title: The use of everolimus in pediatric liver transplant recipients: first experience in a single center.
abstract: The role of mTOR inhibitors, such as EVL, has not been established for pediatric liver transplant recipients up to now, although data from adult solid organ graft transplantation are very promising. Major complications following pediatric liver transplantation in the long-term course include chronic graft rejection and CNI-derived nephrotoxicity. The purpose of our study was to report first results using EVL as a rescue therapy in pediatric liver transplant recipients for the following indications: chronic graft dysfunction n=12, suspected CNI toxicity n=3, hepatoblastoma n=2, and recurrence of primary sclerosing cholangitis post-Ltx n=1. Four patients with chronic graft dysfunction developed completely normal liver function tests using EVL, six patients showed partial improvement, and two patients did not respond at all. One patient with CNI-induced nephropathy showed a slightly improved GFR. Both patients with hepatoblastoma did not develop any metastasis post-Ltx. First experience with EVL in pediatric liver transplant recipients shows promising results in patients with chronic graft failure when standard immunosuppression has failed. The future role of EVL in immunosuppressive protocols for children post-Ltx has to be proven by controlled clinical trials.
authors.affiliation: Departments of General Pediatrics Pediatric Hepatology and Liver Transplantation Hepatobiliary Surgery, University Medical Center Hamburg-Eppendorf, Hamburg, Germany.
authors.forename: Dirk
authors.initials: D
authors.lastname: Nielsen
authors.forename: Andrea
authors.initials: A
authors.lastname: Briem-Richter
authors.forename: Marijke
authors.initials: M
authors.lastname: Sornsakrin
authors.forename: Lutz
authors.initials: L
authors.lastname: Fischer
authors.forename: Bjoern
authors.initials: B
authors.lastname: Nashan
authors.forename: Rainer
authors.initials: R
authors.lastname: Ganschow
mesh_terms: D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D000068338:Everolimus; D005260:Female; D005919:Glomerular Filtration Rate; D006084:Graft Rejection; D006085:Graft Survival; D018197:Hepatoblastoma / Q000628:therapy; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D007223:Infant; D008111:Liver Function Tests; D016031:Liver Transplantation / Q000379:methods*; D008297:Male; D010372:Pediatrics / Q000379:methods*; D011446:Prospective Studies; D020123:Sirolimus / Q000031:analogs & derivatives* / Q000627:therapeutic use; D058570:TOR Serine-Threonine Kinases / Q000378:metabolism
chemical_list: D007166:Immunosuppressive Agents; D000068338:Everolimus; D058570:TOR Serine-Threonine Kinases; D020123:Sirolimus