Defibrotide is indicated for the treatment of severe hepatic VOD in HSCT therapy in adults and infants aged over 1 month.
(PRWEB UK) 2 June 2015
European Oncology & Haematology, a peer-reviewed, open access, bi-annual oncology journal, published a cutting-edge article by Katrina Mountfort.
Abstract: Severe veno-occlusive disease (VOD) is a serious and life-threatening complication of haematopoietic stem cell transplantation (HSCT), for which the standard of care has until recently been supportive care. VOD is the result of a primary injury to sinusoidal endothelial cells and severe VOD is characterised by sinusoidal narrowing and occlusion, which leads to portal hypertension, MOF and, ultimately, death. Defibrotide regulates multiple pathways involved in the pathological processes underlying VOD and is the first drug to be approved in Europe for the treatment of severe VOD. Defibrotide is indicated for the treatment of severe hepatic VOD in HSCT therapy in adults and infants aged over 1 month. A phase III study found significant increases in complete response (CR) and survival with defibrotide compared with historical controls. These data together with earlier studies and an ongoing expanded access protocol in a large patient cohort demonstrate improved outcomes with defibrotide in severe VOD and highlight the importance of treatment with defibrotide.
Haematopoietic stem cell transplantation (HSCT) has become the standard of care for many haematological malignancies, selected solid tumours and some non-malignant disorders. However, it can be associated with serious complications, in particular, veno-occlusive disease (VOD). Approximately 14% of HSCT patients develop VOD, although incidences of up to 60% have been reported. Incidence is higher in children and is partly due to certain malignant and inherited diseases that are associated with a substantially increased risk of VOD during HSCT.4 VOD can also occur in cancer patients undergoing aggressive chemotherapy regimens and has been reported after solid organ transplantation. VOD places a significant burden on healthcare providers and can add considerable additional expense to HSCT costs.5 VOD presents with different spectrums of severity, from mild VOD, which is self-limiting and requires no treatment, to severe VOD, which is associated with a mortality of over 80%.
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