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- W2006172316 abstract "Summary Hepatic veno‐occlusive disease (VOD) is a common complication of haematopoietic stem cell transplantation (HSCT), with reported incidences of 5–40% in children. Recently, defibrotide (DF) has been successfully used as prophylaxis and treatment of VOD. This study reports data on 63 human leucocyte antigen‐matched HSCT performed in 57 children affected by beta thalassemia at very high risk for developing VOD (liver fibrosis, iron overload, hepatitis C virus infections, busulphan‐based conditioning, methotraexate + ciclosporine). All patients received a busulphan‐based conditioning regimen, either orally (four HSCT) or intravenously (59 HSCT). All patients received oral DF (40 mg/kg per day, final dose) as VOD prophylaxis from median day −9 to median day +29. In order to overcome the lack of oral paediatric formulations, a galenic formulation was administered. DF was well tolerated. Only one patient fulfilled Seattle Criteria for VOD diagnosis. This patient had discontinued DF 6 d prior to VOD onset, due to high risk of haemorrhage. We concluded that oral defibrotide prophylaxis and i.v. busulphan safely abated VOD incidence in high‐risk patients who had undergone HSCT. A galenic preparation of oral DF also permits this treatment in low‐weight patients. Costs of DF prophylaxis are acceptable considering the reduced incidence of VOD." @default.
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- W2006172316 date "2009-10-27" @default.
- W2006172316 modified "2023-10-17" @default.
- W2006172316 title "Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan" @default.
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- W2006172316 doi "https://doi.org/10.1111/j.1365-2141.2009.07871.x" @default.
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