Hari Pankaj, Khandelwal Priyanka, Boyer Olivia, Bhimma Rajendra, Cano Francesco, Christian Martin, Duzova Ali, Iijima Kazumoto, Kang Hee Gyung, Qian Shen, Safouh Hesham, Samuels Susan, Smoyer William E, Vivarelli Marina, Bagga Arvind, Schaefer Franz
Division of Pediatric Nephrology, Department of Pediatrics, All India Institute of Medical Sciences, New Delhi, India.
Centre de Référence MARHEA, Institut Imagine, Néphrologie Pédiatrique, Université Paris Cité, Hôpital Necker - Enfants Malades, Paris, France.
Pediatr Nephrol. 2025 Mar;40(3):865-872. doi: 10.1007/s00467-024-06543-x. Epub 2024 Oct 10.
Assessment of the true impact of therapeutic interventions is a challenge in the absence of universal, standardized definitions for clinical trial endpoints in children with kidney diseases. Steroid-resistant nephrotic syndrome (SRNS) is a difficult kidney disease to treat, with unremitting disease progressing to kidney failure. Currently, available therapies result in suboptimal cure rates. Clinical trials with innovative, targeted treatments will likely be conducted for this disease in the foreseeable future. An international consortium of the IPNA Best Practices and Standards Committee and the Pediatric Nephrology Expert Group of the conect4children (c4c) network developed through consensus, standardized, internationally acceptable definitions for trial outcomes for SRNS. The endpoint definitions were formulated for use with urine protein to creatinine ratios and estimated glomerular filtration rates. Definitions of complete remission, partial remission, non-remission of disease, reduction in proteinuria, kidney disease progression, kidney failure, and composite kidney outcome were refined using an iterative process until a consensus was achieved.
在缺乏针对肾病患儿临床试验终点的通用、标准化定义的情况下，评估治疗干预措施的真正影响是一项挑战。激素抵抗型肾病综合征（SRNS）是一种难以治疗的肾脏疾病，病情持续进展直至肾衰竭。目前，现有的治疗方法治愈率欠佳。在可预见的未来，可能会针对这种疾病开展采用创新、靶向治疗的临床试验。国际儿科肾脏病协会最佳实践与标准委员会以及conect4children（c4c）网络的儿科肾脏病专家组组成的国际联盟，通过共识制定了SRNS试验结果的标准化、国际认可定义。终点定义是为配合尿蛋白与肌酐比值以及估计肾小球滤过率而制定的。通过反复迭代过程完善了完全缓解、部分缓解、疾病未缓解、蛋白尿减少、肾病进展、肾衰竭和复合肾脏结局的定义，直至达成共识。
