Veno Occlusive Disease
Conditions
Interventions
Defibrotide(DF-01) 1)Intravenous infusion of DF 6.25 mg/kg/dose over 2 hours,4 doses per day (every 6 hours). 2)From 1 day before starting conditioning regimen until day 30 post stem cell transplant
Sponsors
Fukushima Medical University Hospital Atsushi Kikuta
LINK Phamaceuticals KK
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.younger than 50 years old (at informed consent). 2.Primary disease is one of the following: 1)malignant tumor not in remission 2)malignant tumor in remission. 3)osteopetrosis 4)non-malignant disease other than osteopetrosis 3.Patients with one or more following risk factors of VOD who undergo allogeneic stem cell transplantation with myeloablative conditioning regimen. -Second myeloablative transplant -Not in remission at transplant -Performance status (ECOG) of 2 or more. -Conditioning regimen including Bu/Mel or Bu/Cy. -Liver dysfunction before stem cell transplant. -Positive for anti-HCV antibody. -Administration of gemutuzumab ozogamicin within 100 days before stem cell transplant. -Osteopetrosis 4.Witten informed consent to participate in the study from the subject or legally acceptable representative before screening tests.
Exclusion criteria
Exclusion criteria: 1.Using medication that increases risk of hemorrhage. 2.Acute bleeding that is not controlled. 3.Unstable hemodynamic status that require more than one vasopressor or decreased mean atrial pressure (MAP). 4.Complicated with viral fulminant hepatitis 5.Past history of organ transplant other than hematopoietic cell transplant. 6.Complicated with Grade IV GVHD 7.Females with pregnancy, breastfeeding, possible pregnancy. Male who will not consent contraception 8.Judged as inappropriate for participating in the study by the principal or other investigator for other reasons.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of VOD until day 30 post stem cell transplant in patients who undergo prophylaxis with DF. Principal or other investigator should evaluate the development of VOD according to the revised Seattle criteria. VOD is defined as those who meet at least 2 of the following criteria by day 35 post stem cell transplant. -T-Bil>=2mg/dL -Hepatomegaly -Right hypochondriac pain -Ascites -Unexplained weight gain of>5% from baseline. | — |
Secondary
| Measure | Time frame |
|---|---|
| To compare the following outcomes both in DF prophylaxis group and control (no prophylaxis) group. 1)Incidence of VOD at day 30, 100 post stem cell transplant. 2)Incidence of VOD according to the Baltimore criteria at day 30, 100 post stem cell transplant. 3)Severity of VOD in patients who developed VOD. 4)Incidence of total, grade II-IV, and III-IV acute GVHD at day 100 post stem cell transplant. 5)Survival at day 100, 180 post stem cell transplant. 6)Survival at day 100, 180 post stem cell transplant in patients who developed VOD. 7)Incidence and severity of adverse events and drug-related adverse event. 8)Date of engraftment. 9)Remission status of the original disease at day 30, 100, and 180 after stem cell transplant in patients with malignancy. | — |
Countries
Japan
Contacts
Public ContactMiwa Izutsu
CTD Inc. -
Outcome results
None listed