title: Delayed immune recovery following sequential orthotopic liver transplantation and haploidentical stem cell transplantation in erythropoietic protoporphyria.
abstract: A nine-yr-old boy with EPP suffered from severe skin burns and liver failure caused by progressive cholestasis and fibrosis. OLT was performed without major complications. Four months following liver transplantation he underwent parental haploidentical HSCT. The myeloablative conditioning regimen was relatively well tolerated and hematological engraftment was rapid (on day 10). Protoporphyrin concentrations returned to normal following HSCT. However, immune recovery was significantly delayed. Varicella zoster virus reactivation resulted in impaired vision, prolonged hospitalization and eventually in multiorgan failure and death. Sequential liver and haploidentical HSCT proved feasible though a high risk procedure in this EPP patient. The management of post-IST after these combined transplantations remains a challenge and needs to be further established.
authors.affiliation: Division of Immunology, Hematology, Oncology, Bone marrow transplantation and Auto-immune disease, Department of Pediatrics, Leiden University Medical Center, Leiden, The Netherlands. f.j.smiers@lumc.nl
authors.forename: Frans J
authors.initials: FJ
authors.lastname: Smiers
authors.forename: Els
authors.initials: E
authors.lastname: Van de Vijver
authors.forename: Bas J P
authors.initials: BJ
authors.lastname: Delsing
authors.forename: Arjan C
authors.initials: AC
authors.lastname: Lankester
authors.forename: Lynne M
authors.initials: LM
authors.lastname: Ball
authors.forename: Edmund H H M
authors.initials: EH
authors.lastname: Rings
authors.forename: Patrick F
authors.initials: PF
authors.lastname: van Rheenen
authors.forename: Robbert G M
authors.initials: RG
authors.lastname: Bredius
mesh_terms: D002648:Child; D017809:Fatal Outcome; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006648:Histocompatibility; D006801:Humans; D016031:Liver Transplantation / Q000276:immunology*; D008297:Male; D046351:Protoporphyria, Erythropoietic / Q000276:immunology* / Q000601:surgery*; D019172:Transplantation Conditioning / Q000379:methods