Supavekin Suroj, Surapaitoolkorn Wantanee, Kurupong Thitima, Chaiyapak Thanaporn, Piyaphanee Nuntawan, Pattaragarn Anirut, Sumboonnanonda Achra
Department of Pediatrics, Faculty of Medicine Siriraj Hospital, Mahidol University, Bangkok, Thailand.
J Med Assoc Thai. 2013 Jan;96(1):33-40.
To evaluate the efficacy of tacrolimus (Tac) in steroid resistant and steroid dependent nephrotic syndrome (NS) in children.
Retrospective chart reviews of 18 children from outpatient clinic at the Department of Pediatrics, Faculty of Medicine Siriraj Hospital were diagnosed with steroid resistant (SR) and steroid dependent (SD) NS during 2002-2008 were enrolled in the present study
The boy to girl ratio was 2:1. The mean age at diagnosis was 6.0 years (1-14.4 years). There were nine SR and nine SDNS. Nine patients had focal segmental glomerulosclerosis (FSGS), 4 IgMnephropathy and two had minimal change diseases (MCD). Three children did not receive renal biopsy. All patients received prednisolone at the start of Tac. The average time from the diagnosis to initiation of Tac was 3.5 years (0.2-14years). The mean duration of Tac treatment was 1.3 year (0.3-6.2 years). The average Tac trough blood level was 4.09 mcg/L (1.3-9.9 mcg/L). The average dosage of Tac was 0.09 mg/kg/day (0.03-0.2 mg/kg/day). Thirteen (72.2%) children achieved complete response (CR). Five (27.80%) children did not respond to Tac. Nine (69.2%) children could stop prednisolone whereas four (30.8%) could lower prednisolone doses. The mean time to achieve CR was 24.6 days (0.1-3 months). The mean follow up period was 3.1 years (0.2-6.4 years). There was no change in an estimation of glomerular filtration rate (eGFR). In SRNS, there were CR in four (44.4%) and five (55.6%) children that FSGS did not respond to Tac. In SDNS, all responded to Tac and four (44.4%) children relapsed while on Tac and had upper respiratory tract infection (URI).
Tac is well-tolerated and effective treatment for SR and SDNS.
评估他克莫司（Tac）治疗儿童激素抵抗型和激素依赖型肾病综合征（NS）的疗效。
对诗里拉吉医院医学院儿科学门诊的18例在2002年至2008年间被诊断为激素抵抗型（SR）和激素依赖型（SD）NS的儿童进行回顾性病历审查，并纳入本研究。
男女比例为2:1。诊断时的平均年龄为6.0岁（1 - 14.4岁）。有9例SR和9例SDNS。9例患者患有局灶节段性肾小球硬化（FSGS），4例为IgM肾病，2例为微小病变病（MCD）。3名儿童未接受肾活检。所有患者在开始使用Tac时均接受泼尼松龙治疗。从诊断到开始使用Tac的平均时间为3.5年（0.2 - 14年）。Tac治疗的平均持续时间为1.3年（0.3 - 6.2年）。Tac的平均谷血浓度为4.09 mcg/L（1.3 - 9.9 mcg/L）。Tac的平均剂量为0.09 mg/kg/天（0.03 - 0.2 mg/kg/天）。13名（72.2%）儿童达到完全缓解（CR）。5名（27.80%）儿童对Tac无反应。9名（69.2%）儿童可以停用泼尼松龙，而4名（30.8%）儿童可以降低泼尼松龙剂量。达到CR的平均时间为24.6天（0.1 - 3个月）。平均随访期为3.1年（0.2 - 6.4年）。肾小球滤过率（eGFR）估计值无变化。在SRNS中，4名（44.4%）儿童达到CR，5名（55.6%）FSGS儿童对Tac无反应。在SDNS中，所有患者对Tac均有反应，4名（44.4%）儿童在使用Tac期间复发并患有上呼吸道感染（URI）。
Tac对SR和SDNS是耐受性良好且有效的治疗方法。
