HOMEPRODUCTSCOMPANYCONTACTFAQResearchDictionaryPharmaSign Up FREE or Login

Defibrotide for the treatment of hepatic veno-occlusive disease: results of the European compassionate-use study.

Abstract
Severe hepatic veno-occlusive disease (VOD) is a recognized complication of autologous and allogeneic stem cell transplantation (SCT) that is often fatal. Defibrotide (DF) is a polydeoxyribonucleotide that has been found to have anti-thrombotic, anti-ischaemic and thrombolytic properties without causing significant anticoagulation. Preliminary studies have demonstrated activity for DF in the treatment of VOD, with minimal associated toxicity. In the present study, 40 patients who fulfilled established criteria for VOD were treated with DF on compassionate grounds in 19 European centres; 28 patients met risk criteria predicting progression of VOD and fatality or had evidence of multiorgan failure (MOF), and were defined as 'poor-risk'. DF was commenced intravenously at a median of 14 d (range, -2 d to 53 d) post SCT at doses ranging from 10 to 40 mg/kg. The median duration of therapy was 18 d (range, 2--71 d). Twenty-two patients showed a complete response (CR) (bilirubin < 34.2 micromol/l and resolution of signs/symptoms of VOD and end-organ dysfunction) [CR = 55%, confidence interval (CI) 40--70%] and 17 patients (43%) are alive beyond d +100. Ten poor-risk patients showed a complete response (CR = 36%, CI 21--51%). These results demonstrate that DF is an active treatment for VOD following SCT and a randomized trial is now underway in order to further evaluate its role.
AuthorsR Chopra, J D Eaton, A Grassi, M Potter, B Shaw, C Salat, P Neumeister, G Finazzi, M Iacobelli, K Bowyer, H G Prentice, T Barbui
JournalBritish journal of haematology (Br J Haematol) Vol. 111 Issue 4 Pg. 1122-9 (Dec 2000) ISSN: 0007-1048 [Print] England
PMID11167751 (Publication Type: Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
Chemical References
  • Fibrinolytic Agents
  • Polydeoxyribonucleotides
  • defibrotide
  • Bilirubin
Topics
  • Acute Disease
  • Adolescent
  • Adult
  • Bilirubin (analysis)
  • Breast Neoplasms (drug therapy, surgery)
  • Child
  • Child, Preschool
  • Female
  • Fibrinolytic Agents (therapeutic use)
  • Hematopoietic Stem Cell Transplantation
  • Hepatic Veno-Occlusive Disease (blood, drug therapy)
  • Hodgkin Disease (drug therapy, surgery)
  • Humans
  • Infant
  • Leukemia, Myeloid (drug therapy, surgery)
  • Male
  • Middle Aged
  • Multiple Myeloma (drug therapy, surgery)
  • Polydeoxyribonucleotides (therapeutic use)
  • Postoperative Complications (drug therapy)
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma (drug therapy, surgery)
  • Treatment Outcome

Join CureHunter, for free Research Interface BASIC access!

Take advantage of free CureHunter research engine access to explore the best drug and treatment options for any disease. Find out why thousands of doctors, pharma researchers and patient activists around the world use CureHunter every day.
Realize the full power of the drug-disease research graph!


Choose Username:
Email:
Password:
Verify Password:
Enter Code Shown: