title: Defibrotide in the treatment of children with veno-occlusive disease (VOD): a retrospective multicentre study demonstrates therapeutic efficacy upon early intervention.
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.
authors.affiliation: Universitätsklinik und Poliklinik für Kinder- und Jugendmedizin, Universität Ulm, Prittwitzstrasse 43, D-89075 Ulm Donau, Germany. selim.corbacioglu@medizin.uni-ulm.de
authors.forename: S
authors.initials: S
authors.lastname: Corbacioglu
authors.forename: J
authors.initials: J
authors.lastname: Greil
authors.forename: C
authors.initials: C
authors.lastname: Peters
authors.forename: N
authors.initials: N
authors.lastname: Wulffraat
authors.forename: H J
authors.initials: HJ
authors.lastname: Laws
authors.forename: D
authors.initials: D
authors.lastname: Dilloo
authors.forename: B
authors.initials: B
authors.lastname: Straham
authors.forename: U
authors.initials: U
authors.lastname: Gross-Wieltsch
authors.forename: K W
authors.initials: KW
authors.lastname: Sykora
authors.forename: A
authors.initials: A
authors.lastname: Ridolfi-Lüthy
authors.forename: O
authors.initials: O
authors.lastname: Basu
authors.forename: B
authors.initials: B
authors.lastname: Gruhn
authors.forename: T
authors.initials: T
authors.lastname: Güngör
authors.forename: W
authors.initials: W
authors.lastname: Mihatsch
authors.forename: A S
authors.initials: AS
authors.lastname: Schulz
authors.forename: B
authors.initials: B
authors.lastname: Strahm
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006504:Hepatic Veno-Occlusive Disease / Q000188:drug therapy* / Q000453:epidemiology; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D008297:Male; D010975:Platelet Aggregation Inhibitors / Q000008:administration & dosage* / Q000009:adverse effects; D011089:Polydeoxyribonucleotides / Q000008:administration & dosage* / Q000009:adverse effects; D012189:Retrospective Studies; D012307:Risk Factors; D012720:Severity of Illness Index; D019172:Transplantation Conditioning; D016896:Treatment Outcome
chemical_list: D010975:Platelet Aggregation Inhibitors; D011089:Polydeoxyribonucleotides; C036901:defibrotide