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

Barbara Cappelli, Robert Chiesa, Costanza Evangelio, Alessandra Biffi, Tito Roccia, Ilaria Frugnoli, Erika Biral, Anna Noè, Marco Fossati, Valentina Finizio, Roberto Miniero, Sara Napolitano, Francesca Ferrua, Clara Soliman, Fabio Ciceri, Maria G. Roncarolo, Sarah Marktel

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)554-560
Number of pages7
JournalBritish Journal of Haematology
Issue number4
Publication statusPublished - Nov 2009


  • Busulphan
  • Defibrotide
  • Paediatric
  • Thalassemia
  • Veno-occlusive disease

ASJC Scopus subject areas

  • Hematology


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