Defibrotide: Real World Management of Veno-Occlusive Disease/ Sinusoidal Obstructive Syndrome after Stem Cell Transplant.

Hepatic veno-occlusive disease or sinusoidal obstructive syndrome (VOD/ SOS) is a life-threatening complication of hematopoietic stem cell transplantation (HSCT). Defibrotide is the only FDA-approved medication for the management of severe VOD/ SOS after HSCT. We report our center's experience with commercially available defibrotide as treatment for patients with VOD/SOS. We retrospectively identified 28 cases of VOD/ SOS, based on the European Society for Blood and Marrow Transplantation criteria, from March 2016 through June 2019. The median day of VOD/ SOS onset was 25 days (range, 8 to 69) and defibrotide was initiated on day of diagnosis in 71% of patients. Complete resolution of VOD/ SOS occurred in 75% of patients. Day +100 survival was 64% for all HSCT patients and 53% for those with very severe VOD/ SOS. Response rates and survival were similar in patients with VOD/SOS after myeloablative or reduced intensity chemotherapy HSCT. Therapy related adverse events were mild and included hematuria (43%), epistaxis (18%) and hypotension (11%). Severe hemorrhagic adverse events occurred in 2 patients (pulmonary and upper gastrointestinal hemorrhage; 7%) and both in the setting of progressive VOD/SOS. Early diagnosis, prompt initiation of defibrotide, and minimizing dosing interruptions may be key to successful treatment of VOD/ SOS.