Buratti S, Szer I S, Spencer C H, Bartosh S, Reiff A
Department of Pediatrics, University of Southern California, Keck School of Medicine, Childrens Hospital Los Angeles, 90027, USA.
J Rheumatol. 2001 Sep;28(9):2103-8.
To report the first clinical experience with mycophenolate mofetil (MMF, CellCept) in: children with lupus nephritis.
Eleven children with various forms of lupus nephritis were treated with oral MMF at a mean dose of 22 mg/kg/day (range 17-42) for a mean of 9.8 months (range 3-17). All children received concomitant prednisone and 7/11 were taking concomitant hydroxychloroquine. Indications for MMF included treatment refractory nephritis despite high dose oral or IV prednisone, azathioprine, and/or cyclophosphamide. Treatment outcome was monitored through assessment of Systemic Lupus Erythematosus Disease Activity Index (SLEDAI) score, renal function, and serologic markers such as complement and anti dsDNA antibodies.
While renal function normalized in 4/4 patients with membranous glomerulonephritis, little effect was observed in children with proliferative glomerulonephritis. Ten children experienced a marked reduction in SLEDAI score. Anti-dsDNA antibody and serum complement levels improved or remained stable in 80% of the children. Concomitant prednisone was decreased in 6/11 patients (55%) without deterioration of renal function. Adverse events, observed in 8 patients (73%), were not dose dependent, and included infections, leukopenia, nausea, pruritus, headache, and fatigue.
MMF may represent a valuable alternative to traditional cytotoxic agents for children with class V lupus nephritis, but was less effective in attenuating disease progression in class IV glomerulonephritis. MMF had a steroid sparing effect and appeared to be effective in controlling serologic disease activity in pediatric onset SLE. Adverse events such as infections may limit its use and remain a concern.
报告霉酚酸酯（MMF，骁悉）治疗儿童狼疮性肾炎的首例临床经验。
11例各种类型狼疮性肾炎患儿接受口服MMF治疗，平均剂量为22mg/kg/天（范围17 - 42mg/kg/天），平均治疗时间为9.8个月（范围3 - 17个月）。所有患儿均同时服用泼尼松，11例中有7例同时服用羟氯喹。MMF的用药指征包括尽管使用了高剂量口服或静脉注射泼尼松、硫唑嘌呤和/或环磷酰胺，但仍为治疗难治性肾炎。通过评估系统性红斑狼疮疾病活动指数（SLEDAI）评分、肾功能以及补体和抗双链DNA抗体等血清学标志物来监测治疗结果。
4例膜性肾小球肾炎患者中有4例肾功能恢复正常，而增殖性肾小球肾炎患儿疗效甚微。10例患儿的SLEDAI评分显著降低。80%的患儿抗双链DNA抗体和血清补体水平有所改善或保持稳定。11例中有6例（55%）患者的泼尼松用量减少，且肾功能未恶化。8例患者（73%）出现不良事件，这些不良事件与剂量无关，包括感染、白细胞减少、恶心、瘙痒、头痛和疲劳。
对于Ⅴ型狼疮性肾炎患儿，MMF可能是传统细胞毒性药物的一种有价值的替代药物，但在减轻Ⅳ型肾小球肾炎疾病进展方面效果较差。MMF具有激素节省作用，似乎对控制儿童期起病的系统性红斑狼疮的血清学疾病活动有效。感染等不良事件可能会限制其使用，仍是一个需要关注的问题。
