title: Hepatic veno-occlusive disease with severe capillary leakage after peripheral stem cell transplantation: treatment with recombinant plasminogen activator and C1-esterase inhibitor concentrate.
abstract: Severe veno-occlusive disease (VOD), characterised by elevated serum bilirubin levels, is a known complication in the first 3 weeks after peripheral blood stem cell transplantation (PBSCT). Severe VOD is associated with capillary leakage and multiple organ dysfunction and leads to high mortality. We report a 17-year-old male, who developed VOD with capillary leakage (CL) after allogeneic PBSCT. The patient presented with a maximum serum bilirubin of 25.4 mg/dl, weight gain (10% of baseline weight), generalized edema, cardiovascular insufficiency, complement activation, jaundice and a decreased AT and protein C functional activity. After VOD and CL were diagnosed the patient was treated with recombinant human plasminogen activator (rt-PA) and C1 esterase-inhibitor concentrate (C1-INH-C). The clinical symptoms resolved and the patient's status stabilized. The patient was in an adequate clinical state 5 months after transplantation. We noted that the combined therapy with rt-PA and C1-INH-C in this high-risk situation led to a resolution of VOD with CL.
authors.affiliation: Department for Pediatric Hematology and Oncology, Heinrich-Heine-University Medical Center, Düsseldorf, Germany.
authors.forename: R
authors.initials: R
authors.lastname: Heying
authors.forename: W
authors.initials: W
authors.lastname: Nürnberger
authors.forename: U
authors.initials: U
authors.lastname: Spiekerkötter
authors.forename: U
authors.initials: U
authors.lastname: Göbel
mesh_terms: D000293:Adolescent; D002051:Burkitt Lymphoma / Q000628:therapy; D002199:Capillary Permeability / Q000187:drug effects; D003174:Complement C1 Inactivator Proteins / Q000008:administration & dosage*; D004359:Drug Therapy, Combination; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006504:Hepatic Veno-Occlusive Disease / Q000188:drug therapy* / Q000209:etiology* / Q000503:physiopathology; D006801:Humans; D008297:Male; D011994:Recombinant Proteins / Q000008:administration & dosage; D010959:Tissue Plasminogen Activator / Q000008:administration & dosage*; D014184:Transplantation, Homologous
chemical_list: D003174:Complement C1 Inactivator Proteins; D011994:Recombinant Proteins; D010959:Tissue Plasminogen Activator