This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical or MUD allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS. The Funding Source is FDA OOPD.
Disease: Homozygous Hemoglobin S Disease, or Hemoglobin S B0/+ thalassemia, or Hemoglobin SC Disease, or Beta thalassemia intermedia/majora
Patients must demonstrate one or more of the following Sickle Cell Disease Complications
Clinically significant neurologic event (stroke) or any neurologic deficit lasting >24 hours that is accompanied by an infarct on cerebral MRI
Acute chest syndrome in the preceding two year period prior to enrollment that have failed, been non-compliant or declined hydroxyurea treatment, or prior to chronic RBC transfusion therapy, exchange transfusion or erythrocyte pheresis.
Patients who are receiving concomitant systemic anticoagulants and/or fibrinolytic therapies.
Patients with a previously known hypersensitivity reaction to defibrotide.
Females who are pregnant or breast-feeding are not eligible
Patients with documented uncontrolled infection at the time of study entry are not eligible.
Single group
Defibrotide
Drugdefibrotide will be given prophylactically prior to AlloSCT to determine if it decreases the incidence of SOS in this high risk population, and determine that it is safe and feasible to give along with myeloimmunoablative therapy and allogeneic transplant.
All patients will be monitored for known and unknown side effects of defibrotide with daily physical exams while in the hospital and then as needed in addition to daily laboratory values including chemistries, hematology labs as needed
Patients will be given Defibrotide prophylaxis starting 10 days before the stem cell infusion at 6.25 mg/kg IV q6h and continue through Day +21.
All patients will be monitored for the development of SOS.
All patients will get daily lab values while in patients and then as needed to monitor for elevation in liver function tests and other abnormal chemistry or hematology values. Imaging on the liver will be performed as needed to determine if they develop SOS with defibrotide.
Click on the lead sponsor to view all of their trials.
New York Medical College
Lead sponsor
University of California, Los Angeles
Collaborator
Medical College of Wisconsin
Collaborator
Johns Hopkins University
Collaborator
Dana-Farber Cancer Institute
Collaborator
Children's Hospital Los Angeles
Collaborator
Children's Hospital Medical Center, Cincinnati
Collaborator
University of Florida
Collaborator
