Zeitschrift für Knochenforschung
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Defibrotide for Prevention and Treatment of Veno-Occlusive Disease in Adults: Single-Center Experience

Yonal Ipek, Kırkızlar Onur Hakkı, Kalayoglu-Besisik Sevgi and Sargin Fatma Deniz

Background: Hepatic veno-occlusive disease (VOD) has been reported at a rate up to 50% following intensive conditioning regimens used in allogeneic hematopoietic stem cell transplantation (AHSCT). Studies on the prophylactic effect of defibrotide to prevent hepatic VOD in adults are rare.
Purpose: The research presented here aimed to evaluate whether propylactic defibrotide use can reduce incidence and severity of VOD in adults undergoing AHSCT. Also, we aimed to assess the benefit of defibrotide for treatment of VOD.
Methods: Study population comprised 86 consecutive AHSCT patients transplanted between January 2005 and December 2009. 17 of the patients at high risk of developing VOD could have access to defibrotide and received defibrotide prophylaxis.
Results: Modified Seattle criteria were used for VOD diagnosis. 14 of 86 patients (10 severe, 3 moderate, 1 mild) were diagnosed with VOD (16.2%). VOD incidence was similar between patients transplanted before December 2004 and after January 2005 (9.3% and 16.2%, respectively; p =0.14, HR =1.88, 95% CI 0.82- 4.29). 13 of 14 patients diagnosed with VOD in the study population were treated with defibrotide whereas only 2 of 12 in the control group received defibrotide for treatment (92.8% and 16.6%, respectively; p =0.0002, HR =65 , 95% CI 5.13-823.1). Mortality rate of VOD in the controls was significantly higher than the study population (66.6% and 21.4%, respectively; p =0.044, HR =0.13, 95% CI 0.02-0.78).
Conclusions: Mortality rate related to VOD was lower in the defibrotide group. Therefore, we conclude defibrotide might be beneficial for treatment of VOD in adults.