儿童类固醇敏感肾病综合征中使用类固醇保留疗法的实践差异:一项前瞻性队列研究的结果。

IF 2.6 3区 医学 Q1 PEDIATRICS
Pediatric Nephrology Pub Date : 2025-11-01 Epub Date: 2025-07-01 DOI:10.1007/s00467-025-06853-8
Angie Aguilar-González, Ava Zardynezhad, Catherine Morgan, Allison Dart, Cherry Mammen, Rulan S Parekh, Pavel Geier, Genevieve Benoit, Guido Filler, Janusz Feber, James Tee, Steven Arora, Damien Noone, Silviu Grisaru, Rahul Chanchlani, Augustina Okpere, Susan Samuel
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引用次数: 0

摘要

背景:在儿童类固醇敏感肾病综合征(SSNS)中,保留类固醇治疗常用于延长疾病缓解期,同时将类固醇毒性降至最低。本研究旨在描述加拿大儿童SSNS使用类固醇保留药物的实践差异。方法:将来自加拿大11个儿科肾病中心的1-18岁肾病综合征(NS)儿童纳入2013-2019年加拿大儿童肾病综合征(CHILDNEPH)前瞻性队列。采用汇总统计方法分析儿童接受环磷酰胺、他克莫司、霉酚酸酯、环孢素和利妥昔单抗治疗的首次复发时间。在入组前接受类固醇保留治疗的参与者被排除在外。结果:在入组的371名儿童中,321名(86.5%)患有SSNS, 133名符合研究标准。入组时中位年龄为3.0岁(IQR: 2.0-6.3)。中位随访期为3.0年(IQR 1.0-4.0)。起始时间和保留类固醇治疗的选择在各中心有所不同。大多数患者(72.2%)在中位3次复发后开始治疗(IQR: 2-4)。环磷酰胺和他克莫司是最常用的初始治疗药物,分别为39.1%和23.3%。30例(22.6%)患者在中位时间4个月后改用第二种药物(IQR 0.25-11);其中,40%的患者从环磷酰胺或霉酚酸酯转为他克莫司。初始治疗到首次复发的时间差异无统计学意义(log rank p值0.36)。结论:在SSNS的治疗中,类固醇保留疗法的使用存在显著差异。临床试验需要检查这些药物的疗效,以优化治疗和减少实践变化。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Practice variation in the use of steroid-sparing therapies in childhood steroid-sensitive nephrotic syndrome: results from a prospective cohort study.

Background: Steroid-sparing therapies are often used to prolong disease remission while minimizing steroid toxicity in childhood steroid-sensitive nephrotic syndrome (SSNS). This study aims to describe practice variation in the use of steroid-sparing medications for childhood SSNS in Canada.

Methods: Children (1-18 years) with nephrotic syndrome (NS) from eleven pediatric nephrology centers in Canada were enrolled in the Canadian Childhood Nephrotic Syndrome (CHILDNEPH) prospective cohort from 2013-2019. Data, including time to first relapse for children treated with cyclophosphamide, tacrolimus, mycophenolate mofetil, cyclosporine and rituximab were analyzed using summary statistics. Participants who received steroid-sparing therapies prior to enrolment were excluded.

Results: Of the 371 children enrolled, 321 (86.5%) had SSNS and 133 met the study criteria. Median age at enrollment was 3.0 years (IQR: 2.0-6.3). Median follow-up period was 3.0 years (IQR 1.0-4.0). Timing of initiation and choice of steroid-sparing therapy varied across centers. The majority (72.2%) initiated therapies after a median of 3 relapses (IQR: 2-4). Cyclophosphamide and tacrolimus were the most frequently used initial therapies at 39.1% and 23.3%, respectively. Thirty (22.6%) patients switched to a second medication after a median time of 4 months (IQR 0.25-11); of which, 40% switched from either cyclophosphamide or mycophenolate mofetil to tacrolimus. There were no statistically significant differences in time to first relapse with initial therapies (log rank P-value 0.36).

Conclusions: Significant variation in the use of steroid-sparing therapies exists in the treatment of SSNS. A clinical trial is needed to examine the efficacy of these medications to optimize treatment and decrease practice variation.

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来源期刊
Pediatric Nephrology
Pediatric Nephrology 医学-泌尿学与肾脏学
CiteScore
4.70
自引率
20.00%
发文量
465
审稿时长
1 months
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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