Defibrotide: a review on clinical use and future development

BACKGROUND: Defibrotide is a deoxyribonucleic acid derivative that has been developed for the treatment of different vascular disorders. OBJECTIVE: The authors reviewed the literature to give due representation to the spectrum of pharmacological properties and clinical application of this drug, evaluating consolidate and innovative application. METHODS: The authors used PubMed from November 1982 to December 2007 and meeting abstracts (form American Society of Hematology Annual Meeting) with updated data as the sources for this review and selecting the most relevant papers when two or more articles covered the same point of interest. CONCLUSIONS: Defibrotide has been used effectively in the treatment of endothelial complications of allogeneic stem cell transplantation and recent preclinical evidences suggest an antiangiogenic effect and an anticancer activity. Further in vivo and in vitro investigations are needed.