Abstract：
The best treatment for IgAN is still debated. The trials NEFIGAN and NEFIGARD have demonstrated that TRF-budesonide (Nefecon) efficiently and safely reduced proteinuria in adults, leading to FDA approval of Nefecon for adult IgAN. In pediatric IgAN, an etiological treatment does not yet exist, and the main therapies remain RAAS inhibitors and oral steroids. To our knowledge, this is one of the few pediatric reports of TRF-budesonide therapy.
A 13-year-old boy underwent a kidney biopsy for recurrent macrohematuria and proteinuria, resulting in an IgAN diagnosis (MEST-C score M1-E1-S0-T0-C1). At admission, serum creatinine and UPCR were slightly increased. Three methylprednisolone pulses were performed, followed by prednisone and RAAS inhibitors therapy. However, after 10 months, macrohematuria became constant, and UPCR increased. A new kidney biopsy was performed, showing an increase in sclerotic lesions. Prednisone was discontinued, and a trial with IBD TRF-budesonide 9 mg/day started. One month later, macrohematuria episodes disappeared and UPCR decreased, with a stable kidney function. After 5 months, due to a reduction in morning cortisol levels and difficulty in drug provisioning, we started to wean TRF-budesonide by 3 mg every 3 months, with complete withdrawal after 1 year. During this period, episodes of macrohematuria dramatically decreased, and UPCR and kidney function were maintained stable.
Our case demonstrates that TRF-budesonide could be considered an effective second-line treatment in pediatric IgAN, particularly when a long course of steroids is necessary to control active inflammation. However, pediatric clinical trials to identify the correct dosage and tolerability of TRF-budesonide are urgently needed.
A 13-year-old boy underwent a kidney biopsy for recurrent macrohematuria and proteinuria, resulting in an IgAN diagnosis (MEST-C score M1-E1-S0-T0-C1). At admission, serum creatinine and UPCR were slightly increased. Three methylprednisolone pulses were performed, followed by prednisone and RAAS inhibitors therapy. However, after 10 months, macrohematuria became constant, and UPCR increased. A new kidney biopsy was performed, showing an increase in sclerotic lesions. Prednisone was discontinued, and a trial with IBD TRF-budesonide 9 mg/day started. One month later, macrohematuria episodes disappeared and UPCR decreased, with a stable kidney function. After 5 months, due to a reduction in morning cortisol levels and difficulty in drug provisioning, we started to wean TRF-budesonide by 3 mg every 3 months, with complete withdrawal after 1 year. During this period, episodes of macrohematuria dramatically decreased, and UPCR and kidney function were maintained stable.
Our case demonstrates that TRF-budesonide could be considered an effective second-line treatment in pediatric IgAN, particularly when a long course of steroids is necessary to control active inflammation. However, pediatric clinical trials to identify the correct dosage and tolerability of TRF-budesonide are urgently needed.
摘要：
背景：IgAN的最佳治疗方法仍存在争议。试验NEFIGAN和NEFIGARD已经证明TRF-布地奈德（Nefecon）有效和安全地减少成人的蛋白尿，导致FDA批准Nefecon治疗成人IgAN。在小儿IgAN中，病因治疗尚不存在，和主要的疗法仍然是RAAS抑制剂和口服类固醇。据我们所知,这是少数关于TRF-布地奈德治疗的儿科报告之一.
■一名13岁男孩因复发性大血尿和蛋白尿而接受肾活检，导致IgAN诊断（MEST-C评分M1-E1-S0-T0-C1）。入院时,血清肌酐和UPCR略有升高.进行了三次甲基强的松龙脉冲，其次是泼尼松和RAAS抑制剂治疗。然而,10个月后,巨大血尿变得恒定，UPCR增加。做了新的肾活检,显示硬化病变增加。泼尼松停药,并开始使用IBDTRF-布地奈德9mg/天的试验。一个月后,巨大血尿发作消失，UPCR减少，有稳定的肾功能.5个月后,由于早晨皮质醇水平降低和药物供应困难，我们开始每3个月用3毫克的TRF-布地奈德断奶，1年后完全退出。在此期间,巨大血尿的发作显著减少，UPCR和肾功能维持稳定。
结论：我们的病例表明TRF-布地奈德可被认为是小儿IgAN的有效二线治疗，特别是当需要长期服用类固醇来控制活动性炎症时。然而,迫切需要进行儿科临床试验,以确定TRF-布地奈德的正确剂量和耐受性.
■一名13岁男孩因复发性大血尿和蛋白尿而接受肾活检，导致IgAN诊断（MEST-C评分M1-E1-S0-T0-C1）。入院时,血清肌酐和UPCR略有升高.进行了三次甲基强的松龙脉冲，其次是泼尼松和RAAS抑制剂治疗。然而,10个月后,巨大血尿变得恒定，UPCR增加。做了新的肾活检,显示硬化病变增加。泼尼松停药,并开始使用IBDTRF-布地奈德9mg/天的试验。一个月后,巨大血尿发作消失，UPCR减少，有稳定的肾功能.5个月后,由于早晨皮质醇水平降低和药物供应困难，我们开始每3个月用3毫克的TRF-布地奈德断奶，1年后完全退出。在此期间,巨大血尿的发作显著减少，UPCR和肾功能维持稳定。
结论：我们的病例表明TRF-布地奈德可被认为是小儿IgAN的有效二线治疗，特别是当需要长期服用类固醇来控制活动性炎症时。然而,迫切需要进行儿科临床试验,以确定TRF-布地奈德的正确剂量和耐受性.
