Defibrotide for the prevention of hepatic veno‐occlusive disease after hematopoietic stem cell transplantation: a systematic review

Abstract Prophylactic use of defibrotide ( DF ) to prevent veno‐occlusive disease ( VOD ), a relatively common and high‐risk complication of hematopoietic stem cell transplantation ( HSCT ), may be an encouraging modality to reduce morbidity and mortality from VOD . However, conclusions remain unclear. We carried out a systematic review to summarize the state of knowledge. One randomized controlled trial ( RCT ), four cohort studies and eight case series studies were found, including a total of 1230 patients. The overall mean incidence of VOD in patients using DF was 4.7% (95% CI , 3.3–6.1) which was significantly lower than the data 13.7% (95% CI , 13.3–14.1) across 135 studies using no VOD prophylaxis (p < 0.005). The meta‐analysis of the incidence of VOD in controlled trials revealed a statistical reduction in VOD incidence in the DF group ( RR = 0.47, 95% CI , 0.31–0.73). The overall mean incidence of severe VOD was 0.8% (95% CI , 0.2–1.4). The RR was 0.31 (95% CI , 0.09–1.06). However, the lack of RCT s and the methodological weaknesses of the studies may preclude making generalizable conclusions. Our review described that DF appears promising for VOD prevention and large RCT is needed for further confirmation.