Corbacioglu, Selim and Carreras, Enric and Mohty, Mohamad and Pagliuca, Antonio and Boelens, Jaap Jan and Damaj, Gandhi and Iacobelli, Massimo and Niederwieser, Dietger and Olavarria, Eduardo and Suarez, Felipe and Ruutu, Tapani and Verdonck, Leo and Hume, Robin and Nejadnik, Bijan and Lai, Chinglin and Finetto, Giorgia and Richardson, Paul (2016) Defibrotide for the Treatment of Hepatic Veno-Occlusive Disease: Final Results From the International Compassionate Use Program. BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION, 22 (10). pp. 1874-1882. ISSN 1083-8791, 1523-6536
Full text not available from this repository. (Request a copy)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.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | STEM-CELL TRANSPLANTATION; BONE-MARROW-TRANSPLANTATION; HIGH-RISK POPULATION; SIGNIFICANT TOXICITY; MULTIORGAN FAILURE; ENDOTHELIAL-CELLS; LIVER; THROMBOMODULIN; COMPLICATIONS; PHASE-3; Defibrotide; Compassionate-use program; Stem cell transplantation; Hepatic veno-occlusive disease; Sinusoidal obstruction syndrome |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 26 Apr 2019 12:25 |
| Last Modified: | 26 Apr 2019 12:25 |
| URI: | https://pred.uni-regensburg.de/id/eprint/4225 |
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