Ahmed Heba Mostafa
Beni- Suef University.
Iran J Kidney Dis. 2019 Sep;13(5):322-327.
Nephrotic syndrome (NS) is one of the commonest pediatric renal disorders. Most of these patients are steroid responsive. About 10%-20% of children with new onset NS are resistant to steroid treatment. Patients who are resistant to steroids have limited treatment options such as calcineurin inhibitors (CNIs), mycophenolate mofetil (MMF) and rituximab. Despite several studies had documented that tacrolimus is superior to cyclosporine A (CsA) and MMF in treating SRNS but there are no studies on the efficacy of tacrolimus in treating CsA and MMF resistant NS in pediatric populations. Study objective was to evaluate the role of tacrolimus in treating refractory idiopathic nephrotic syndrome .One hundred-twenty patients with idiopathic nephrotic syndrome were included in the study. Patients with steroid resistant NS were given cyclosporine (CsA) (first step protocol). In patients with cyclosporine resistant NS a combination of CsA+ MMF was given as a second step protocol. Unresponsive patients received tacrolimus as a third step treatment protocol. Tacrolimus was given at a starting dose of 0.1mg/kg/day then the dose was modified according to serum trough levels and patients were followed up for 12 months to evaluate the outcome. Out of 120 patients, 15 were both cyclosporine and MMF resistant and received tacrolimus. Tacrolimus had induced remission in 11 (73.3%) patients during the 1st 6 months of therapy. Eight patients achieved complete remission and three patients had partial remission.Conclusions: Tacrolimus is effective in treating refractory multi-drug resistant NS with favorable outcomes in childhood onset NS.
肾病综合征(NS)是最常见的儿童肾脏疾病之一。这些患者中的大多数对类固醇有反应。约10%-20%新诊断的NS患儿对类固醇治疗耐药。对类固醇耐药的患者治疗选择有限,如钙调神经磷酸酶抑制剂(CNIs)、霉酚酸酯(MMF)和利妥昔单抗。尽管多项研究表明他克莫司在治疗类固醇耐药肾病综合征(SRNS)方面优于环孢素A(CsA)和MMF,但尚无关于他克莫司治疗儿童CsA和MMF耐药NS疗效的研究。研究目的是评估他克莫司在治疗难治性特发性肾病综合征中的作用。本研究纳入了120例特发性肾病综合征患者。类固醇耐药NS患者给予环孢素(CsA)(第一步方案)。对于环孢素耐药的NS患者,第二步方案给予CsA+MMF联合治疗。无反应的患者接受他克莫司作为第三步治疗方案。他克莫司起始剂量为0.1mg/kg/天,然后根据血药谷浓度调整剂量,并对患者进行12个月的随访以评估结果。120例患者中,15例对环孢素和MMF均耐药,接受了他克莫司治疗。在治疗的前6个月,他克莫司使11例(73.3%)患者获得缓解。8例患者完全缓解,3例患者部分缓解。结论:他克莫司治疗难治性多药耐药NS有效,对儿童期发病的NS有良好疗效。