title: Combined liver-kidney transplantation for children with autosomal recessive polycystic kidney disease (ARPKD): indication and outcome.
abstract: In ARPKD, mutations in the PKHD1 gene lead to remodeling of the kidneys and liver. These may result in progressive liver fibrosis with portal hypertension requiring combined liver and kidney transplantation (CLKT). There is currently no consensus on the indication for CLKT and data on long-term outcomes are scarce. We analyzed in detail the pretransplant liver symptomatology, laboratory and ultrasound data, histological studies, and genotypes in eight patients undergoing CLKT. The median age was 10.1 years (range 1.7-16) and median follow-up was 4.6 years (range 1.1-8.9). All patients had clinical signs of portal hypertension and abnormal ultrasound findings. Congenital hepatic fibrosis was present in all pretransplant biopsies (6 out of 8 patients) and in all explanted livers. All patients survived; liver and kidney graft survival was 72% and 88%, respectively. Liver and kidney function were stable in all patients with a median eGFR of 70 ml/min/1.73 m² (range 45-108 ml/min/1.73 m²). Height-SDS improved significantly after 12, 24, and 36 months (P = 0.016, 0.022 and 0.018 respectively). The indication for CLKT remains challenging and controversial. A favorable outcome for patients with ARPKD can be achieved by using the degree of portal hypertension, longitudinal ultrasound examinations, and preoperative liver histology as parameters for CLKT.
authors.affiliation: Department of Pediatrics, Pediatric Gastroenterology and Hepatology, University Medical Center Hamburg-Eppendorf, Hamburg, Germany. f.brinkert@uke.de
authors.forename: Florian
authors.initials: F
authors.lastname: Brinkert
authors.forename: Anja
authors.initials: A
authors.lastname: Lehnhardt
authors.forename: Carmen
authors.initials: C
authors.lastname: Montoya
authors.forename: Knut
authors.initials: K
authors.lastname: Helmke
authors.forename: Hansjoerg
authors.initials: H
authors.lastname: Schaefer
authors.forename: Lutz
authors.initials: L
authors.lastname: Fischer
authors.forename: Bjoern
authors.initials: B
authors.lastname: Nashan
authors.forename: Carsten
authors.initials: C
authors.lastname: Bergmann
authors.forename: Rainer
authors.initials: R
authors.lastname: Ganschow
authors.forename: Markus J
authors.initials: MJ
authors.lastname: Kemper
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D005260:Female; D006085:Graft Survival; D006801:Humans; D006975:Hypertension, Portal / Q000473:pathology / Q000601:surgery; D007223:Infant; D007676:Kidney Failure, Chronic / Q000473:pathology / Q000601:surgery; D016030:Kidney Transplantation; D008099:Liver / Q000000981:diagnostic imaging / Q000473:pathology*; D008103:Liver Cirrhosis / Q000473:pathology; D016031:Liver Transplantation; D008297:Male; D017044:Polycystic Kidney, Autosomal Recessive / Q000235:genetics / Q000473:pathology / Q000601:surgery*; D011956:Receptors, Cell Surface / Q000235:genetics; D012189:Retrospective Studies; D014463:Ultrasonography
chemical_list: C451750:PKHD1 protein, human; D011956:Receptors, Cell Surface