Department of Pediatric Nephrology, Lanzhou University Second Hospital, Lanzhou, Gansu, 730030, China.
Nantong Institute of Genetics and Reproductive Medicine, Affiliated Maternity and Child Healthcare Hospital of Nantong University, Nantong, Jiangsu, 226018, China.
Paediatr Drugs. 2022 Jul;24(4):389-401. doi: 10.1007/s40272-022-00506-1. Epub 2022 May 5.
Children with severe Henoch-Schönlein purpura nephritis (HSPN) may progress to end-stage renal disease without appropriate treatment.
This study aimed to investigate the efficacy and safety of tacrolimus combined with glucocorticoids in the treatment of pediatric HSPN.
A total of 87 HSPN patients with urinary protein ≥ 0.75 g/24 h received standard of care, including angiotensin II receptor blockers/angiotensin-converting enzyme inhibitors and glucocorticoids. Patients were divided into three groups and additionally received tacrolimus (n = 30), cyclophosphamide (n = 31), or mycophenolate mofetil (MMF) (n = 26). We monitored outcome measures, including proteinuria, hematuria, and renal function and analyzed the efficacy and side effects in each group.
At 2-month follow-up, the overall efficacy was 93.3%, 83.9%, and 61.5% for tacrolimus, cyclophosphamide, and MMF, respectively (P < 0.05). Urinary protein significantly decreased for all groups. Urinary red blood cell counts significantly decreased for patients treated with tacrolimus (P < 0.001) and cyclophosphamide (P < 0.05), whereas no significant decrease was seen for those receiving MMF (P = 0.09). Although urine β2-microglobulin significantly decreased following 2 months of treatment with all medications, efficacy was greater with tacrolimus than with cyclophosphamide and MMF (P < 0.001). Major adverse events were respiratory and urinary infections, with MMF having the highest infection rate. The cyclophosphamide group also experienced additional adverse events, including arrhythmia, hemorrhagic cystitis, leukocytosis, thrombocytopenia, and hyperglycemia.
These results indicate that tacrolimus is more effective at reducing proteinuria and hematuria and improving renal function, with relatively milder side effects, in the treatment of pediatric HSPN.
ChiCTR2200055323, retrospectively registered on January 7, 2022.
患有严重过敏性紫癜肾炎（HSPN）的儿童如果得不到适当的治疗，可能会进展为终末期肾病。
本研究旨在探讨他克莫司联合糖皮质激素治疗儿童 HSPN 的疗效和安全性。
共纳入 87 例尿蛋白≥0.75 g/24 h 的 HSPN 患儿，给予血管紧张素 II 受体阻滞剂/血管紧张素转换酶抑制剂和糖皮质激素标准治疗。患者分为三组，分别加用他克莫司（n=30）、环磷酰胺（n=31）或霉酚酸酯（MMF）（n=26）。我们监测了包括蛋白尿、血尿和肾功能在内的结局指标，并分析了各组的疗效和不良反应。
在 2 个月的随访中，他克莫司、环磷酰胺和 MMF 的总体有效率分别为 93.3%、83.9%和 61.5%（P<0.05）。所有组的尿蛋白均显著下降。他克莫司（P<0.001）和环磷酰胺（P<0.05）治疗组的尿红细胞计数显著下降，而 MMF 组无明显下降（P=0.09）。尽管所有药物治疗 2 个月后尿β2-微球蛋白均显著下降，但他克莫司的疗效优于环磷酰胺和 MMF（P<0.001）。主要不良事件为呼吸道和泌尿道感染，MMF 感染率最高。环磷酰胺组还出现了心律失常、出血性膀胱炎、白细胞增多、血小板减少和高血糖等其他不良反应。
这些结果表明，他克莫司在治疗儿童 HSPN 时，能更有效地减少蛋白尿和血尿，改善肾功能，且不良反应相对较轻。
ChiCTR2200055323，于 2022 年 1 月 7 日进行了回顾性注册。
