Abstract: FR-OR105
Efficacy and Safety of Tac or MMF for Children with Steroid-Sensitive but Frequent Relapse or Steroid-Dependent Nephrotic Syndrome: A Nationwide, Multicentre Randomized Study
Session Information
- High-Impact Clinical Trials - 1
October 25, 2024 | Location: Hall D, Convention Center
Abstract Time: 12:00 PM - 12:15 PM
Category: Glomerular Diseases
- 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics
Authors
- Liu, Fei, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hang Zhou, China
- Wang, Jingjing, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hang Zhou, China
- Yan, Weili, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China
- Zhou, Jianhua, Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology, Wuhan, China
- Jiang, Xiaoyun, The First Affiliated Hospital, Sun Yat-sen University, Guangzhou, China
- Zhao, Fei, Children Hospital of Nanjing Medical University, Nanjing, China
- Wu, Xiaochuan, The Second Xiangya Hospital of Central South University, Hunan, China
- Sun, Shuzhen, Shandong Provincial Hospital, Cheeloo College of Medicine, Shandong University, Jinan, China
- Wang, Mo, Children Hospital of Chongqing Medical University, National Clinical Research Center for Child Health and Disorders, Ministry of Education Key Laboratory, Chongqing, China
- Xu, Hong, Children's Hospital of Fudan University, Shanghai, China
- Chen, Jing, Children's Hospital of Fudan University, Shanghai, China
- Liu, Cuihua, Zhengzhou Key Laboratory of Pediatric Kidney Disease Research, Children Hospital Affiliated to Zhengzhou University, Zhengzhou, China
- Feng, Shipin, The Affiliated Women's and Children's Hospital, School of Medicine, UESTC; Chengdu Women's and Children's Central Hospital, Chengdu, China
- Zhong, Xuhui, Peking University First Hospital, Beijing, China
- Fu, Haidong, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hang Zhou, China
- Huang, Lingfei, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hang Zhou, China
- Ma, Daqing, Children’s Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China
- Ding, Jie, Peking University First Hospital, Beijing, China
- Ye, Qing, Children Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China
- Mao, Jianhua, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hang Zhou, China
Background
Both tacrolimus (TAC) and mycophenolate mofetil (MMF) are recommended for children with frequently relapsing nephrotic syndrome (FRNS) and steroid-dependent nephrotic syndrome (SDNS). However, their comparative effectiveness and safety have not been evaluated through randomized controlled studies.
Methods
In this multicenter, randomized, open-label, controlled trial, 270 children aged 2-18 years with FRNS/SDNS were allocated at a 1:1 ratio to receive either TAC (0.025-0.05 mg/kg, orally twice daily) or MMF (10-15 mg/kg, orally twice daily) for one year, along with a tapering regimen of steroids. The primary endpoint was 1-year relapse-free survival. Relapse frequency, cumulative steroid dosage and safety profiles were also evaluated. The trial was prospectively registered at ClinicalTrials.gov (NCT04048161).
Results
A total of 243 out of 270 patients completed the trial, and their data were analyzed and reported. Compared with MMF, TAC significantly reduced the risk of relapse by 65% (HR = 0.35, 95% CI: 0.21–0.56, P <0.0001) in the intention-to-treat analysis. This difference was also significant after adjusting for the per-protocol analysis (HR = 0.36, 95% CI: 0.22–0.58, P <0.0001). Among the relapsed patients, the median time to first relapse was 225.5 days in the TAC group and 165.5 days in the MMF group. The TAC group had fewer annual relapses (mean [SD], 0.319 [0.936] vs. 0.778 [1.274]) and a reduced cumulative dose of steroid (0.221 [0.103] vs. 0.337 [0.222] mg/kg/d) than did the MMF group. The safety profile was similar in both groups, with infections being the most common adverse event.
Conclusion
In children with FRNS/SDNS, a 1-year course of TAC therapy significantly extended the period of relapse-free survival in comparison to MMF treatment.
Funding
- Commercial Support – The STAMP trial was sponsored by Hangzhou Zhongmei Huadong Pharmaceutical Co., Ltd., Hangzhou, Zhejiang, China