Ehren Rasmus, Benz Marcus R, Doetsch Jorg, Fichtner Alexander, Gellermann Jutta, Haffner Dieter, Höcker Britta, Hoyer Peter F, Kästner Bärbel, Kemper Markus J, Konrad Martin, Luntz Steffen, Querfeld Uwe, Sander Anja, Toenshoff Burkhard, Weber Lutz T
Department of Pediatrics, University Children's Hospital Köln, University Hospital Köln, Köln, Germany.
Department of Pediatrics I, University Children's Hospital Heidelberg, Heidelberg, Germany.
BMJ Open. 2018 Oct 10;8(10):e024882. doi: 10.1136/bmjopen-2018-024882.
Idiopathic nephrotic syndrome is the most common glomerular disease in childhood with an incidence of 1.8 cases per 100 000 children in Germany. The treatment of the first episode implies two aspects: induction of remission and sustainment of remission. The recent Kidney Disease Improving Global Outcomes, American Academy of Pediatrics and German guidelines for the initial treatment of the first episode of a nephrotic syndrome recommend a 12-week course of prednisone. Despite being effective, this treatment is associated with pronounced glucocorticoid-associated toxicity due to high-dose prednisone administration over a prolonged period of time. The aim of the INTENT study (Initial treatment of steroid-sensitive idiopathic nephrotic syndrom in children with mycophenolate mofetil versus prednisone: protocol for a randomised, controlled, multicentre trial) is to show that an alternative treatment regimen with mycophenolic acid is not inferior regarding sustainment of remission, but with lower toxicity compared with treatment with glucocorticoids only.
The study is designed as an open, randomised, controlled, multicentre trial. 340 children with a first episode of steroid-sensitive nephrotic syndrome and who achieved remission by a standard prednisone regimen will be enrolled in the trial and randomised to one of two treatment arms. The standard care group will be treated with prednisone for a total of 12 weeks; in the experimental group the treatment is switched to mycophenolate mofetil, also for a total of 12 weeks in treatment duration. The primary endpoint is the occurrence of a treated relapse within 24 months after completion of initial treatment.
Ethics approval for this trial was granted by the ethics committee of the Medical Faculty of the University of Heidelberg (AFmu-554/2014). The study results will be published in accordance with the Consolidated Standards of Reporting Trials statement and the Standard Protocol Items: Recommendations for Interventional Trials guidelines. Our findings will be submitted to major international paediatric nephrology and general paediatric conferences and submitted for publication in a peer-reviewed, open-access journal.
DRKS0006547; EudraCT2014-001991-76; Pre-result.
30 October 2014; 24 February 2017.
特发性肾病综合征是儿童期最常见的肾小球疾病，在德国每10万名儿童中的发病率为1.8例。首次发作的治疗包括两个方面：诱导缓解和维持缓解。最近的改善全球肾脏病预后组织、美国儿科学会以及德国关于肾病综合征首次发作初始治疗的指南推荐使用泼尼松进行为期12周的疗程。尽管这种治疗有效，但由于长期大剂量使用泼尼松，会带来明显的糖皮质激素相关毒性。INTENT研究（霉酚酸酯与泼尼松治疗儿童激素敏感性特发性肾病综合征的初始治疗：一项随机、对照、多中心试验方案）的目的是表明，使用霉酚酸的替代治疗方案在维持缓解方面并不逊色，但与仅使用糖皮质激素治疗相比，毒性更低。
本研究设计为开放、随机、对照、多中心试验。340名首次发作的激素敏感性肾病综合征且通过标准泼尼松方案实现缓解的儿童将被纳入试验，并随机分配至两个治疗组之一。标准治疗组将接受总共12周的泼尼松治疗；在试验组中，治疗改为霉酚酸酯，治疗时长同样为总共12周。主要终点是初始治疗完成后24个月内出现经治疗的复发。
本试验已获得海德堡大学医学院伦理委员会的伦理批准（AFmu - 554/2014）。研究结果将按照《报告试验的统一标准》声明和《干预试验标准方案条目：建议》指南进行发表。我们的研究结果将提交至主要的国际儿科肾脏病学和普通儿科学会议，并提交至同行评审的开放获取期刊发表。
DRKS0006547；EudraCT2014 - 001991 - 76；预结果。
2014年10月30日；2017年2月24日。
