title: Sickle cell hepatopathy: clinical presentation, treatment, and outcome in pediatric and adult patients.
abstract: BACKGROUND
  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.
  
  
  METHODS
  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).
  
  
  RESULTS
  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).
  
  
  CONCLUSIONS
  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.
authors.affiliation: Department of Hematology/Oncology, St. Jude Children's Research Hospital, Memphis, Tennessee 38105, USA.
authors.forename: Hyunah
authors.initials: H
authors.lastname: Ahn
authors.forename: Chin-Shang
authors.initials: CS
authors.lastname: Li
authors.forename: Winfred
authors.initials: W
authors.lastname: Wang
mesh_terms: D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000150:complications*; D001803:Blood Transfusion / Q000379:methods; D002648:Child; D002675:Child, Preschool; D002780:Cholestasis, Intrahepatic / Q000175:diagnosis* / Q000209:etiology / Q000628:therapy*; D005260:Female; D006801:Humans; D006932:Hyperbilirubinemia / Q000175:diagnosis / Q000209:etiology / Q000628:therapy; D008297:Male; D012189:Retrospective Studies; D018709:Statistics, Nonparametric; D016896:Treatment Outcome