TY - JOUR
T1 - Absence of VOD in paediatric thalassaemic HSCT recipients using defibrotide prophylaxis and intravenous Busulphan
AU - Cappelli, Barbara
AU - Chiesa, Robert
AU - Evangelio, Costanza
AU - Biffi, Alessandra
AU - Roccia, Tito
AU - Frugnoli, Ilaria
AU - Biral, Erika
AU - Noè, Anna
AU - Fossati, Marco
AU - Finizio, Valentina
AU - Miniero, Roberto
AU - Napolitano, Sara
AU - Ferrua, Francesca
AU - Soliman, Clara
AU - Ciceri, Fabio
AU - Roncarolo, Maria G.
AU - Marktel, Sarah
PY - 2009/11
Y1 - 2009/11
N2 - 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.
AB - 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.
KW - Busulphan
KW - Defibrotide
KW - Paediatric
KW - Thalassemia
KW - Veno-occlusive disease
UR - http://www.scopus.com/inward/record.url?scp=70350433279&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=70350433279&partnerID=8YFLogxK
U2 - 10.1111/j.1365-2141.2009.07871.x
DO - 10.1111/j.1365-2141.2009.07871.x
M3 - Article
C2 - 19747363
AN - SCOPUS:70350433279
VL - 147
SP - 554
EP - 560
JO - British Journal of Haematology
JF - British Journal of Haematology
SN - 0007-1048
IS - 4
ER -