Novak Inna, Frank Rachel, Vento Suzanne, Vergara Marcela, Gauthier Bernard, Trachtman Howard
Department of Pediatrics, Division of Nephrology, Schneider Children's Hospital of the North Shore-Long Island Jewish Health System, 269-01 76th Ave., New Hyde Park, NY 11040, USA.
Pediatr Nephrol. 2005 Sep;20(9):1265-8. doi: 10.1007/s00467-005-1957-y. Epub 2005 Jun 8.
Most patients with minimal change nephrotic syndrome are steroid responsive and tolerate this medication. However, a substantial number of patients relapse frequently and become steroid dependent. These patients often require treatment with alternative immunosuppressive drugs to maintain remission and minimize steroid toxicity. Previous studies have suggested that mycophenolate mofetil is effective in treating these patients. However, there are limited data on the effectiveness of this agent in pediatric patients, specifically those with steroid-dependent nephrotic syndrome. The purpose of this study was to assess the efficacy and safety of mycophenolate mofetil therapy in children and adolescents with steroid-dependent nephrotic syndrome who failed other treatments. A retrospective chart review was performed on all patients with steroid-dependent nephrotic syndrome. Clinical characteristics, laboratory data and the relapse rate were assessed prior to and during mycophenolate mofetil treatment. Twenty-one patients, ages 2-17 years, with steroid-dependent nephrotic syndrome who were treated with mycophenolate mofetil between 2001-2005 were included in this review. The indication for mycophenolate mofetil use was steroid dependence in 17 and steroid toxicity in 4 patients. The mean duration of treatment was 1.0+/-0.5 years (range: 0.2-2.0 years). Patients treated with mycophenolate mofetil had a reduction in relapse rate from 0.80+/-0.41 to 0.47+/-0.43 relapses per month ( P <0.02). Side effects were mild and mostly gastrointestinal in nature. In 1 child, mycophenolate mofetil was discontinued due to varicella infection and not restarted. The findings indicate that mycophenolate mofetil is a useful adjunctive therapy in the treatment of patients with steroid-dependent nephrotic syndrome. It lowers the relapse rate by 40% and is well tolerated by patients with steroid-dependent nephrotic syndrome.
大多数微小病变型肾病综合征患者对类固醇药物有反应且能耐受该药物治疗。然而,相当一部分患者频繁复发并出现类固醇依赖。这些患者常需使用其他免疫抑制药物进行治疗,以维持病情缓解并将类固醇毒性降至最低。既往研究提示,霉酚酸酯对治疗这些患者有效。然而,关于该药物在儿科患者,尤其是类固醇依赖型肾病综合征患者中的有效性数据有限。本研究的目的是评估霉酚酸酯治疗对其他治疗无效的类固醇依赖型肾病综合征儿童及青少年的疗效和安全性。对所有类固醇依赖型肾病综合征患者进行了回顾性病历审查。在霉酚酸酯治疗前及治疗期间评估了临床特征、实验室数据及复发率。本回顾纳入了2001年至2005年间接受霉酚酸酯治疗的21例年龄在2至17岁的类固醇依赖型肾病综合征患者。使用霉酚酸酯的指征为17例患者存在类固醇依赖,4例患者存在类固醇毒性。平均治疗时长为1.0±0.5年(范围:0.2至2.0年)。接受霉酚酸酯治疗的患者复发率从每月0.80±0.41次降至0.47±0.43次(P<0.02)。副作用较轻,主要为胃肠道反应。1名儿童因水痘感染停用霉酚酸酯且未再重新使用。研究结果表明,霉酚酸酯是治疗类固醇依赖型肾病综合征患者的一种有用的辅助治疗药物。它可使复发率降低40%,且类固醇依赖型肾病综合征患者对其耐受性良好。