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治疗巨细胞动脉炎的研究性药物:评估未来发展的关键需求。

Investigational agents for polymyalgia rheumatica treatment: assessing the critical needs for future development.

机构信息

Rheumatology Unit, Department of Medicine DIMED, University of Padua, Padua, Italy.

Department of Rheumatology, Hospital of Bruneck (ASAA-SABES), Teaching Hospital of the Paracelsus Medical University, Brunico, Italy.

出版信息

Expert Opin Investig Drugs. 2024 Jul;33(7):671-676. doi: 10.1080/13543784.2024.2366847. Epub 2024 Jun 16.

Abstract

INTRODUCTION

Polymyalgia rheumatica (PMR) is an inflammatory rheumatic disorder characterized by pain and stiffness in the shoulder and pelvic girdles, constitutional symptoms, and elevated acute-phase reactants. Glucocorticoids (GCs) remain the first-choice treatment for PMR, but relapses are common. Identification of steroid-sparing agents is therefore of utmost importance.

AREAS COVERED

The efficacy of conventional immunosuppressive drugs is controversial. The use of interleukin (IL)-6 receptor inhibitors proved to be effective and safe in treating PMR patients. Currently, there are 12 ongoing clinical trials exploring potential treatments such as leflunomide, low-dose IL-2, rituximab, abatacept, secukinumab, Janus kinase inhibitors, and selective inhibitors like SPI-62 and ABBV 154.

EXPERT OPINION

The high efficacy of IL-6 R receptor inhibitors as well as the numerous drug trials currently recruiting suggest that several therapeutic options will be available in the near future. Accurate diagnosis and early stratification of PMR patients according to the giant cell arteritis-PMR Spectrum Disease 'GPSD' and potential risk factors for relapsing disease or GC-related adverse events are crucial to identify patients who would benefit most from GC-sparing agents. The development of internationally accepted definitions for remission and relapse is urgently needed. Early referral strategies to specialist settings would improve disease stratification and personalized treatment.

摘要

简介

巨细胞动脉炎-多发性肌痛症谱疾病(GPSD)是一种以肩部和骨盆带疼痛和僵硬、全身症状和急性期反应物升高为特征的炎性风湿性疾病。糖皮质激素(GCs)仍然是 PMR 的首选治疗方法,但复发很常见。因此,确定类固醇节约剂至关重要。

涵盖领域

传统免疫抑制剂的疗效存在争议。白细胞介素(IL)-6 受体抑制剂在治疗 PMR 患者方面已被证明是有效且安全的。目前,有 12 项正在进行的临床试验正在探索潜在的治疗方法,如来氟米特、低剂量 IL-2、利妥昔单抗、阿巴西普、司库奇尤单抗、Janus 激酶抑制剂和选择性抑制剂 SPI-62 和 ABBV 154。

专家意见

IL-6R 受体抑制剂的高疗效以及目前正在招募的众多药物试验表明,在不久的将来将有几种治疗选择。根据巨细胞动脉炎-多发性肌痛症谱疾病(GPSD)和潜在的复发性疾病或 GC 相关不良事件的风险因素,对 PMR 患者进行准确诊断和早期分层,对于确定最受益于 GC 节约剂的患者至关重要。迫切需要制定国际公认的缓解和复发定义。向专家机构的早期转诊策略将改善疾病分层和个体化治疗。

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