Safety and Efficacy of Rituximab in Severe Juvenile Dermatomyositis: Results from 9 Patients from the French Autoimmunity and Rituximab Registry
Safety and Efficacy of Rituximab in Severe Juvenile Dermatomyositis: Results from 9 Patients from the French Autoimmunity and Rituximab Registry
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DOI：
10.3899/jrheum.101321
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发表时间：
2011-07-01
影响因子：
3.9
通讯作者：
Bodemer, Christine
中科院分区：
文献类型：
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作者：
Bader-Meunier, Brigitte;Decaluwe, Helene;Bodemer, Christine
Objective.目的评价利妥昔单抗（RTX）治疗非试验期儿童皮肌炎（JDM）的安全性和有效性。我们对法国自身免疫和利妥昔单抗（AIR）注册的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治疗的主要适应症为重度和/或难治性肌肉受累（7例患者）、重度钙质沉着症（1例患者）或与腹部脂肪增多症相关的重度慢性腹痛（1例患者）。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例患者发生钙质沉着部位轻度感染，1例发生输注相关事件。在接受RTX治疗肌肉受累的3/6例患者中实现了完全临床缓解。在这些反应者中，类固醇治疗停止或逐渐减少至基线剂量的< 15%，无复发，随访时间为1.3至3年。6例患者的钙质沉着没有改善。这个小系列研究表明，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，其安全性也令人满意。需要进一步的研究来确定严重JDM患者对RTX反应的预测因素。（2011年6月15日首次发布; J Rheumol 2011;38：1436-40; doi：10.3899/jrheum.101321）
Objective. To evaluate the safety and efficacy of rituximab (RTX) in juvenile dermatomyositis (JDM) in off-trial patients.Methods. We conducted a multicenter prospective study of patients with JDM included in the French Autoimmunity and Rituximab (AIR) registry.Results. 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 I. 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.Conclusion. 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. (First Release June 15 2011; J Rheumatol 2011;38:1436-40; doi:10.3899/jrheum.101321)