Department of Gastroenterology, Hacettepe University, Ankara, Turkey.
Department of Medicine, Cleveland Clinic Foundation, Cleveland, OH, USA.
Dig Dis Sci. 2018 May;63(5):1348-1354. doi: 10.1007/s10620-018-5011-x. Epub 2018 Mar 22.
We studied the efficacy and safety of mycophenolate mofetil (MMF) and tacrolimus as second-line therapy in pediatric patients with autoimmune hepatitis (AIH) who were intolerant or non-responders to standard therapy (corticosteroid and azathioprine).
We performed a retrospective study of data from 13 centers in Europe, USA, and Canada. Thirty-eight patients (< 18 years old) who received second-line therapy (18 MMF and 20 tacrolimus), for a median of 72 months (range 8-182) were evaluated. Patients were categorized into two groups: Group 1 (n = 17) were intolerant to corticosteroid or azathioprine, and group 2 (n = 21) were non-responders to standard therapy.
Overall complete response rates were similar in patients treated with MMF and tacrolimus (55.6 vs. 65%, p = 0.552). In group 1, MMF and tacrolimus maintained a biochemical remission in 88.9 and 87.5% of patients, respectively (p = 0.929). More patients in group 2 given tacrolimus compared to MMF had a complete response, but the difference was not statistically significant (50.0 vs. 22.2%, p = 0.195). Biochemical remission was achieved in 71.1% (27/38) of patients by tacrolimus and/or MMF. Decompensated cirrhosis was more commonly seen in MMF and/or tacrolimus non-responders than in responders (45.5 vs. 7.4%, p = 0.006). Five patients who received second-line therapy (2 MMF and 3 tacrolimus) developed side effects that led to therapy withdrawal.
Long-term therapy with MMF or tacrolimus was generally well tolerated by pediatric patients with AIH. Both MMF and tacrolimus had excellent efficacy in patients intolerant to corticosteroid or azathioprine. Tacrolimus might be more effective than MMF in patients failing previous therapy.
我们研究了霉酚酸酯（MMF）和他克莫司在对标准治疗（皮质类固醇和硫唑嘌呤）不耐受或无反应的自身免疫性肝炎（AIH）儿科患者中的疗效和安全性。
我们对来自欧洲、美国和加拿大的 13 个中心的数据进行了回顾性研究。38 名（<18 岁）接受二线治疗（18 名 MMF 和 20 名他克莫司）的患者，中位时间为 72 个月（范围 8-182），进行了评估。患者分为两组：第 1 组（n=17）对皮质类固醇或硫唑嘌呤不耐受，第 2 组（n=21）对标准治疗无反应。
接受 MMF 和他克莫司治疗的患者的总体完全缓解率相似（55.6% vs. 65%，p=0.552）。在第 1 组中，MMF 和他克莫司分别维持 88.9%和 87.5%的患者生化缓解（p=0.929）。第 2 组中接受他克莫司治疗的患者比接受 MMF 治疗的患者有更高的完全缓解率，但差异无统计学意义（50.0% vs. 22.2%，p=0.195）。通过他克莫司和/或 MMF，71.1%（27/38）的患者达到生化缓解。在 MMF 和/或他克莫司无反应者中，失代偿性肝硬化更为常见（45.5% vs. 7.4%，p=0.006）。5 名接受二线治疗（2 名 MMF 和 3 名他克莫司）的患者因出现副作用而停止治疗。
MMF 或他克莫司长期治疗儿科 AIH 患者通常耐受良好。MMF 和他克莫司在对皮质类固醇或硫唑嘌呤不耐受的患者中均具有良好的疗效。在既往治疗失败的患者中，他克莫司可能比 MMF 更有效。
