Veno-occlusive disease (VOD) is a rare disease most often occurring as a result of a conditioning treatment administered prior to hematopoietic stem cell transplantation (HSCT). Defibrotide (Defitelio®) is a large, single-stranded deoxyribonucleotide with antithrombotic, profibrinolytic and anti-inflammatory effects.

In 2013, the EMA granted marketing authorisation for Defitelio® "for the treatment of severe VOD, also known as sinusoidal obstructive syndrome, (SOS) in HSCT therapy". The market authorisation was granted "under exceptional circumstances", and Defitelio® is under additional monitoring. Defibrotide is not approved for the prophylaxis of VOD. In the U.S., Defitelio® has not been approved by the FDA, but it is available under an expanded-access protocol.

Overall, the strength of evidence for the effectiveness and safety of defibrotide for the prophylaxis of VOD was very low to moderate. Even though there are indications that the prophylaxis of VOD with defibrotide reduces the incidence of VOD at least in children at high risk, and few adverse events are associated with this therapy, an inclusion in the Austrian catalogue of benefits for an off-label indication cannot be recommended. A re-evaluation is recommended once the licensing status of the drug changes. Overall, the strength of evidence for the effectiveness and safety of defibrotide for the treatment of VOD is very low. The current evidence is not sufficient to evaluate the effectiveness and safety of defibrotide for the treatment of VOD. Since VOD is a rare and life-threatening disease with no other therapeutic options available, defibrotide offers a new therapeutic approach for patients with severe VOD and meets an area of high unmet clinical need. Therefore, an inclusion in the catalogue of benefits is recommended with restrictions.