Defibrotide in the treatment of children with veno-occlusive disease (VOD): a retrospective multicentre study demonstrates therapeutic efficacy upon early intervention
- 8 December 2003
- journal article
- research article
- Published by Springer Science and Business Media LLC in Bone Marrow Transplantation
- Vol. 33 (2), 189-195
- https://doi.org/10.1038/sj.bmt.1704329
Abstract
Veno-occlusive disease (VOD) of the liver is a complication observed particularly in patients undergoing hematopoietic stem cell transplantation (HSCT). Defibrotide (DF) is a polydeoxyribonucleotide with aptameric activity on endothelium. We evaluated in a retrospective analysis the efficacy of DF in pediatric patients developing hepatic VOD after HSCT. A total of 45 patients between 0.2 and 20 years (median age: 8.2 years) with hepatic VOD were treated with DF: 22 patients (49%) met risk criteria for severe or progressive disease and 23 (51%) for moderately severe and mild disease. The median duration of DF treatment was 17 days. In all, 34 patients (76%) achieved complete response (CR) with a survival rate of 64% at day 100. CR rate in patients with severe disease was 50% with long-term survival of 36%. The average DF dose in the CR group was 45 mg/kg/day and in the no responder (NR) group 27 mg/kg/day. The use of additional drugs besides DF to treat VOD made no difference in the outcome compared to DF alone. The average interval from diagnosis to start of DF was 1 day in the CR and 5.5 days in NR group. In multivariate analysis, early intervention remained the only significant factor for a CR.Keywords
This publication has 35 references indexed in Scilit:
- Comparison of chronic graft-versus-host disease after transplantation of peripheral blood stem cells versus bone marrow in allogeneic recipients: long-term follow-up of a randomized trialBlood, 2002
- Hepatic sinusoidal obstruction after gemtuzumab ozogamicin (Mylotarg) therapyBlood, 2002
- Veno-occlusive disease of the liver in children with solid tumors undergoing autologous hematopoietic progenitor cell transplantation: a high incidence in patients with neuroblastomaBone Marrow Transplantation, 2002
- Development of hepatic veno-occlusive disease after Mylotarg infusion for relapsed acute myeloid leukemiaBone Marrow Transplantation, 2001
- Hepatitis C virus infection and bone marrow transplantation: A cohort study with 10-year follow-upHepatology, 1999
- Veno-occlusive disease of the liver after allogeneic bone marrow transplantation in children with hematologic malignancies: incidence, onset time and risk factorsBone Marrow Transplantation, 1998
- Busulfan disposition and hepatic veno-occlusive disease in children undergoing bone marrow transplantationCancer Chemotherapy and Pharmacology, 1995
- Veno-occlusive Disease of the Liver and Multiorgan Failure after Bone Marrow Transplantation: A Cohort Study of 355 PatientsAnnals of Internal Medicine, 1993
- Second allogeneic marrow transplantation for patients with recurrent leukemia after initial transplant with total-body irradiation-containing regimens.Journal of Clinical Oncology, 1993
- Bone Marrow Transplantation in Patients with ThalassemiaNew England Journal of Medicine, 1990