title: Drug safety evaluation of  defibrotide.
abstract: BACKGROUND
  Hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is a potentially life-threatening complication of chemotherapeutic conditioning used in preparation for hematopoietic stem-cell transplantation (SCT). Defibrotide (DF) has been shown in Phase II and III trials to improve complete response in patients with severe VOD (sVOD). None of the articles, to date, provide a comprehensive review of the safety of DF in VOD and/or a range of other conditions.
  
  
  METHODS
  This article reviews current clinical findings on DF, primarily in terms of safety for use in treatment and prophylaxis of VOD, and relevant safety data for its use in other diseases. The literature review was conducted using a PubMed search with the fixed term 'defibrotide' in combination with ≥ 1 of 'safety', 'veno-occlusive disease' (with and without 'treatment', 'prevention'), 'oncology', 'myeloma', 'microangiopathy', 'anti-thrombotic' and 'peripheral vascular disorder'. Related articles from the EBMT and ASH conference websites were also included.
  
  
  CONCLUSIONS
  DF was well tolerated in majority of the studies. The safety profile of DF is largely favourable with toxicities comparable to control populations in the setting of SCT complicated by sVOD.
authors.affiliation: Dana-Farber Cancer Institute, Medical Oncology, 450 Brookline Avenue, Mayer 232, Boston 02215, USA. paul_richardson@dfci.harvard.edu
authors.forename: Paul G
authors.initials: PG
authors.lastname: Richardson
authors.forename: Selim
authors.initials: S
authors.lastname: Corbacioglu
authors.forename: Vincent Trien-Vinh
authors.initials: VT
authors.lastname: Ho
authors.forename: Nancy A
authors.initials: NA
authors.lastname: Kernan
authors.forename: Leslie
authors.initials: L
authors.lastname: Lehmann
authors.forename: Craig
authors.initials: C
authors.lastname: Maguire
authors.forename: Michelle
authors.initials: M
authors.lastname: Maglio
authors.forename: Margaret
authors.initials: M
authors.lastname: Hoyle
authors.forename: Marco
authors.initials: M
authors.lastname: Sardella
authors.forename: Sergio
authors.initials: S
authors.lastname: Giralt
authors.forename: Ernst
authors.initials: E
authors.lastname: Holler
authors.forename: Enric
authors.initials: E
authors.lastname: Carreras
authors.forename: Dietger
authors.initials: D
authors.lastname: Niederwieser
authors.forename: Robert
authors.initials: R
authors.lastname: Soiffer
mesh_terms: D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006504:Hepatic Veno-Occlusive Disease / Q000175:diagnosis / Q000188:drug therapy* / Q000209:etiology; D006801:Humans; D018579:Patient Selection; D011089:Polydeoxyribonucleotides / Q000009:adverse effects / Q000627:therapeutic use*; D018570:Risk Assessment; D012307:Risk Factors; D016896:Treatment Outcome
chemical_list: D011089:Polydeoxyribonucleotides; C036901:defibrotide