Department of Pediatrics and Division of Pediatric Gastroenterology and Hepatology, University Hospital Santa Chiara, Pisa, Italy.
J Pediatr. 2011 Jul;159(1):127-132.e1. doi: 10.1016/j.jpeds.2010.12.050. Epub 2011 Feb 24.
To assess the outcome of giant cell hepatitis combined with autoimmune hemolytic anemia in early childhood.
We report on 16 children with this disease evaluated over a 28-year period.
Children (nine boys; median age, 6 months) presented with jaundice, hepatomegaly, elevated aminotransferases, a positive Coombs test, and diffuse giant-cell transformation of hepatocytes on histology. Treatment with prednisone and azathioprine, plus, in three children, cyclosporine, resulted in complete remission in eight, partial remission in six, and failure in two. Relapses of hepatitis and/or anemia occurred in 11 and 10 children, respectively, requiring prolonged high levels of immunosuppression, and splenectomy or Rituximab, respectively. Treatment was stopped after a mean duration of 6 years, with no relapse, in seven children, with a median follow-up of 14 years. One child is alive 9 years after liver transplantation. Four children died of sepsis or multiple organ failure.
Giant cell hepatitis combined with autoimmune hemolytic anemia requires rigorous treatment. Immunosuppressive therapy results in remission in most cases. A complete cure can be expected after several years of intensive treatment. Liver transplantation may be associated with prolonged survival.
评估儿童期巨细胞肝炎合并自身免疫性溶血性贫血的结局。
我们报告了 16 例患有该病的儿童，评估时间为 28 年。
患儿（9 名男孩；中位年龄 6 个月）表现为黄疸、肝大、转氨酶升高、Coombs 试验阳性和组织学上弥漫性巨细胞转化。泼尼松和硫唑嘌呤治疗，加用环孢素治疗 3 例患儿，结果 8 例完全缓解，6 例部分缓解，2 例无效。分别有 11 例和 10 例患儿出现肝炎和/或贫血复发，分别需要长期高剂量免疫抑制治疗和脾切除术或利妥昔单抗治疗。在中位随访 14 年后，7 例患儿停止治疗 6 年后无复发。1 例患儿在肝移植后 9 年仍存活。4 例患儿死于脓毒症或多器官功能衰竭。
巨细胞肝炎合并自身免疫性溶血性贫血需要严格治疗。免疫抑制治疗可使大多数患儿缓解。经过数年强化治疗，有望获得完全治愈。肝移植可能与延长生存相关。
