Clinical Pharmacy Services Publications

A successful veno-occlusive disease (VOD) prophylaxis strategy for patients undergoing stem cell transplantation

David C. Gammon, University of Massachusetts Medical School
Kristi Holt
Traci M. Dutton
Janet Rogers, UMass Memorial Medical Center
Samer Al-Homsi


Veno-occlusive disease (VOD) is a common and serious complication associated with stem cell transplant regimens. Risk factors for VOD include chemotherapy and irradiation used in pre-transplant conditioning. Certain transplant conditioning regimens including drugs such as cytarabine, cyclophosphamide, and busulfan, as well as total body irradiation increase the risk of VOD. Because of the serious, often fatal, outcomes associated with VOD in transplant patients, strategies have been proposed to prevent it without consensus, some including the use of heparin. Ursodiol, studied in the past for the prevention or treatment of veno-occlusive disease, is thought to increase biliary flow, decrease biliary viscosity, and protect hepatocytes from harmful hydrophobic bile acids.

Methods: Twenty-five patients admitted between February and December 2007 were identified as having received ursodiol as part of their transplant regimens. Seventeen of the twenty-five patients received at least one of the treatment regimens including cytarabine, cyclophosphamide, busulfan, and/or total body irradiation. None of the patients received prophylactic heparin for VOD. The records of each patient were screened retrospectively for documentation of veno-occlusive disease.

Results: None of the twenty-five patients who received Ursodiol as part of their stem cell transplant regimen developed VOD.

Conclusion: Stem cell transplant patients are at risk for VOD, based on their conditioning regimens. Ursodiol was used as VOD prophylaxis in twenty-five patients, none of whom developed VOD. Ursodiol should be further studied as a prophylactic part of stem cell transplant regimens.