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 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 (or patients in Cohort 2 can meet other high risk criteria instead)
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
SCD Patients with documented uncontrolled infection at the time of study entry are not eligible.
New York Medical College
Lead sponsor
University of California, Los Angeles
Collaborator
Medical College of Wisconsin
Collaborator
Tufts Medical Center
Collaborator
Baylor College of Medicine
Collaborator
Johns Hopkins University
Collaborator
Dana-Farber Cancer Institute
Collaborator
Children's Hospital Los Angeles
Collaborator