Cal H Robinson, Simon Carter, Nowrin Aman, Valentina Bruno, Shelby Chan, Brian Cuthbertson, Mackenzie Garner, Eddy Fan, Christoph Licht, Ashlene M McKay, Seetha Radhakrishnan, Veronique Rowley, Chia Wei Teoh, Jovanka Z Vasilevska-Ristovska, Anna Heath, Rulan S Parekh
{"title":"建立儿童类固醇敏感性肾病综合征临床试验的核心结果和最小的临床重要差异：德尔菲共识过程的结果。","authors":"Cal H Robinson, Simon Carter, Nowrin Aman, Valentina Bruno, Shelby Chan, Brian Cuthbertson, Mackenzie Garner, Eddy Fan, Christoph Licht, Ashlene M McKay, Seetha Radhakrishnan, Veronique Rowley, Chia Wei Teoh, Jovanka Z Vasilevska-Ristovska, Anna Heath, Rulan S Parekh","doi":"10.1007/s00467-025-06960-6","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>Few randomized controlled trials are conducted in childhood nephrotic syndrome, and substantial global practice variation exists. Trials have not reported consistent outcomes, patient-reported outcomes, or justified minimal clinically important differences (MCID). Our aim was to establish consensus on core outcomes, assessment timepoints, MCIDs, and desirability of outcome ranking (DOOR) endpoints for future childhood steroid-sensitive nephrotic syndrome research.</p><p><strong>Methods: </strong>We conducted a two-stage Delphi consensus survey of international healthcare providers and Canadian patients and caregivers with experience in childhood nephrotic syndrome. Respondents rated potential outcomes using Likert scales and assigned MCID values for relapse and remission endpoints. Following the surveys, we held workshops for healthcare providers and patients/caregivers in Toronto, Canada, with facilitated discussions. Qualitative data was analyzed thematically to explore perspectives on childhood nephrotic syndrome clinical trial outcomes and MCIDs.</p><p><strong>Results: </strong>Eighty-one participants (45 providers, 36 patients/caregivers, 63% Canadian) responded to the surveys. Three disease outcomes (relapse rate, relapse-free survival, and relapse occurrence) met consensus criteria as core outcomes. Median MCID values were a 25% absolute difference between two treatments in relapse risk by 1-year (for relapse prevention) choices and 10% absolute difference in remission by 2 weeks (for relapse treatment). Consensus was achieved for five core patient-reported outcomes (pain, physical symptoms, physical function and mobility, life participation, and social function) and DOOR endpoints for nephrotic syndrome relapse and remission.</p><p><strong>Conclusions: </strong>Consensus exists among surveyed participants on core disease and patient-reported outcomes for childhood nephrotic syndrome research. Findings can improve the quality and reporting of future trials in this population.</p>","PeriodicalId":19735,"journal":{"name":"Pediatric Nephrology","volume":" ","pages":""},"PeriodicalIF":2.6000,"publicationDate":"2025-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Establishing core outcomes and minimal clinically important differences for childhood steroid sensitive nephrotic syndrome clinical trials: results from a Delphi consensus process.\",\"authors\":\"Cal H Robinson, Simon Carter, Nowrin Aman, Valentina Bruno, Shelby Chan, Brian Cuthbertson, Mackenzie Garner, Eddy Fan, Christoph Licht, Ashlene M McKay, Seetha Radhakrishnan, Veronique Rowley, Chia Wei Teoh, Jovanka Z Vasilevska-Ristovska, Anna Heath, Rulan S Parekh\",\"doi\":\"10.1007/s00467-025-06960-6\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"<p><strong>Background: </strong>Few randomized controlled trials are conducted in childhood nephrotic syndrome, and substantial global practice variation exists. Trials have not reported consistent outcomes, patient-reported outcomes, or justified minimal clinically important differences (MCID). Our aim was to establish consensus on core outcomes, assessment timepoints, MCIDs, and desirability of outcome ranking (DOOR) endpoints for future childhood steroid-sensitive nephrotic syndrome research.</p><p><strong>Methods: </strong>We conducted a two-stage Delphi consensus survey of international healthcare providers and Canadian patients and caregivers with experience in childhood nephrotic syndrome. Respondents rated potential outcomes using Likert scales and assigned MCID values for relapse and remission endpoints. Following the surveys, we held workshops for healthcare providers and patients/caregivers in Toronto, Canada, with facilitated discussions. Qualitative data was analyzed thematically to explore perspectives on childhood nephrotic syndrome clinical trial outcomes and MCIDs.