Department of Pediatrics, Division of Pediatric Rheumatology, Hacettepe University Faculty of Medicine, Ankara, Turkey.
Clin Rheumatol. 2024 Feb;43(2):591-602. doi: 10.1007/s10067-023-06740-3. Epub 2023 Aug 16.
There is no clear consensus in the literature regarding the choice of biologic therapies and efficacy in juvenile dermatomyositis (JDM). In this review, we aimed to examine previous studies regarding biologic drug use in JDM patients. We screened MEDLINE and Scopus for articles involving JDM patients treated with biologic drugs. We identified 74 articles describing 495 JDM patients treated with biologic drugs (538 biologic treatments) during our literature search. The median (min-max) age of these patients was 9.8 (1-17) years (F/M:1.8). The most frequently used biologic drugs were rituximab (RTX, 50%) and tumor necrosis factor (TNF) inhibitors (34.8%). In a few cases, abatacept (4.3%), anti-interleukin-1 agents (0.9%), tocilizumab (0.9%), bortezomib (0.4%), ustekinumab (0.2%), eculizumab (0.2%), and golimumab (0.2%) were used. RTX was most frequently preferred in patients with severe skin involvement (46.3%). Improvement with RTX was obtained in 60.1% of RTX treatments. Infliximab was most frequently preferred in calcinosis (43.3%), while adalimumab in skin involvement (50%) and etanercept in resistant/recurrent diseases (80%). Improvement was achieved in 44.4% of anti-TNF treatments. Adverse events were observed in 46.8% (58/124) of all treatments. Our results suggest that biologic agents may be a promising alternative for the treatment of particularly resistant JDM cases. Controlled studies are required to provide higher level of evidence for the timing of biologic use in JDM treatment. Key Points • There is no consensus on the choice and efficacy of biologic therapies in JDM. • RTX and TNF inhibitors are the most commonly used biologic drugs. • Biologics were especially preferred in severe skin involvement, calcinosis, and resistant diseases.
目前，对于幼年特发性关节炎（JDM）患者的生物治疗选择和疗效，文献中尚无明确共识。在本综述中，我们旨在研究 JDM 患者使用生物药物的既往研究。我们在 MEDLINE 和 Scopus 中筛选了涉及使用生物药物治疗 JDM 患者的文章。在文献检索中，我们共发现 74 篇描述 495 例 JDM 患者接受生物药物治疗（538 次生物治疗）的文章。这些患者的中位（最小-最大）年龄为 9.8 岁（1-17 岁）（男/女：1.8）。最常使用的生物药物是利妥昔单抗（RTX，50%）和肿瘤坏死因子（TNF）抑制剂（34.8%）。在少数情况下，使用了阿巴西普（4.3%）、抗白细胞介素-1 制剂（0.9%）、托珠单抗（0.9%）、硼替佐米（0.4%）、乌司奴单抗（0.2%）、依库珠单抗（0.2%）和戈利木单抗（0.2%）。RTX 最常用于有严重皮肤受累的患者（46.3%）。RTX 治疗的 60.1%获得改善。英夫利昔单抗最常用于钙质沉着症（43.3%），阿达木单抗最常用于皮肤受累（50%），依那西普最常用于耐药/复发疾病（80%）。抗 TNF 治疗的 44.4%获得改善。所有治疗中观察到 46.8%（58/124）出现不良反应。我们的结果表明，生物制剂可能是治疗特别耐药 JDM 病例的一种有前途的选择。需要进行对照研究，为 JDM 治疗中生物制剂使用的时机提供更高水平的证据。关键要点 • 对于 JDM 患者的生物治疗选择和疗效，目前尚无共识。 • RTX 和 TNF 抑制剂是最常用的生物药物。 • 生物制剂尤其常用于严重皮肤受累、钙质沉着症和耐药疾病。
