DEFIBROTIDE FOR THE PREVENTION AND TREATMENT OF HEPATIC VENO-OCCLUSIVE DISEASE AFTER HEMATOPOIETIC STEM CELL TRANSPLANTATION; A SINGLE CENTER EXPERIENCE
(Abstract release date: 05/19/16)
EHA Library. Antmen B. 06/09/16; 133068; E1519
Prof. Bulent Antmen
Contributions
Contributions
Abstract
Abstract: E1519
Type: Eposter Presentation
Background
Hepatic veno-occlusive disease (VOD) is a common and serious complication of hemotopoietic stem cell transplantation (HSCT) in children.
Aims
We aimed to assess prospectively the use of prophylactic defibrotide in pediatric patients undergoing HSCT.
Methods
In this study, 76 patients who underwent HSCT were given defibrotide prophylaxis as 25 mg/kg per day in four divided intravenous infusions over 2h, starting on the same day as the pretransplantation conditioning regimen. The mean duration of use of defibrotide is 20 days as a prophylaxis.
Results
In this study, 76 patients were recruited, 53 male patients and 23 female patients, with the average of 9.3 years, range 1-20; 4% infants, 55% children and 41% adolescent. There were 33 patients with thalassemia major, 30 patients with leukemia, 7 patients with aplastic anemia, one patient with Diamond Blackfan anemia, two patients with congenitale dyserythropoetic anemia, one patient with osteopetrosis, one patient with famial hemophagocytic lymphohistiocytosis, and one patient with Kostman syndrome. All transplants were allogeneic. No serious side effects were seen. In eight patients developed clinical VOD (Seattle criteria). In these patients, defibrotide dose was increased to a treatment dose of 40-60 mg/kg per day. One infant patient with Kostman syndrome and one patient with aplastic anemia died due to hepatic and pulmonary veno-occlusive disease. After 24 months of follow up, 6 patients who developed VOD are being well and no patient have transplant related complications.
Conclusion
Hepatic veno-occlusive disease, which is caused by hepatocyte and sinusoidal vessel endothelium damage, can ocur early after HSCT, and in its severe form, may lead tol iver faillure, hepatorenal syndrom, portal hypertension, and eventually death from multiorgan faillure. In this prospective study, we demonstrated that the use of defibrotide is safe and effective in preventing and treating VOD in pediatric patients at high risk.
Session topic: E-poster
Keyword(s): Bone marrow transplant, Childhood, Defibrotide, Veno-occlusive disease
Type: Eposter Presentation
Background
Hepatic veno-occlusive disease (VOD) is a common and serious complication of hemotopoietic stem cell transplantation (HSCT) in children.
Aims
We aimed to assess prospectively the use of prophylactic defibrotide in pediatric patients undergoing HSCT.
Methods
In this study, 76 patients who underwent HSCT were given defibrotide prophylaxis as 25 mg/kg per day in four divided intravenous infusions over 2h, starting on the same day as the pretransplantation conditioning regimen. The mean duration of use of defibrotide is 20 days as a prophylaxis.
Results
In this study, 76 patients were recruited, 53 male patients and 23 female patients, with the average of 9.3 years, range 1-20; 4% infants, 55% children and 41% adolescent. There were 33 patients with thalassemia major, 30 patients with leukemia, 7 patients with aplastic anemia, one patient with Diamond Blackfan anemia, two patients with congenitale dyserythropoetic anemia, one patient with osteopetrosis, one patient with famial hemophagocytic lymphohistiocytosis, and one patient with Kostman syndrome. All transplants were allogeneic. No serious side effects were seen. In eight patients developed clinical VOD (Seattle criteria). In these patients, defibrotide dose was increased to a treatment dose of 40-60 mg/kg per day. One infant patient with Kostman syndrome and one patient with aplastic anemia died due to hepatic and pulmonary veno-occlusive disease. After 24 months of follow up, 6 patients who developed VOD are being well and no patient have transplant related complications.
Conclusion
Hepatic veno-occlusive disease, which is caused by hepatocyte and sinusoidal vessel endothelium damage, can ocur early after HSCT, and in its severe form, may lead tol iver faillure, hepatorenal syndrom, portal hypertension, and eventually death from multiorgan faillure. In this prospective study, we demonstrated that the use of defibrotide is safe and effective in preventing and treating VOD in pediatric patients at high risk.