</p><p><strong>Results: </strong>Eighty-one participants (45 providers, 36 patients/caregivers, 63% Canadian) responded to the surveys. Three disease outcomes (relapse rate, relapse-free survival, and relapse occurrence) met consensus criteria as core outcomes. Median MCID values were a 25% absolute difference between two treatments in relapse risk by 1-year (for relapse prevention) choices and 10% absolute difference in remission by 2 weeks (for relapse treatment). Consensus was achieved for five core patient-reported outcomes (pain, physical symptoms, physical function and mobility, life participation, and social function) and DOOR endpoints for nephrotic syndrome relapse and remission.</p><p><strong>Conclusions: </strong>Consensus exists among surveyed participants on core disease and patient-reported outcomes for childhood nephrotic syndrome research. Findings can improve the quality and reporting of future trials in this population.</p>\",\"PeriodicalId\":19735,\"journal\":{\"name\":\"Pediatric Nephrology\",\"volume\":\" \",\"pages\":\"\"},\"PeriodicalIF\":2.6000,\"publicationDate\":\"2025-10-14\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Pediatric Nephrology\",\"FirstCategoryId\":\"3\",\"ListUrlMain\":\"https://doi.org/10.1007/s00467-025-06960-6\",\"RegionNum\":3,\"RegionCategory\":\"医学\",\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"Q1\",\"JCRName\":\"PEDIATRICS\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Nephrology","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s00467-025-06960-6","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PEDIATRICS","Score":null,"Total":0}
Establishing core outcomes and minimal clinically important differences for childhood steroid sensitive nephrotic syndrome clinical trials: results from a Delphi consensus process.
Background: Few randomized controlled trials are conducted in childhood nephrotic syndrome, and substantial global practice variation exists. Trials have not reported consistent outcomes, patient-reported outcomes, or justified minimal clinically important differences (MCID). Our aim was to establish consensus on core outcomes, assessment timepoints, MCIDs, and desirability of outcome ranking (DOOR) endpoints for future childhood steroid-sensitive nephrotic syndrome research.
Methods: We conducted a two-stage Delphi consensus survey of international healthcare providers and Canadian patients and caregivers with experience in childhood nephrotic syndrome. Respondents rated potential outcomes using Likert scales and assigned MCID values for relapse and remission endpoints. Following the surveys, we held workshops for healthcare providers and patients/caregivers in Toronto, Canada, with facilitated discussions. Qualitative data was analyzed thematically to explore perspectives on childhood nephrotic syndrome clinical trial outcomes and MCIDs.
Results: Eighty-one participants (45 providers, 36 patients/caregivers, 63% Canadian) responded to the surveys. Three disease outcomes (relapse rate, relapse-free survival, and relapse occurrence) met consensus criteria as core outcomes. Median MCID values were a 25% absolute difference between two treatments in relapse risk by 1-year (for relapse prevention) choices and 10% absolute difference in remission by 2 weeks (for relapse treatment). Consensus was achieved for five core patient-reported outcomes (pain, physical symptoms, physical function and mobility, life participation, and social function) and DOOR endpoints for nephrotic syndrome relapse and remission.
Conclusions: Consensus exists among surveyed participants on core disease and patient-reported outcomes for childhood nephrotic syndrome research. Findings can improve the quality and reporting of future trials in this population.
期刊介绍：
International Pediatric Nephrology Association
Pediatric Nephrology publishes original clinical research related to acute and chronic diseases that affect renal function, blood pressure, and fluid and electrolyte disorders in children. Studies may involve medical, surgical, nutritional, physiologic, biochemical, genetic, pathologic or immunologic aspects of disease, imaging techniques or consequences of acute or chronic kidney disease. There are 12 issues per year that contain Editorial Commentaries, Reviews, Educational Reviews, Original Articles, Brief Reports, Rapid Communications, Clinical Quizzes, and Letters to the Editors.
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