Defibrotide in the treatment of children with veno-occlusive disease (VOD): a retrospective multicentre study demonstrates therapeutic efficacy upon early intervention.
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.
Corbacioglu
S
S
Universitätsklinik und Poliklinik für Kinder- und Jugendmedizin, Universität Ulm, Prittwitzstrasse 43, D-89075 Ulm Donau, Germany. selim.corbacioglu@medizin.uni-ulm.de
Greil
J
J
Peters
C
C
Wulffraat
N
N
Laws
H J
HJ
Dilloo
D
D
Straham
B
B
Gross-Wieltsch
U
U
Sykora
K W
KW
Ridolfi-Lüthy
A
A
Basu
O
O
Gruhn
B
B
Güngör
T
T
Mihatsch
W
W
Schulz
A S
AS
Strahm
B
B
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
D010975:Platelet Aggregation Inhibitors; D011089:Polydeoxyribonucleotides; C036901:defibrotide