title: Defibrotide as salvage therapy for refractory veno-occlusive disease of the liver complicating allogeneic bone marrow transplantation.
abstract: A 30-year-old woman developed veno-occlusive disease of the liver during an allogeneic BMT for acute leukemia. Treatment with recombinant human tissue plasminogen activator and heparin resulted in an incomplete and transient response followed by progressive disease. The patient was then given defibrotide (DF), a mammalian tissue-derived polydeoxyribonucleotide developed for the treatment of a number of vascular disorders, which has thrombolytic and anti-thrombotic properties. No significant bleeding or other major toxicities were observed during treatment and she made a full recovery. At 6 months after the onset of VOD her liver function tests and color flow Doppler ultrasound scan are normal. Our experience supports the preliminary results already obtained with DF. Its efficacy should be evaluated in a prospective randomized fashion.
authors.affiliation: BMT Unit, Instituto Português de Oncologia, Lisbon.
authors.forename: M M
authors.initials: MM
authors.lastname: Abecasis
authors.forename: J P
authors.initials: JP
authors.lastname: Conceição Silva
authors.forename: I
authors.initials: I
authors.lastname: Ferreira
authors.forename: A
authors.initials: A
authors.lastname: Guimarães
authors.forename: A
authors.initials: A
authors.lastname: Machado
mesh_terms: D000328:Adult; D016026:Bone Marrow Transplantation / Q000009:adverse effects*; D005260:Female; D005343:Fibrinolytic Agents / Q000627:therapeutic use*; D006504:Hepatic Veno-Occlusive Disease / Q000188:drug therapy*; D006801:Humans; D011089:Polydeoxyribonucleotides / Q000627:therapeutic use*; D016879:Salvage Therapy; D010959:Tissue Plasminogen Activator / Q000627:therapeutic use; D014184:Transplantation, Homologous
chemical_list: D005343:Fibrinolytic Agents; D011089:Polydeoxyribonucleotides; C036901:defibrotide; D010959:Tissue Plasminogen Activator