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Veno-Occlusive Disease of Liver

Defibrotide in the treatment of children with veno-occlusive disease (VOD): a retrospective multicentre study demonstrates therapeutic efficacy upon early intervention

Bone Marrow Transplantation volume 33, pages 189–195 (2004)Cite this article

A Corrigendum to this article was published on 08 March 2004

Summary:

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.

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  1. Universitätsklinik und Poliklinik für Kinder- und Jugendmedizin, Universität Ulm, Germany

    S Corbacioglu, J Greil, C Peters, N Wulffraat, H J Laws, D Dilloo, B Strahm, U Gross-Wieltsch, K W Sykora, A Ridolfi-Lüthy, O Basu, B Gruhn, T Güngör, W Mihatsch & A S Schulz

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Corbacioglu, S., Greil, J., Peters, C. et al. Defibrotide in the treatment of children with veno-occlusive disease (VOD): a retrospective multicentre study demonstrates therapeutic efficacy upon early intervention. Bone Marrow Transplant 33, 189–195 (2004). https://doi.org/10.1038/sj.bmt.1704329

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