Effects and safety of dapagliflozin in paediatric hereditary kidney disease: protocol for a multicentric, prospective, open and randomised crossover study (DAPA-PedHKD)

作者：Wei Zhang, Yalan Dou, Jiaojiao Liu, Tianyi Liu, Weili Yan, Qian Shen, Hong Xu, Yi­hui Zhai · 发表于：BMJ Open · 年份：2025 · DOI：10.1136/bmjopen-2025-109833 · 被引用次数：1 · 研究领域：Diabetes Treatment and Management、Chronic Kidney Disease and Diabetes、Pancreatic function and diabetes

INTRODUCTION: Sodium-glucose cotransporter-2 inhibitors have demonstrated cardiorenal protective effects in adults with chronic kidney disease (CKD), but their efficacy and safety in paediatric CKD remain uncertain. Treatment for hereditary kidney disease in children is limited. This DAPA-PedHKD study aims to evaluate the effectiveness and safety of combining renin-angiotensin-aldosterone system inhibitors (RAASi) with dapagliflozin compare to RAASi alone in children with hereditary kidney disease accompanied by proteinuria. METHODS AND ANALYSIS: , and either a 24-hour urinary protein level of >0.2 g or a urinary protein-to-creatinine ratio (UPCR) of >0.2 mg/mg. Participants will be randomised in a 1:1 ratio to receive either dapagliflozin in addition to standard RAASi therapy or RAASi therapy alone for 12 weeks. After a 4-week washout period with continued RAASi therapy, participants will crossover to the other treatment for another 12 weeks. Outcome assessments will be conducted at baseline and at weeks 2, 6, 12, 16, 18, 22 and 28. The primary outcome is the change in 24-hour urinary protein excretion from baseline to week 12. Secondary outcomes include changes in the UPCR, urinary albumin-to-creatinine ratio, serum albumin, eGFR, blood pressure and body weight from baseline to week 12. Neither participants nor investigators are blinded to treatment allocation, and placebo control is not used. ETHICS AND DISSEMINATION: This study has been approved by the Ethics Committee of...