沙利度胺治疗幼年特发性关节炎的效果分析

Efficacy of thalidomide for treatment of juvenile idiopathic arthritis

摘要:

目的：评价沙利度胺治疗幼年特发性关节炎（juvenile idiopathic arthritis，JIA）的临床疗效和安全性。方法：12例传统治疗方案治疗病情无缓解的JIA患儿，应用沙利度胺治疗（每日2 mg/kg），记录治疗前后患儿的症状、体征、实验室指标及沙利度胺治疗的不良反应。结果：沙利度胺治疗6个月后，患儿泼尼松用量由每日1.92±0.16 mg/kg 降至0.49±0.42 mg/kg (P<0.01)，并有4例能停用泼尼松治疗。12例患儿外周血白细胞计数、ESR、CRP、血清铁蛋白（SF）较治疗前均明显下降（P<0.01）； 8例患儿血红蛋白明显升高(P<0.01)。多关节型JIA患儿平均受累关节数由5个以上降至0～2个（P<0.01），附着点炎症型患儿双“4”字征转阴，Schober′s征转阴。治疗过程中未出现严重不良反应。结论：沙利度胺对JIA有显著的临床疗效，可以作为传统治疗方案无效后的治疗措施。

Abstract:

OBJECTIVE: To evaluate the efficacy of thalidomide in the treatment of juvenile idiopathic arthritis (JIA). METHODS: Twelve children with JIA who did not respond to conventional treatment were administered with thalidomide (2 mg/kg daily). The symptoms, signs, and laboratory test results were compared before and after treatment. The thalidomide-related side effects were observed. RESULTS: The average dosage of prednisone was reduced from 1.92±0.16 mg/kg?d to 0.49±0.42 mg/kg?d in the 12 patients 6 months after thalidomide treatment (P<0.01). Four patients did not need prednisone treatment any more. White blood cell count, erythrocyte sedimentation rate (ESR), C reactive protein (CRP) and serum ferritin (SF) significantly decreased after treatment in all of 12 patients (P<0.01). Hemoglobin level increased to normal in 8 patients after treatment (P<0.01). The number of affected joints decreased from 5 before treatment to zero to 2 after treatment in patients with polyarticular JIA (P<0.01). Signs of hip involvement and Schober′s sign turned negative in enthesitis-related cases. No thalidomide-related side effects were observed. CONCLUSIONS: Thalidomide is effective in the treatment of JIA in children who do not respond to conventional treatment.