甲泼尼龙和环磷酰胺冲击治疗儿童重症系统性红斑狼疮的临床研究

目的　了解甲泼尼龙 (MP)和环磷酰胺 (CTX)冲击治疗儿童重症系统性红斑狼疮(SeverJOSLE)的临床效果和转归。方法　确诊的儿童系统性红斑狼疮 30例 ,初治 2 2例 ,其中狼疮性肾炎 (LN) 2 0例 ,9例纳入前病程 3～ 1 2个月。 30例患儿中 ,2 3例有肾损伤的临床表现 ,其中 ,WHO Ⅳ型 4例 ,WHO Ⅱ型 2例 ,WHO Ⅴ型 1例 ,WHO Ⅲ型 1例 ,7例肾损伤同时伴 1项或以上的神经系统损伤的临床表现 :舞蹈症、癫样发作、脑血管意外 (CAV)和器质性脑综合征 (OBS)等 ;有神经系统症状不伴肾损伤临床表现的 9例 ;3例狼疮危象 (lupuscrisis)。其余 7例不伴肾损伤临床表现 ,仅有轻微或一过性中枢神经系统损伤。根据治疗方法分为 3组 :A组 ,1 8例 ,临床或病理证实合并LN或精神神经性狼疮 (NPLE) ,采用MP +CTX冲击治疗 ;B组 ,7例 ,临床无LN和 (或 )NPLE ,MP冲击诱导后 ,小剂量泼尼松维持治疗 ,并以雷公藤多甙 (Twhf)接续治疗 ;C组 ,5例 ,临床以LN为主 ,糖皮质激素 (GS)治疗。观察各组的近期、远期疗效和远期临床转归。结果　平均随访时间为 (37 2± 2 4 8)个月。近期疗效 :A组与B组间SLEDAI 2K的差别主要是肾脏权重计分 ,9个月时 ,A组的变化最大 ,计分与B组相似 ,C组SLEDAI 2K计分在中度活动范围 ,主要是肾脏的权重结果。远期

Methylprednisolone and cyclophosphamide pulse therapy of severe systemic lupus erythematosus in children

OBJECTIVE: To study the effect of methylprednisolone (MP) and cyclophosphamide (CPA) intermittent intravenous pulse therapy and the clinical prognosis in children with severe juvenile onset systemic lupus erythematosus (JOSLE). METHODS: Thirty patients with JOSLE, diagnosed by clinical, laboratory or renal histological examinations, were enrolled in this study. Of the 30 patients, 27 were females and 3 were males, the mean age was (12 +/- 3) years, and 20 of the 22 patients who had undergone initial therapy had LN, and the clinical courses before being involved in the study were 3 to 12 months in nine patients. Twenty-three of the 30 patients had clinical manifestations of renal damages, of whom 4 patients were proven by initial renal biopsy to have WHO type IV, 2 had type II,1 had type V and 1 had type III, and 7 patients had one or more manifestations of central nervous system, including chorea, seizures, cerebrovascular accident (CVA) and organic brain syndrome (OBS), simultaneously, 9 patients had nervous system symptoms without the clinical manifestations of renal damages, 3 patients had lupus crisis, 7 patients did not have any manifestations of renal or neurological damages. According to the protocol of the therapy, the patients were divided into 3 groups: group A (n = 18) patients were treated with MP plus CPA intermittent intravenous pulse for children with lupus nephritis, and with or without neuropsychiatric lupus erythematosus (NPLE), group B (n = 7) with pulsed doses of MP, followed by prednisone and tripterygium wilfordii hook f(T(whf)) for patients without renal or central nerves system damage, and group C (n = 5) with prednisone alone for patients with LN determined by clinical and laboratory features. The effects of those regimes and the clinical prognosis were observed. RESULTS: On short-term follow-up, the SLEDAI-2K (by weight of the renal damage) showed significant difference between group A and group B, but there was no significant difference at the 9th months of the therapy. The long-term follow-up lasted in average for (37.2 +/- 24.8) months. Nineteen patients were followed up for more than 18 months. At the end of follow-up, the mean age was 14 to 19 years. There was no difference on the effect of both group A and group B, and no frequent infections were seen, ANAs were negative and SLEDAI-2K = 0-point in two patients of each group 12 months after discontinuation of the therapy. Four patients in group C died within 18 months. CONCLUSION: The immunosuppressive regimen MP + CPA in patients with severe JOSLE and MP + prednisone + T(whf) in patients without major organs damage were superior to the regimen of prednisone alone.