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Abstract：
Background: Focal segmental glomerulosclerosis (FSGS) causes end stage renal disease (ESRD) in significant proportion of patients worldwide. Primary FSGS carries poor prognosis and management of FSGS patients, refractory to standard treatments or resistant to steroids, remains a major challenge. Lipoprotein apheresis is a therapeutic approach for drug resistant primary FSGS and post-renal transplant primary FSGS recurrence. Objectives: To examine the safety and probable benefit at 1, 3, 6, 12, and 24-months following completion of apheresis treatment using Liposorber® LA-15 system in patients with nephrotic syndrome (NS), due to refractory primary FSGS or primary FSGS associated NS, in post renal transplant children. Material and Methods: Prospective, multicenter, single-arm intervention study using Liposorber® LA-15 system. Patients ≤21 years old with drug resistant or drug intolerant NS secondary to primary FSGS with glomerular filtration rate (GFR) ≥60 ml/min/1.73 m2 or post renal transplant patients ≤21 years old with primary FSGS associated NS were included in the study. Each patient had 12 dextran-sulfate plasma adsorption lipoprotein apheresis sessions over a period of 9 weeks. All patients were followed up at 1, 3, 6, 12, and 24-months following completion of treatment. Results: Of 17 patients enrolled, six were excluded from the outcome analysis (protocol deviations). Of the remaining 11 patients, all but one have completed apheresis treatments. Three patients were lost to follow-up immediately after completion of apheresis and excluded from outcome analysis. At one-month follow-up, 1 of 7 patients (14.3%) attained partial remission of NS while 2 of 4 subjects (50%) and 2 of 3 subjects (66.7%) had partial/complete remission at 3- and 6-months follow-up, respectively. One of two patients followed up for 12 months had complete remission and one patient had partial remission of NS after 24 months. Improved or stable eGFR was noted in all patients over the follow-up period. Conclusion: The results of our multicenter study showed improvement in the response rates to steroid or immunosuppressive therapy and induced complete or partial remission of proteinuria in some of the patients with drug resistant primary FSGS. The main limitation of our study is the small number of subjects and high dropout rate.
摘要：
背景：局灶性节段性肾小球硬化（FSGS）在全球相当大比例的患者中导致终末期肾病（ESRD）。原发性FSGS患者的预后和管理较差，标准治疗难以治疗或对类固醇有抗性，仍然是一个重大挑战。脂蛋白单采术是耐药原发性FSGS和肾移植后原发性FSGS复发的治疗方法。目标：在肾病综合征（NS）患者使用Liposorber®LA-15系统完成单采治疗后的1、3、6、12和24个月，检查安全性和可能的益处，由于主要FSGS或主要FSGS相关NS，肾移植后的儿童。材料和方法：前瞻性，多中心，使用Liposorber®LA-15系统进行单臂干预研究。本研究包括年龄≤21岁的原发性FSGS继发耐药或不耐受NS且肾小球滤过率（GFR）≥60ml/min/1.73m2的患者，或年龄≤21岁的原发性FSGS相关NS的肾移植后患者。每位患者在9周内进行了12次硫酸葡聚糖血浆吸附脂蛋白单采术。所有患者均在治疗完成后1、3、6、12和24个月进行随访。结果：在17名患者中，结果分析中排除了6例(方案偏差).剩下的11个病人,除了一个人之外，所有的人都完成了单采治疗。三名患者在单采手术完成后立即失去随访，并被排除在结果分析之外。在一个月的随访中，在3个月和6个月的随访中，7例患者中有1例（14.3％）达到NS部分缓解，而4例受试者中有2例（50％）和3例受试者中有2例（66.7％）部分/完全缓解。分别。随访12个月的2例患者中有1例完全缓解，1例患者24个月后NS部分缓解。在随访期间，所有患者的eGFR均得到改善或稳定。结论：我们的多中心研究结果表明，在某些耐药的原发性FSGS患者中，对类固醇或免疫抑制治疗的反应率有所提高，并引起蛋白尿的完全或部分缓解。我们研究的主要局限性是受试者数量少，辍学率高。
