Bhimma Rajendra, Adhikari Miriam, Asharam Kareshma, Connolly Catherine
Department of Maternal and Child Health, Nelson R. Mandela School of Medicine, University of KwaZulu-Natal, Durban, South Africa.
Am J Nephrol. 2006;26(6):544-51. doi: 10.1159/000097864. Epub 2006 Dec 7.
The use of tacrolimus in steroid-resistant (SR) focal segmental glomerulosclerosis (FSGS) has been reported in single and small series case reports.
To determine the efficacy of tacrolimus in the management of SR FSGS in children.
This was a prospective study of 20 children with SR FSGS treated with tacrolimus (0.2-0.4 mg/kg/day in two divided doses over 12 h adjusted to a trough level between 7 and 15 ng/ml) for 12 months in combination with low-dose steroids. Other therapies included angiotensin-converting enzyme inhibitors, folic acid, multivitamins and lipid-lowering agents.
The mean age at study entry was 11.1 years (range 5.6-16.8). The mean duration of nephrotic syndrome before initiation of tacrolimus therapy was 4.7 years (range 2.1-7.6). At the end of the treatment period, 8 (40%) children were in complete remission, 9 (45%) were in partial remission, and 3 (15%) failed to respond. The average follow-up period following cessation of tacrolimus treatment was 27.5 months (range 13.7-43.7). At last hospital follow-up, 5 (25%) children were in complete remission, 10 (50%) in partial remission, and 2 (10%) in relapse. Three children died from dialysis-related complications following cessation of tacrolimus treatment. Adverse events included sepsis (2), nausea (2), diarrhea (2), anemia (4) and worsening of hypertension (4).
Tacrolimus is a safe and effective treatment for SR FSGS. However, like cyclosporine, some children tend to relapse following cessation of treatment.
在单个病例报告及小系列病例报告中，已有使用他克莫司治疗激素抵抗型（SR）局灶节段性肾小球硬化症（FSGS）的相关报道。
确定他克莫司治疗儿童SR FSGS的疗效。
这是一项前瞻性研究，对20例SR FSGS儿童患者使用他克莫司（0.2 - 0.4mg/kg/天，分两次给药，12小时内服用完毕，并根据谷浓度调整至7 - 15ng/ml）联合小剂量激素治疗12个月。其他治疗包括使用血管紧张素转换酶抑制剂、叶酸、多种维生素及降脂药物。
研究开始时的平均年龄为11.1岁（范围5.6 - 16.8岁）。在开始他克莫司治疗前，肾病综合征的平均病程为4.7年（范围2.1 - 7.6年）。治疗期结束时，8例（40%）儿童完全缓解，9例（45%）部分缓解，3例（15%）无反应。他克莫司治疗停止后的平均随访期为27.5个月（范围13.7 - 43.7个月）。在最后一次医院随访时，5例（25%）儿童完全缓解，10例（50%）部分缓解，2例（10%）复发。3例儿童在他克莫司治疗停止后死于透析相关并发症。不良事件包括败血症（2例）、恶心（2例）、腹泻（2例）、贫血（4例）及高血压加重（4例）。
他克莫司是治疗SR FSGS的一种安全有效的方法。然而，与环孢素一样，一些儿童在治疗停止后容易复发。
