Dr Braunstein on Novel Risk Factors for Veno-Occlusive Disease Development

Marc J. Braunstein, MD, PhD, associate professor, Department of Medicine, co-director, Hematology-Oncology System, New York University (NYU) Grossman Long Island School of Medicine, discusses risk factors for veno-occlusive disease (VOD), management strategies, and prevention strategies in the post–hematopoietic stem cell transplant (HSCT) setting for patients with hematologic malignancies.

Prophylaxis for VOD is administered to most patients undergoing HSCT, but vigilance in identifying this disease is crucial, especially for patients with elevated risk, Braunstein begins. Specific patient-related risk factors include a history of liver or lung disease, older age, and poor baseline performance status prior to transplant, according to Braunstein. Transplant-related factors also play a role in VOD risk, he notes. Additionally, prior exposure to certain agents, such as gemtuzumab ozogamicin (Mylotarg), may elevate VOD risk, as could the use of certain agents used as prophylaxis for graft-versus-host-disease (GVHD), he states.

VOD is a leading cause of mortality in the post-HSCT setting, surpassing GVHD, Braunstein reports. Given its prevalence, effective prevention and treatment strategies are essential, he says. All patients with hematologic malignancies receiving treatment at NYU receive prophylactic ursodiol, which is initiated on day 0 of transplant and continued for approximately 60 days post-transplant, he explains. Ursodiol serves as the primary preventive agent in this field, he notes. Additionally, certain modifiable risk factors can be addressed, such as avoiding pre-transplant hepatotoxic agents like gemtuzumab ozogamicin, optimizing therapies to reduce hepatotoxicity, and ensuring that liver conditions, such as hepatitis, are managed preemptively, he emphasizes.

For VOD management, defibrotide (Defitelio) remains the standard of care (SOC), Braunstein reports. Early VOD intervention is critical, regardless of disease severity, he adds. Some institutions classify patients by VOD severity—mild, moderate, or severe—based on symptoms and organ dysfunction, he states. However, defibrotide is the primary therapeutic agent across all disease categories, according to Braunstein. Additional supportive measures can help avoid volume overload and minimize patient exposure to agents that may impair liver function, he notes.

There is no established SOC for patients who with refractory VOD and unresponsive to defibrotide, Braunstein says. Some guidelines suggest the use of steroids for advanced, refractory VOD, although defibrotide remains the cornerstone of VOD management, he concludes.