title: Liver Transplantation in Polish Children With α1-Antitrypsin Deficiency: A Single-Center Experience.
abstract: BACKGROUND
  α1-Antitrypsin deficiency (ATD) is the most common genetic cause of liver injury in young children. Asymptomatic hepatitis is observed in most patients. However, the course of liver disease due to ATD is unpredictable, and some children develop liver cirrhosis. Liver transplantation (Ltx) dramatically improves their outcome and in some cases is required in the first years of life. The aim of the study was to evaluate the course of the disease in children with ATD treated with Ltx in a single center.
  
  
  METHODS
  We retrospectively reviewed the clinical features (ascites, esophageal varices, esophageal bleeding) and laboratory parameters of liver function in children with ATD who were treated with Ltx.
  
  
  RESULTS
  Twenty-two Ltxs were performed in 20 children (13 boys, 7 girls). Median age at transplantation was 12 years (range 0.5 to 17.1). Four children were transplanted in the first 2 years of life and 16 patients were over 7 years old. The indications for Ltx in younger children were progressive cholestasis with coagulopathy and ascites. In older patients, the indications were as follows: liver failure presenting with variceal bleeding in 7 patients, ascites in 5 patients, hypersplenism in all but 1 patient. In the group of children transplanted over 7 years old, the frequency of cholestasis decreased intermittently in the second year of life: 4 patients (25%) compared to 15 patients (94%) and 10 patients (63%) in the neonatal and pretransplant period, respectively. In the group of children transplanted earlier, cholestasis and hepatitis were maintained until Ltx. Of transplanted patients, 50% were malnourished at the transplantation, and 50% were followed for more than 10 years. Five-year post-transplant survival was 100% (n = 14), and 10-year survival was 90%. Two patients died as adults with biliary post-transplant complications and problems with compliance.
  
  
  CONCLUSIONS
  Our experience suggests that transient normalization of liver parameters in some patients with ATD do not exclude the liver disease progression to cirrhosis and unfavorable outcome of liver disease in childhood. In our group of patients, median age at transplantation was high compared to other centers. The long-term prognosis in children after transplantation is very good, but early post-transplant complications and probable problems with compliance in young adults may lead to graft failure.
authors.affiliation: Gastroenterology, Hepatology, Nutrition Disorders and Paediatrics, The Children's Memorial Health Institute, Warsaw, Poland. Electronic address: agnieszkabakula@gmail.com.
authors.forename: A
authors.initials: A
authors.lastname: Bakula
authors.affiliation: Gastroenterology, Hepatology, Nutrition Disorders and Paediatrics, The Children's Memorial Health Institute, Warsaw, Poland.
authors.forename: J
authors.initials: J
authors.lastname: Pawlowska
authors.affiliation: Gastroenterology, Hepatology, Nutrition Disorders and Paediatrics, The Children's Memorial Health Institute, Warsaw, Poland.
authors.forename: O
authors.initials: O
authors.lastname: Niewiadomska
authors.affiliation: Gastroenterology, Hepatology, Nutrition Disorders and Paediatrics, The Children's Memorial Health Institute, Warsaw, Poland.
authors.forename: I
authors.initials: I
authors.lastname: Jankowska
authors.affiliation: Gastroenterology, Hepatology, Nutrition Disorders and Paediatrics, The Children's Memorial Health Institute, Warsaw, Poland.
authors.forename: M
authors.initials: M
authors.lastname: Teisseyre
authors.affiliation: Pediatric Surgery and Organ Transplantation, The Children's Memorial Health Institute, Warsaw, Poland.
authors.forename: P
authors.initials: P
authors.lastname: Kaliciński
authors.affiliation: Gastroenterology, Hepatology, Nutrition Disorders and Paediatrics, The Children's Memorial Health Institute, Warsaw, Poland.
authors.forename: P
authors.initials: P
authors.lastname: Socha
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D002779:Cholestasis / Q000209:etiology / Q000601:surgery; D004932:Esophageal and Gastric Varices / Q000209:etiology; D005260:Female; D006471:Gastrointestinal Hemorrhage / Q000209:etiology; D006085:Graft Survival; D006801:Humans; D006971:Hypersplenism / Q000209:etiology; D007223:Infant; D017093:Liver Failure / Q000209:etiology / Q000601:surgery*; D016031:Liver Transplantation / Q000379:methods*; D008297:Male; D011044:Poland; D011379:Prognosis; D012189:Retrospective Studies; D019896:alpha 1-Antitrypsin Deficiency / Q000150:complications / Q000601:surgery*