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成人及青少年皮肌炎中 Janus 激酶抑制剂的现有证据及关键比较。

Current evidence for janus kinase inhibitors in adult and juvenile dermatomyositis and key comparisons.

作者信息

Wallwork Rachel S, Paik Julie J, Kim Hanna

机构信息

Division of Rheumatology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.

National Institute of Arthritis Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA.

出版信息

Expert Opin Pharmacother. 2024 Aug;25(12):1625-1645. doi: 10.1080/14656566.2024.2392021. Epub 2024 Aug 18.

Abstract

INTRODUCTION

Adult dermatomyositis (DM) and juvenile dermatomyositis (JDM) are rare autoimmune diseases with characteristic skin rashes, weakness, and other systemic features. Upregulated interferon signaling has been consistently described in both adult and juvenile DM which makes janus kinase inhibitors (jakinibs) an attractive therapeutic agent that has a targeted mechanism of action.

AREAS COVERED

Herein is a review of the growing literature of jakinib use in adult and juvenile DM, including reports on specific disease features and safety of jakinibs in this population and a comparison between adult and juvenile DM. We performed a literature review using PubMed including all English-language publications before 1 February 2024 and abstracts from key recent rheumatology conferences.

EXPERT OPINION

Jakinibs are an exciting and promising treatment in both adult and juvenile DM. Current Phase 2 and 3 randomized placebo-controlled trials of jakinibs in both adult and JDM will provide significant insights into the efficacy of this class of medication as a potentially more mechanistically targeted treatment of both skin and muscle disease. In fact, these results will likely inform the treatment paradigm of dermatomyositis in that it may even be considered as first or second line. The next five years in the therapeutic landscape of both juvenile and adult DM is an exciting time for both patients and medical providers.

摘要

引言

成人皮肌炎(DM)和青少年皮肌炎(JDM)是罕见的自身免疫性疾病,具有特征性皮疹、肌无力及其他全身表现。在成人和青少年DM中均持续发现干扰素信号上调,这使得 Janus 激酶抑制剂(jakinibs)成为一种具有靶向作用机制的有吸引力的治疗药物。

涵盖领域

本文综述了jakinib在成人和青少年DM中应用的不断增长的文献,包括关于jakinib在该人群中的特定疾病特征和安全性的报告,以及成人和青少年DM之间的比较。我们使用PubMed进行了文献综述,纳入了2024年2月1日前所有英文出版物以及近期主要风湿病学会议的摘要。

专家观点

Jakinibs在成人和青少年DM中都是令人兴奋且有前景的治疗方法。目前正在进行的jakinib在成人和青少年DM中的2期和3期随机安慰剂对照试验,将为这类药物作为对皮肤和肌肉疾病可能更具机制靶向性的治疗方法的疗效提供重要见解。事实上,这些结果可能会为皮肌炎的治疗模式提供参考,甚至可能被视为一线或二线治疗。在未来五年里,无论是青少年还是成人DM的治疗领域,对患者和医疗服务提供者来说都是令人兴奋的时期。

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