Mycophenolate mofetil as rescue treatment for autoimmune liver disease in children: A 5-year follow-up
Mycophenolate mofetil as rescue treatment for autoimmune liver disease in children: A 5-year follow-up
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DOI：
10.1016/j.jhep.2009.02.024
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发表时间：
2009-07-01
影响因子：
25.7
通讯作者：
Mieli-Vergani, Giorgina
中科院分区：
文献类型：
--
作者：
Aw, Marion M.;Dhawan, Anil;Mieli-Vergani, Giorgina
背景/目的：本研究的目的是评估结果霉酚酸酯（MM F）治疗儿童自身免疫性肝病谁是耐药或不耐受的标准immunosuppression.Methods：纳入标准：（a）未能达到/维持缓解与泼尼松龙/硫唑嘌呤治疗或（B）显着的治疗副作用。初始MMF剂量为20 mg/kg/天，逐渐增加至最大剂量40 mg/kg/天。硫唑嘌呤停止时开始MMF.Results：26名儿童（17名女性）被招募。诊断时的中位（范围）年龄为9.9（1.2-14.4）岁。16例为I型自身免疫性肝炎（AIH），2例为2型AIH，8例为自身免疫性硬化性胆管炎（ASC）。从诊断到添加MMF的中位（范围）时间为14.9（0.2-108.6）个月。18名儿童对MMF有反应，14名天冬氨酸转氨酶（AST）正常化。中位（范围）随访61.5（19.5-96.3）个月时，12例患者的AST保持正常。所有18名儿童都很好，但有两名儿童有门静脉高压症的临床体征。8例（6例ASC）无应答：7例AST仍升高，1例因失代偿性肝病而被列为移植，1例有门静脉高压症的临床体征。MMF耐受性良好。结论：霉酚酸酯对AIH患儿是一种有效的抢救治疗，但对ASC患儿无效。(C)2009年由Elsevier B. V.代表欧洲肝脏研究协会发表。
Background/Aim: The aim of this study was to evaluate the outcome of mycophenolate mofetil (MM F) therapy in children with autoimmune liver disease who are resistant to or intolerant of standard immunosuppression.Methods: Inclusion criteria: (a) failure to achieve/maintain remission with prednisolone/azathioprine therapy or (b) significant treatment side-effects. Initial MMF dose was 20 mg/kg/day, gradually increased to a maximum of 40 mg/kg/day. Azathioprine was stopped when MMF was commenced.Results: Twenty-six children (17 female) were recruited. Median (range) age at diagnosis was 9.9 (1.2-14.4) years. Sixteen had Type I autoimmune hepatitis (AIH), two Type 2 AIH, and eight had autoimmune sclerosing cholangitis (ASC). Median (range) time from diagnosis to addition of MMF was 14.9 (0.2-108.6) months. Eighteen children responded to MMF, aspartate aminotransferase (AST) normalising in 14. At median (range) follow-up of 61.5 (19.5-96.3) months, AST remained normal in 12. All 18 children were well, but two had clinical signs of portal hypertension. Eight (6 ASC) did not respond: AST remained elevated in seven, one was listed for transplant for decompensated liver disease and one had clinical signs of portal hypertension. MMF was well tolerated. Leukopenia (n = 7) was the most common side-effect.Conclusions:MMF is an effective rescue therapy for children with AIH, but not for those with ASC. (C) 2009 Published by Elsevier B.V. on behalf of the European Association for the Study of the Liver.