Corbacioglu , S , Carreras , E , Mohty , M , Pagliuca , A , Boelens , J J , Damaj , G , Iacobelli , M , Niederwieser , D , Olavarria , E , Suarez , F , Ruutu , T , Verdonck , L , Hume , R , Nejadnik , B , Lai , C , Finetto , G & Richardson , P 2016 , ' Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease : Final Results From the International Compassionate Use Program ' , Biology of Blood and Marrow Transplantation , vol. 22 , no. 10 , pp. 1874-1882 . https://doi.org/10.1016/j.bbmt.2016.07.001
Title: | Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease : Final Results From the International Compassionate Use Program |
Author: | Corbacioglu, Selim; Carreras, Enric; Mohty, Mohamad; Pagliuca, Antonio; Boelens, Jaap Jan; Damaj, Gandhi; Iacobelli, Massimo; Niederwieser, Dietger; Olavarria, Eduardo; Suarez, Felipe; Ruutu, Tapani; Verdonck, Leo; Hume, Robin; Nejadnik, Bijan; Lai, Chinglin; Finetto, Giorgia; Richardson, Paul |
Contributor organization: | Clinicum Department of Oncology |
Date: | 2016-10 |
Language: | eng |
Number of pages: | 9 |
Belongs to series: | Biology of Blood and Marrow Transplantation |
ISSN: | 1083-8791 |
DOI: | https://doi.org/10.1016/j.bbmt.2016.07.001 |
URI: | http://hdl.handle.net/10138/168552 |
Abstract: | Hepatic veno-occlusive disease, also called sinusoidal obstruction syndrome (VOD/SOS), is an unpredictable and potentially fatal complication of hematopoietic cell transplantation (HCT) or nontransplantation-associated chemotherapy/radiotherapy. In cases of severe hepatic VOD/SOS, typically defined by associated multiorgan failure (MOF, also known as multiorgan dysfunction), mortality exceeds 80%. Preclinical and early clinical data have provided a rationale for defibrotide treatment in hepatic VOD/SOS. Based on this evidence and in recognition of the dismal prognosis for these patients, defibrotide was made available through an international multicenter compassionate-use program conducted from December 1998 to March 2009. Physicians participating in the program voluntarily provided demographic and outcome data for patients given defibrotide. Efficacy and safety analyses were performed using the data received for 710 treated patients. Defibrotide was given at 10, 25, 40, 60, or 80 mg/kg/day for a median of 15 days (range, 1 to 119 days). By Kaplan-Meier analysis, the estimated overall day +100 survival was 54% (58% in the 25 mg/kg/day dose group). Adverse events (AEs) were reported in 53% of patients. The most common AEs were MOF, progression of hepatic VOD/SOS, sepsis, and graft-versus-host disease, which were consistent with the AEs expected for this patient population. No clinically meaningful trends in AEs were identified by gender, age, or dose group. Safety and efficacy results were consistent with prior studies of defibrotide in hepatic VOD/SOS, and subgroup analyses lend support to the use of the 25 mg/kg/day dose. (C) 2016 American Society for Blood and Marrow Transplantation. |
Subject: |
Defibrotide
Compassionate-use program Stem cell transplantation Hepatic veno-occlusive disease Sinusoidal obstruction syndrome STEM-CELL TRANSPLANTATION BONE-MARROW-TRANSPLANTATION HIGH-RISK POPULATION SIGNIFICANT TOXICITY MULTIORGAN FAILURE ENDOTHELIAL-CELLS LIVER THROMBOMODULIN COMPLICATIONS PHASE-3 3122 Cancers 3121 General medicine, internal medicine and other clinical medicine |
Peer reviewed: | Yes |
Rights: | cc_by |
Usage restriction: | openAccess |
Self-archived version: | publishedVersion |
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