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

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 RCTs 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.