Defibrotide for the treatment of hepatic veno-occlusive disease: results of the European compassionate-use study.

2.50
Hdl Handle:
http://hdl.handle.net/10541/86394
Title:
Defibrotide for the treatment of hepatic veno-occlusive disease: results of the European compassionate-use study.
Authors:
Chopra, Rajesh; Eaton, J D; Grassi, A; Potter, M; Shaw, B; Salat, C; Neumeister, P; Finazzi, G; Iacobelli, M; Bowyer, K; Prentice, H G; Barbui, T
Abstract:
Severe hepatic veno-occlusive disease (VOD) is a recognized complication of autologous and allogeneic stem cell transplantation (SCT) that is often fatal. Defibrotide (DF) is a polydeoxyribonucleotide that has been found to have anti-thrombotic, anti-ischaemic and thrombolytic properties without causing significant anticoagulation. Preliminary studies have demonstrated activity for DF in the treatment of VOD, with minimal associated toxicity. In the present study, 40 patients who fulfilled established criteria for VOD were treated with DF on compassionate grounds in 19 European centres; 28 patients met risk criteria predicting progression of VOD and fatality or had evidence of multiorgan failure (MOF), and were defined as 'poor-risk'. DF was commenced intravenously at a median of 14 d (range, -2 d to 53 d) post SCT at doses ranging from 10 to 40 mg/kg. The median duration of therapy was 18 d (range, 2--71 d). Twenty-two patients showed a complete response (CR) (bilirubin < 34.2 micromol/l and resolution of signs/symptoms of VOD and end-organ dysfunction) [CR = 55%, confidence interval (CI) 40--70%] and 17 patients (43%) are alive beyond d +100. Ten poor-risk patients showed a complete response (CR = 36%, CI 21--51%). These results demonstrate that DF is an active treatment for VOD following SCT and a randomized trial is now underway in order to further evaluate its role.
Affiliation:
Department of Medical Oncology, Christie Hospital, Wilmslow Road, Manchester, UK. rchopra@picr.man.ac.uk
Citation:
Defibrotide for the treatment of hepatic veno-occlusive disease: results of the European compassionate-use study. 2000, 111 (4):1122-9 Br. J. Haematol.
Journal:
British Journal of Haematology
Issue Date:
Dec-2000
URI:
http://hdl.handle.net/10541/86394
DOI:
10.1111/j.1365-2141.2000.02475.x
PubMed ID:
11167751
Type:
Article
Language:
en
ISSN:
0007-1048
Appears in Collections:
All Christie Publications

Full metadata record

DC FieldValue Language
dc.contributor.authorChopra, Rajeshen
dc.contributor.authorEaton, J Den
dc.contributor.authorGrassi, Aen
dc.contributor.authorPotter, Men
dc.contributor.authorShaw, Ben
dc.contributor.authorSalat, Cen
dc.contributor.authorNeumeister, Pen
dc.contributor.authorFinazzi, Gen
dc.contributor.authorIacobelli, Men
dc.contributor.authorBowyer, Ken
dc.contributor.authorPrentice, H Gen
dc.contributor.authorBarbui, Ten
dc.date.accessioned2009-11-18T11:59:14Z-
dc.date.available2009-11-18T11:59:14Z-
dc.date.issued2000-12-
dc.identifier.citationDefibrotide for the treatment of hepatic veno-occlusive disease: results of the European compassionate-use study. 2000, 111 (4):1122-9 Br. J. Haematol.en
dc.identifier.issn0007-1048-
dc.identifier.pmid11167751-
dc.identifier.doi10.1111/j.1365-2141.2000.02475.x-
dc.identifier.urihttp://hdl.handle.net/10541/86394-
dc.description.abstractSevere hepatic veno-occlusive disease (VOD) is a recognized complication of autologous and allogeneic stem cell transplantation (SCT) that is often fatal. Defibrotide (DF) is a polydeoxyribonucleotide that has been found to have anti-thrombotic, anti-ischaemic and thrombolytic properties without causing significant anticoagulation. Preliminary studies have demonstrated activity for DF in the treatment of VOD, with minimal associated toxicity. In the present study, 40 patients who fulfilled established criteria for VOD were treated with DF on compassionate grounds in 19 European centres; 28 patients met risk criteria predicting progression of VOD and fatality or had evidence of multiorgan failure (MOF), and were defined as 'poor-risk'. DF was commenced intravenously at a median of 14 d (range, -2 d to 53 d) post SCT at doses ranging from 10 to 40 mg/kg. The median duration of therapy was 18 d (range, 2--71 d). Twenty-two patients showed a complete response (CR) (bilirubin < 34.2 micromol/l and resolution of signs/symptoms of VOD and end-organ dysfunction) [CR = 55%, confidence interval (CI) 40--70%] and 17 patients (43%) are alive beyond d +100. Ten poor-risk patients showed a complete response (CR = 36%, CI 21--51%). These results demonstrate that DF is an active treatment for VOD following SCT and a randomized trial is now underway in order to further evaluate its role.en
dc.language.isoenen
dc.subjectBreast Canceren
dc.subjectMyeloid Leukaemiaen
dc.subjectHaematopoietic Stem Cell Transplantationen
dc.subjectPrecursor Cell Lymphoblastic Leukaemia-Lymphomaen
dc.subject.meshAcute Disease-
dc.subject.meshAdolescent-
dc.subject.meshAdult-
dc.subject.meshBilirubin-
dc.subject.meshBreast Neoplasms-
dc.subject.meshChild-
dc.subject.meshChild, Preschool-
dc.subject.meshFemale-
dc.subject.meshFibrinolytic Agents-
dc.subject.meshHematopoietic Stem Cell Transplantation-
dc.subject.meshHepatic Veno-Occlusive Disease-
dc.subject.meshHodgkin Disease-
dc.subject.meshHumans-
dc.subject.meshInfant-
dc.subject.meshLeukemia, Myeloid-
dc.subject.meshMale-
dc.subject.meshMiddle Aged-
dc.subject.meshMultiple Myeloma-
dc.subject.meshPolydeoxyribonucleotides-
dc.subject.meshPostoperative Complications-
dc.subject.meshPrecursor Cell Lymphoblastic Leukemia-Lymphoma-
dc.subject.meshTreatment Outcome-
dc.titleDefibrotide for the treatment of hepatic veno-occlusive disease: results of the European compassionate-use study.en
dc.typeArticleen
dc.contributor.departmentDepartment of Medical Oncology, Christie Hospital, Wilmslow Road, Manchester, UK. rchopra@picr.man.ac.uken
dc.identifier.journalBritish Journal of Haematologyen

Related articles on PubMed

All Items in Christie are protected by copyright, with all rights reserved, unless otherwise indicated.