In 2019, defibrotide (DF) was approved for use to treat sinusoidal obstruction syndrome in Japan. However, few studies in the literature report on its use in children. We retrospectively analyzed cases of pediatric patients who underwent hematopoietic cell transplantation and developed sinusoidal obstruction syndrome, which was treated with DF in our center. Four patients with myelodysplastic syndrome (n=1), X-linked inhibitor of apoptosis protein (XIAP) deficiency (n=1), neuroblastoma (n=1), and acute lymphoblastic leukemia (n=1) were included in the study. Total bilirubin levels decreased after the start of DF therapy in all cases, except in the patient with acute lymphoblastic leukemia, who died immediately after starting DF therapy owing to worsening of the primary disease. Of the three patients whose total bilirubin levels decreased, two recovered and one died because of multiple organ failure. Apparent adverse events with DF administration were not observed, and a decrease in total bilirubin levels early in the treatment period tended to be a predictor of treatment response. It is hoped that more reports of patients with DF administration in Japan will be accumulated in the future.

View full abstract