Defibrotide as salvage therapy for refractory veno-occlusive disease of the liver complicating allogeneic bone marrow transplantation

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 defibr otide (DF), a mammalian tissue-derived polydeoxyribonucleotide developed fo r the treatment of a number of vascular disorders, which has thrombolytic a nd anti-thrombotic properties. No significant bleeding or other major toxic ities were observed during treatment and she made a full recovery. At 6 mon ths 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 prospectiv e randomized fashion.