关键词: 系统性红斑狼疮, 难治性, 治疗, 儿童

Abstract: AbstractObjectiveIn this trial we investigated the safety and efficacy of nonmyeloablative but intense immunoablative chemotherapy without SCT in the treatment of childhood refractory SLE. MethodsFive refractory SLE patients were female aged 10~16 at the study，4 with WHO class Ⅳ nephritis，2 with immune hemolytic anemia and 1 with immune thrombocytopenia.Previous therapy including monthly I.V.cyclophasphamide (CTX) and high -dose methylprednisolone failed for the 5 cases.Among these cases,4 received regimen A and 1 received regimen B.The disease activity was measured by SLEDAI before and after the treatment. ResultsThere was no treatment-related death.The median time to achieve a neutrophile count of 0.5×109/L was 10 days after the last dose of chemotherapy in regimen A,and the time was 16 days in regimen B.The average SLEDAI decreased from 9.2 to 4.4,and the proteinuria decreased from (++++) to (0~+) in 3 weeks after the treatment.Two cases with complication of hemolytic anemia and 1 with thrombocytopenia before the treatment recovered to normal or nearly normal hemogram respectively.Among these 5 cases,1 patient had a short time improvement during the therapy,but died 50 days later because of refractory thrombosis of renal vein; 4 other patients achieved complete or partial remission within 5~16 months follow-up. ConclusionDose-controlled high-dose chemotherapy leads to hemotopoietic reconstitution without SCT and has significantly clinical benefit in children with refractory SLE by immunoablation.Therefore this approach deserves further study.

Key words: Children , Refractory, Therapy