Abstract
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.
Highlights
Hepatic veno-occlusive disease (VOD) - known as sinusoidal obstruction syndrome- is considered one of the leading causes of morbidity and mortality after hematopoietic stem cell transplantation (HSCT) [1]
VOD incidence was similar between patients transplanted before December 2004 and after January 2005 (9.3% and 16.2%, respectively; p =0.14, hazard ratio (HR) =1.88, 95% confidence limits (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)
Summary
Hepatic veno-occlusive disease (VOD) - known as sinusoidal obstruction syndrome- is considered one of the leading causes of morbidity and mortality after hematopoietic stem cell transplantation (HSCT) [1]. Defibrotide is a mixture of porcine oligodeoxyribonucleotides that has antithrombotic, anti-ischemic and anti-inflammatory properties This agent seems to have a protective effect against endothelial cell injury by increasing fibrinolysis and reducing procoagulant activity yet without an increase in bleeding events [3,4,5]. Defibrotide use was first reported in patients diagnosed with severe VOD following stem cell transplantation performed between 1995 and 1997 [8]. To the best of our knowledge, only few studies have investigated the role of defibrotide for prophylaxis in adult patients following allogeneic hematopoietic stem cell transplantation (AHSCT) [18,19]. 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). We aimed to assess the benefit of defibrotide for treatment of VOD
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