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(Pediatric) Defibrotide for patients with hepatic veno-occlusive disease (VOD): A treatment IND study (Under CFR 312.34)
|Blood & Marrow Transplant (BMT)
|Hepatic Veno-Occlusive Disease (VOD)
- To provide access to Defibrotide as an investigational new drug under an expanded access treatment protocol
- VOD Diagnosis
- A diagnosis of VOD may be made by Baltimore Criteria, Modified Seattle Criteria, or biopsy proven:
- Baltimore Criteria: Bilirubin ≥ 2 mg/dL and at least 2 of the following
- ascites (radiographic or physical exam).
- weight gain > 5% above baseline weight (defined as: for patient who underwent HSCT, weight on the first day of conditioning-- if this value is not available, the weight on the date of admission to the SCT unit may be used; for patient who underwent chemotherapy, weight before the start of last chemo cycle that induced the VOD),
- hepatomegaly increased over baseline.
- Modified Seattle Criteria (McDonald et al Hepatology, 4(1): 116-22, 1984, Shulman et al Bone Marrow Transplantation 1992;10(3):197-214; Carreras et al Ann Hematol 66:77-80, 1993c): At least two of the following
- Patients who do not meet the Baltimore Criteria (126.96.36.199.1) or Modified Seattle Criteria (188.8.131.52.2) and have biopsy proven VOD are eligible.
- Patients must also provide voluntary written informed consent to the protocol to be eligible for the study. For minor patients, parent/legal guardian will provide consent and assent from adolescents will also be obtained. For compromised patients, their designated proxy must provide informed consent.
- Use of any medication which increases the risk of hemorrhage is disallowed. Use of heparin or other anticoagulants is disallowed within 12 hours of Defibrotide therapy initiation unless being used for routine central venous line management, fibrinolytic instillation for central venous line occlusion, intermittent dialysis or ultrafiltration of CVVH.
- Clinically significant uncontrolled acute bleeding, defined as hemorrhage requiring > 15 cc/kg of packed red blood cells/24 hours (e.g., a pediatric patient weighing 20 kg and requiring > 300cc of packed red blood cells/24 hours, or an adult patient weighing 70 kg and requiring >3 units of packed red blood cells/24 hours) to replace blood loss, OR bleeding from a site which in the Investigator's opinion constitutes a potential life-threatening source (e.g. pulmonary hemorrhage or CNS bleeding), irrespective of amount of blood loss, at any point from the date of SCT through the date of VOD diagnosis.
Please note: Transfusion of these amounts in the absence of clinically significant uncontrolled acute bleeding (i.e., for purposes of dialysis or because of hemodilution) is not a contraindication to enrollment.
- Hemodynamic instability as defined by a requirement for 2 or more pressors (not including renal-doses of dopamine), or inability to maintain stable mean arterial pressure (for adults: within 70-105 mmHg; for children: within the 90th percentiles of age-adjusted levels, see Appendix C) with single pressor support.
Please note: 1. Patients on a single pressor must have stable mean arterial pressure for at least 8 hours. 2. Patients requiring renal dose dopamine alone (2-5 mcg/kg/min) are eligible without measurement of mean arterial pressure.
- Woman who are pregnant.
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