Paul Richardson, MD, Dana-Farber Cancer Institute, Boston, MA, shares his views on the results of a Phase III study comparing the safety and efficacy of defibrotide versus best supportive care in the prevention of hepatic veno-occlusive disease (VOD) in children and adults undergoing hematopoietic stem cell transplantation (HSCT) who are at a high risk of developing VOD (NCT02851407). The primary endpoint of this study, incidence of VOD, was diagnosed by investigators and assessed objectively by an Endpoint Adjudication Committee (EPAC). The study did not detect any new safety signals for patients in the defibrotide group. Moreover, whilst the investigator group reported a higher rate of VOD in patients treated with defibrotide, EPAC did not find a significant difference between both treatment arms in terms of preventing VOD, which led to the interruption of the study. These findings highlight the challenges in VOD diagnosis. In addition, Dr Richardson explains that due to regulatory reasons, an important number of patients treated with sirolimus at high risk of developing VOD were not included, which limited enrolment significantly. In addition, previous real-world experiences have suggested a benefit for the prophylactic use of defibrotide to prevent VOD. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.
