Department of Pediatric Immunology and Rheumatology, Paris Descartes University, AP-HP, Necker Hospital, Paris, France.
J Rheumatol. 2011 Jul;38(7):1436-40. doi: 10.3899/jrheum.101321. Epub 2011 Jun 15.
To evaluate the safety and efficacy of rituximab (RTX) in juvenile dermatomyositis (JDM) in off-trial patients.
We conducted a multicenter prospective study of patients with JDM included in the French Autoimmunity and Rituximab (AIR) registry.
Nine patients with severe JDM were studied. The main indication for RTX treatment was severe and/or refractory muscle involvement (7 patients), severe calcinosis (1 patient), or severe chronic abdominal pain associated with abdominal lipomatosis (1 patient). RTX was associated with corticosteroids, immunosuppressive drugs, and plasma exchange therapy in 9/9, 5/9, and 2/9 patients, respectively. Mild infections of the calcinosis sites occurred in 2 patients and an infusion-related event in 1. Complete clinical response was achieved in 3/6 patients treated with RTX for muscle involvement. In these responders steroid therapy was stopped or tapered to < 15% of the baseline dosage, with no relapse, with a followup ranging from 1.3 to 3 years. Calcinosis did not improve in the 6 affected patients.
This small series suggests that rituximab may be effective for treating muscle and skin involvement in a small subset of children with severe JDM, and that its safety profile was satisfactory. Further studies are needed to identify predictive factors of response to RTX in patients with severe JDM.
评估利妥昔单抗（RTX）在非试验患者中治疗幼年皮肌炎（JDM）的安全性和疗效。
我们对纳入法国自身免疫和利妥昔单抗（AIR）登记处的 JDM 患者进行了一项多中心前瞻性研究。
研究了 9 例严重 JDM 患者。RTX 治疗的主要适应证为严重和/或难治性肌肉受累（7 例）、严重钙沉积症（1 例）或严重慢性腹痛伴腹部脂肪瘤（1 例）。RTX 分别与 9/9、5/9 和 2/9 例患者的皮质类固醇、免疫抑制剂和血浆置换治疗相关。2 例钙沉积部位发生轻度感染，1 例发生输注相关事件。6 例接受 RTX 治疗肌肉受累的患者中有 3 例达到完全临床缓解。在这些应答者中，类固醇治疗被停止或减少至基线剂量的<15%，无复发，随访时间为 1.3 至 3 年。6 例受影响的患者钙沉积没有改善。
这项小系列研究表明，利妥昔单抗可能对治疗一小部分严重 JDM 儿童的肌肉和皮肤受累有效，其安全性良好。需要进一步研究以确定严重 JDM 患者对 RTX 反应的预测因素。
