OBJECTIVETo 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).

METHODSThirty 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.

RESULTSOn 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.

CONCLUSIONThe 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.