusion body myositis: a systematic review.
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