Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan

作者：Cappelli Barbara; Chiesa Robert; Evangelio Costanza; Biffi Alessandra; Roccia Tito; Frugnoli Ilaria; Biral Erika; Noe Anna; Fossati Marco; Finizio Valentina; Miniero Roberto; Napolitano Sara; Ferrua Francesca; Soliman Clara; Ciceri Fabio; Roncarolo Maria G; Marktel Sarah^*

来源：British Journal of Haematology, 2009, 147(4): 554-560.

DOI：10.1111/j.1365-2141.2009.07871.x

• veno-occlusive disease
• defibrotide
• busulphan
• thalassemia
• paediatric

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.

• 出版日期2009-11