Session topic: E-poster
Keyword(s): Bone marrow transplant, Childhood, Defibrotide, Veno-occlusive disease
Abstract: E1519
Type: Eposter Presentation
Background
Hepatic veno-occlusive disease (VOD) is a common and serious complication of hemotopoietic stem cell transplantation (HSCT) in children.
Aims
We aimed to assess prospectively the use of prophylactic defibrotide in pediatric patients undergoing HSCT.
Methods
In this study, 76 patients who underwent HSCT were given defibrotide prophylaxis as 25 mg/kg per day in four divided intravenous infusions over 2h, starting on the same day as the pretransplantation conditioning regimen. The mean duration of use of defibrotide is 20 days as a prophylaxis.
Results
In this study, 76 patients were recruited, 53 male patients and 23 female patients, with the average of 9.3 years, range 1-20; 4% infants, 55% children and 41% adolescent. There were 33 patients with thalassemia major, 30 patients with leukemia, 7 patients with aplastic anemia, one patient with Diamond Blackfan anemia, two patients with congenitale dyserythropoetic anemia, one patient with osteopetrosis, one patient with famial hemophagocytic lymphohistiocytosis, and one patient with Kostman syndrome. All transplants were allogeneic. No serious side effects were seen. In eight patients developed clinical VOD (Seattle criteria). In these patients, defibrotide dose was increased to a treatment dose of 40-60 mg/kg per day. One infant patient with Kostman syndrome and one patient with aplastic anemia died due to hepatic and pulmonary veno-occlusive disease. After 24 months of follow up, 6 patients who developed VOD are being well and no patient have transplant related complications.
Conclusion
Hepatic veno-occlusive disease, which is caused by hepatocyte and sinusoidal vessel endothelium damage, can ocur early after HSCT, and in its severe form, may lead tol iver faillure, hepatorenal syndrom, portal hypertension, and eventually death from multiorgan faillure. In this prospective study, we demonstrated that the use of defibrotide is safe and effective in preventing and treating VOD in pediatric patients at high risk.
Session topic: E-poster
Keyword(s): Bone marrow transplant, Childhood, Defibrotide, Veno-occlusive disease
Type: Eposter Presentation
Background
Hepatic veno-occlusive disease (VOD) is a common and serious complication of hemotopoietic stem cell transplantation (HSCT) in children.
Aims
We aimed to assess prospectively the use of prophylactic defibrotide in pediatric patients undergoing HSCT.
Methods
In this study, 76 patients who underwent HSCT were given defibrotide prophylaxis as 25 mg/kg per day in four divided intravenous infusions over 2h, starting on the same day as the pretransplantation conditioning regimen. The mean duration of use of defibrotide is 20 days as a prophylaxis.
Results
In this study, 76 patients were recruited, 53 male patients and 23 female patients, with the average of 9.3 years, range 1-20; 4% infants, 55% children and 41% adolescent. There were 33 patients with thalassemia major, 30 patients with leukemia, 7 patients with aplastic anemia, one patient with Diamond Blackfan anemia, two patients with congenitale dyserythropoetic anemia, one patient with osteopetrosis, one patient with famial hemophagocytic lymphohistiocytosis, and one patient with Kostman syndrome. All transplants were allogeneic. No serious side effects were seen. In eight patients developed clinical VOD (Seattle criteria). In these patients, defibrotide dose was increased to a treatment dose of 40-60 mg/kg per day. One infant patient with Kostman syndrome and one patient with aplastic anemia died due to hepatic and pulmonary veno-occlusive disease. After 24 months of follow up, 6 patients who developed VOD are being well and no patient have transplant related complications.
Conclusion
Hepatic veno-occlusive disease, which is caused by hepatocyte and sinusoidal vessel endothelium damage, can ocur early after HSCT, and in its severe form, may lead tol iver faillure, hepatorenal syndrom, portal hypertension, and eventually death from multiorgan faillure. In this prospective study, we demonstrated that the use of defibrotide is safe and effective in preventing and treating VOD in pediatric patients at high risk.
Session topic: E-poster
Keyword(s): Bone marrow transplant, Childhood, Defibrotide, Veno-occlusive disease
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