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目的探索用联合大剂量免疫抑制剂进行强免疫清除,但保留骨髓造血功能而无需造血干细胞移植(SCT)的方法,治疗儿童难治性系统性红斑狼疮(SLE)的疗效。方法2004-08—2005-04对中山大学附属第一医院儿科收治的难治性SLE5例进行二种方案的疗效分析。5例均有肾损害,其中4例肾病理WHO-Ⅳ型和大量蛋白尿,2例免疫性溶血性贫血,1例免疫性血小板减少,均曾用包括环磷酰胺(CTX)和甲基泼尼松龙双冲击等治疗无效或病情反复无法控制。4例给予方案1治疗,另1例因严重水肿和尿少不宜用大剂量CTX,给予方案2治疗。疗效用SLE疾病活动性指数记分(SLEDAI)评估。结果方案1从CTX结束算起,中性粒细胞恢复到0.5×109/L平均需10d,方案2从Ara-C结束起,中性粒细胞恢复到0.5×109/L需16d;治疗3周时SLEDAI平均从9.2降至4.4,尿蛋白从(++++)降至(0～+),合并有重度难治性贫血或血小板减少者,治疗后血红蛋白或血小板分别恢复正常或接近正常;方案2治疗的1例由于肾血管血栓形成未能控制,病情短暂好转后再度恶化,1个月后死于DIC和肾功能衰竭;另4例随访5～16个月,病情基本控制。结论控制在一定范围内的大剂量免疫抑制剂治疗SLE,无需SCT而能恢复自身造血,同时能相当大程度清除病态免疫系统,使常规治疗无效者病情缓解,值得进一步探索。
Objective To explore the treatment of refractory systemic lupus erythematosus (SLE) in children with intensive immunosuppression combined with large doses of immunosuppressive agents, but without the need for hematopoietic stem cell transplantation (SCT) to preserve the hematopoietic function of bone marrow. Methods From 2004-08 to 2005-04, the curative effects of two kinds of regimens on refractory SLE5 cases admitted to the First Affiliated Hospital of Sun Yat-sen University were analyzed. All of the 5 cases had renal damage, including 4 cases of WHO-Ⅳ type and large amount of albuminuria, 2 cases of immune hemolytic anemia and 1 case of immune thrombocytopenia. Both of them included CTX and methylprednisolone Nixon double impact and other treatment is invalid or the disease can not be controlled repeatedly. 4 cases given program 1 treatment, and the other case due to severe edema and oligomatous should not use large doses of CTX given program 2 treatment. Efficacy was assessed using the SLE Disease Activity Index Score (SLEDAI). Results From the end of CTX, neutrophil recovery to 0.5 × 109 / L an average of 10d, the program from the end of Ara-C, neutrophils recovered to 0.5 × 109 / L required 16d; treatment for 3 weeks When SLEDAI decreased from 9.2 to 4.4 on average, urinary protein decreased from (++++) to (+0 +) with severe refractory anemia or thrombocytopenia. After treatment, hemoglobin or platelet returned to normal or nearly normal respectively. One case treated with regimen 2 failed to control because of renovascular thrombosis, and the condition deteriorated again after a brief improvement. One month later died of DIC and renal failure. The other four cases were followed up for 5 to 16 months and the disease was basically controlled. CONCLUSIONS: SLE controlled by a large dose of immunosuppressive agents within a certain range can recover its own hematopoiesis without SCT, and can clear the pathological immune system to a considerable extent, so as to ease the condition of ineffective conventional therapy and worth further exploration.