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利妥昔单抗治疗儿童期起病的系统性红斑狼疮

Rituximab therapy for childhood-onset systemic lupus erythematosus.

作者信息

Willems M, Haddad E, Niaudet P, Koné-Paut I, Bensman A, Cochat P, Deschênes G, Fakhouri F, Leblanc T, Llanas B, Loirat C, Pillet P, Ranchin B, Salomon R, Ulinski T, Bader-Meunier Brigitte

机构信息

Department of Pediatrics, Hôpital de Bicêtre, Le Kremlin Bicêtre, France.

出版信息

J Pediatr. 2006 May;148(5):623-627. doi: 10.1016/j.jpeds.2006.01.041.

DOI:10.1016/j.jpeds.2006.01.041
PMID:16737873
Abstract

OBJECTIVE

To describe the safety and efficacy of rituximab in the treatment of childhood-onset systemic lupus erythematosus (SLE).

STUDY DESIGN

We conducted a French multicenter retrospective study of childhood-onset SLE treated with rituximab.

RESULTS

Eleven girls with severe SLE, including 8 girls with class IV or V lupus nephritis, 2 girls with severe autoimmune cytopenia, and 1 girl with antiprothrombin antibody with severe hemorrhage, were treated with rituximab. The mean age at onset of rituximab treatment was 13.9 years. Patients received 2 to 12 intravenous infusions of rituximab (350-450 mg/m2/infusion), with corticosteroids. Six patients also received different standard immunosuppressive agents, including Cyclophosphamide (2 patients). Remission was achieved in 6 of 8 patients with lupus nephritis and in the 2 patients with autoimmune cytopenia. Steroid therapy was tapered in 5 patients who responded to treatment, and low-dose prednisone treatment was maintained in 1 patient. The mean follow-up period was 13.2 months (range, 6-26 months), and remission lasted in all who patients who responded to treatment, except 1 patient who was successfully retreated with a second course of rituximab. Anti-double-stranded DNA antibody levels decreased in 6 of 11 patients, and anticardiolipin antibody levels decreased in 3 of 4 patients. Severe adverse events developed in 5 patients. Effective depletion of peripheral blood B cells was observed in 7 of 8 patients who were examined, and this paralleled the remission.

CONCLUSION

Rituximab may be an effective co-therapy; however, further investigations are required because severe adverse events occurred in 45% of the patients in this study.

摘要

目的

描述利妥昔单抗治疗儿童期系统性红斑狼疮(SLE)的安全性和有效性。

研究设计

我们对接受利妥昔单抗治疗的儿童期SLE进行了一项法国多中心回顾性研究。

结果

11名患有严重SLE的女孩接受了利妥昔单抗治疗,其中包括8名患有IV或V级狼疮性肾炎的女孩、2名患有严重自身免疫性血细胞减少症的女孩以及1名患有抗凝血酶原抗体并伴有严重出血的女孩。利妥昔单抗治疗开始时的平均年龄为13.9岁。患者接受了2至12次利妥昔单抗静脉输注(350 - 450 mg/m²/次),同时使用了皮质类固醇。6名患者还接受了不同的标准免疫抑制剂治疗,包括环磷酰胺(2名患者)。8名狼疮性肾炎患者中有6名以及2名自身免疫性血细胞减少症患者实现了缓解。5名对治疗有反应的患者逐渐减少了类固醇治疗,1名患者维持低剂量泼尼松治疗。平均随访期为13.2个月(范围为6 - 26个月),除1名患者成功接受第二个疗程的利妥昔单抗再次治疗外,所有对治疗有反应的患者均持续缓解。11名患者中有6名抗双链DNA抗体水平下降,4名患者中有3名抗心磷脂抗体水平下降。5名患者出现了严重不良事件。在接受检查的8名患者中有7名观察到外周血B细胞有效耗竭,这与缓解情况平行。

结论

利妥昔单抗可能是一种有效的联合治疗药物;然而,由于本研究中45%的患者发生了严重不良事件,因此需要进一步研究。

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