Shishov Michael, Weiss Pamela F, Levy Deborah M, Chang Joyce C, Angeles-Han Sheila T, Ogbu Ekemini A, Nanda Kabita, Sherrard Tina M, Goldmuntz Ellen, Lovell Daniel J, Rider Lisa G, Brunner Hermine I
Phoenix Children's Hospital, Phoenix, Arizona.
The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Arthritis Care Res (Hoboken). 2025 May;77(5):573-583. doi: 10.1002/acr.25482. Epub 2025 Jan 30.
There are over 100 rheumatic diseases and approximately 300,000 children with a pediatric rheumatic disease (PRD) in the United States. The most common PRDs are juvenile idiopathic arthritis (JIA), childhood-onset systemic lupus erythematosus (cSLE), and juvenile dermatomyositis (JDM). Effective and safe medications are essential because there are generally no cures for these conditions. Etanercept was the first biologic therapy for the treatment of JIA, approved in 1999. Since then, other biologic disease-modifying antirheumatic drugs (bDMARDs) and targeted synthetic disease-modifying antirheumatic drugs (tsDMARDs) blocking relevant immunologic pathways have been approved for the treatment of JIA, resulting in a marked improvement of disease prognosis. Conversely, there is only one bDMARD that has been approved for cSLE, but none are approved for the treatment of JDM. Lack of approved therapeutic options, with established dosing regimens and known efficacy and safety, remains a central challenge in the treatment of all PRDs, including autoinflammatory diseases, and for complications of PRDs. This review provides an overview of bDMARD and tsDMARD treatments studied for the treatment of various subtypes of JIA, summarizes information from bDMARD studies in other PRDs, with a focus on pivotal trials that led to regulatory approvals, and highlights improved outcomes in patients with JIA with the reception of these newer medications. Further, we outline barriers and challenges in the treatment of other PRDs. Last, we summarize the current regulatory landscape for bDMARD studies and medication approvals for patients with PRDs.
美国有100多种风湿性疾病，约30万名儿童患有儿科风湿性疾病（PRD）。最常见的PRD是幼年特发性关节炎（JIA）、儿童期起病的系统性红斑狼疮（cSLE）和幼年皮肌炎（JDM）。由于这些疾病通常无法治愈，有效且安全的药物至关重要。依那西普是1999年获批的首个用于治疗JIA的生物疗法。从那时起，其他阻断相关免疫途径的生物性改善病情抗风湿药（bDMARD）和靶向合成改善病情抗风湿药（tsDMARD）已获批用于治疗JIA，使疾病预后有了显著改善。相反，只有一种bDMARD获批用于cSLE的治疗，但尚无获批用于JDM治疗的药物。缺乏已确立给药方案、已知疗效和安全性的获批治疗选择，仍然是包括自身炎症性疾病在内的所有PRD及其并发症治疗中的核心挑战。本综述概述了针对JIA各亚型治疗所研究的bDMARD和tsDMARD治疗方法，总结了其他PRD中bDMARD研究的信息，重点关注导致监管批准的关键试验，并强调接受这些新药的JIA患者的预后改善情况。此外，我们概述了其他PRD治疗中的障碍和挑战。最后，我们总结了目前PRD患者bDMARD研究和药物批准的监管情况。
