Prevention and treatment of veno-occlusive disease

Ann Pharmacother. 2001 Jul-Aug;35(7-8):935-42. doi: 10.1345/aph.10220.


Objective: To review the current options for prevention and treatment of veno-occlusive disease in bone marrow transplant patients.

Data sources: Articles were selected from a MEDLINE search (1966-October 1999) using the key terms veno-occlusive disease and bone marrow transplantation. In addition, references of all articles were examined for articles not found in the computer-based search.

Data extraction: All clinical trials, case-control studies, and case reports were evaluated.

Results: Heparin, low-molecular-weight heparin, prostaglandin E1, ursodiol, and glutamine have been studied for prevention of veno-occlusive disease. Heparin has been studied most extensively; however, no preventive regimen has a defined role in therapy. For treatment, tissue plasminogen activator has been evaluated most thoroughly, yet its safety and efficacy have not been clearly established in patients with veno-occlusive disease. Other possible treatment options include antithrombin-III, defibrotide, glutamine plus vitamin E, and surgery.

Conclusions: Based on the available data, the most promising agents are ursodiol for prevention and defibrotide or glutamineplus vitamin E for treatment of veno-occlusive disease. Further clinical trials are needed to establish the appropriate preventive and treatment options available for bone marrow transplant patients suffering from veno-occlusive disease. To date, such decisions depend largely on poorly designed trials, case reports, and clinical experience.

Publication types

  • Review

MeSH terms

  • Anticoagulants / therapeutic use*
  • Bone Marrow Transplantation / adverse effects*
  • Clinical Trials as Topic
  • Heparin / therapeutic use*
  • Hepatic Veno-Occlusive Disease* / etiology
  • Hepatic Veno-Occlusive Disease* / physiopathology
  • Hepatic Veno-Occlusive Disease* / prevention & control
  • Humans


  • Anticoagulants
  • Heparin