Ahn Hyunah, Li Chin-Shang, Wang Winfred
Department of Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.
Pediatr Blood Cancer. 2005 Aug;45(2):184-90. doi: 10.1002/pbc.20317.
Standard diagnostic criteria and therapy are lacking for sickle cell hepatopathy, an uncommon complication of sickle cell disease. Here we propose diagnostic and therapeutic guidelines based on our experience and on reported cases.
We defined sickle hepatopathy by a total serum bilirubin concentration >13 mg/dl not explained by severe acute hemolysis, viral hepatitis, extrahepatic obstruction, or hepatic sequestration. We reviewed the records of all children with sickle hepatopathy seen at our institution during the past 20 years and the reported cases from the literature. Patients were categorized into two groups based on whether hepatic dysfunction at presentation was mild (Group I) or severe (Group II).
Seven patients were identified from our institution and 37 patients from the literature. The 44 patients were evenly divided between the two groups. Group I patients had a significantly lower mean age (11.8 years vs. 24.5 years, P = 0.0001), maximum bilirubin level (36.2 mg/dl vs. 76.8 mg/dl, P = 0.0008), and frequency of treatment with exchange transfusions (P = 0.03). Overall, mortality was 4% in Group I and 64% in Group II (P = 0.0001). Gender and recurrence rate did not differ. Among Group II patients, only two of nine who received exchange transfusion died, whereas 12 of 13 who did not receive exchange transfusion died (P = 0.0015).
Sickle cell hepatopathy is an uncommon complication characterized by extreme hyperbilirubinemia and either mild or severe hepatic dysfunction. Children and adults can present with either form; however, adults have a higher frequency of the severe form. Exchange transfusion may be the only effective management for initial episodes of severe sickle cell hepatopathy.
镰状细胞性肝病是镰状细胞病的一种罕见并发症，目前缺乏标准的诊断标准和治疗方法。在此，我们根据自身经验及报告病例提出诊断和治疗指南。
我们将血清总胆红素浓度>13mg/dl且无法用严重急性溶血、病毒性肝炎、肝外梗阻或肝内潴留解释的情况定义为镰状细胞性肝病。我们回顾了过去20年在本机构就诊的所有镰状细胞性肝病患儿的病历以及文献中报告的病例。根据就诊时肝功能障碍是轻度（I组）还是重度（II组）将患者分为两组。
我们机构识别出7例患者，文献中识别出37例患者。44例患者在两组中平均分配。I组患者的平均年龄显著更低（11.8岁对24.5岁，P = 0.0001）、最高胆红素水平更低（36.2mg/dl对76.8mg/dl，P = 0.0008），换血治疗频率更低（P = 0.03）。总体而言，I组死亡率为4%，II组为64%（P = 0.0001）。性别和复发率无差异。在II组患者中，接受换血治疗的9例中仅2例死亡，而未接受换血治疗的13例中有12例死亡（P = 0.0015）。
镰状细胞性肝病是一种罕见并发症，其特征为极度高胆红素血症以及轻度或重度肝功能障碍。儿童和成人均可出现任何一种形式；然而，成人中重度形式的发生率更高。换血治疗可能是重度镰状细胞性肝病初始发作的唯一有效治疗方法。
