Cost-effectiveness analysis of treatment with defibrotide for patients with severe sinusoidal obstruction syndrome

Introduction: Severe sinusoidal obstruction syndrome is a potentially fatal complication of hematopoietic stem cell transplantation that can only be pharmacologically treated with defibrotide. The present study was aimed to determine cost-effectiveness of defibrotide as a treatment of severe sinusoidal obstruction syndrome in clinical practice, since some studies suggest that we lack of large randomized trials which can provide evidence to manage this disease in adult patients. Method: This is an observational, retrospective cohort study of adult patients diagnosed with severe sinusoidal obstruction syndrome after hematopoietic stem cell transplantation. The effectiveness endpoints evaluated were overall survival and survival rates by days +100 and +180 after transplantation. Overall survival was described using the Kaplan-Meier method. Cost-effectiveness was described using the median-based cost-effectiveness ratio and a bootstrap procedure was performed as a sensitivity analysis. Twenty adult patients were included in this study. The median overall survival was 56 (95% CI: 15-NA) days. Nine (45%) patients were alive by day +100 after transplantation and three (15%) were alive by day +180. The median-based cost-effectiveness ratio was €800 per life-day gained (95% CI: €365-€2,753.6/day, calculated by bootstrap procedure). Conclusion: Data obtained favor the use of defibrotide for the treatment of severe sinusoidal obstruction syndrome since it has shown a good profile of effectiveness and safety and acceptable acquisition costs compared to the overall transplantation costs