|Erin Morris, RN|
|Mitchell S Cairo, MD|
4 United States sites
6 to 34 Years
– 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.
– Recurrent painful events (at least 3 in the 2 years prior to enrollment or prior to
chronic chronic RBC transfusion therapy, exchange transfusion or erythrocyte
– Abnormal TCD study requiring starting on chronic transfusion therapy and/or exchange
– At least one silent infarct lesion on a MRI scan of the head. Or (directly or probably
related to SCD)
– Sickle Cell nephropathy;
– Splenic sequestration requiring RBC transfusion;
– Aplastic crisis requiring RBC transfusion;
– Avascular necrosis of the hip diagnosed by MRI;
– Two episodes or more of leg ulcerations;
– Recurrent priapism .
– Infant dactylitis.
– OR for Cohort #2 ONLY: Patient must be between 18 and 34.99 years of age,
patients must demonstrate at least two of the following:
– WBC > 13,500 cells/microliter at baseline when not acutely ill (on two separate
occasions) > 2 weeks from a VOC event or hospitalization.
– Tricuspid Regurgitant Jet Velocity (TRV) > 3.0 m/s
– Requiring Chronic Monthly Transfusions ( > 12 transfusions in the 12 months)
– History of sepsis
– N-terminal pro-brain natriuretic peptide (NT-proBNP) > 160 ng/L at clinical baseline
when not acutely ill or hospitalized.
– all patients must meet disease, age, organ function and donor criteria;
– Patients who are receiving concomitant systemic anticoagulants and/or fibrinolytic
– 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
– SCD patients who have an unaffected HLA matched family donor willing to proceed to
donation will not be eligible for this study.
– Karnofsky or Lansky (age appropriate) Performance Score <50% (hemiplegia alone secondary to a previous stroke is not an exclusion) - Demonstrated lack of compliance with medical care. - Patients with clinically significant fibrosis or cirrhosis of the liver will not be eligible. - Patients who have previously received a HSCT will not be eligible. - Patients with contraindications to the use of defibrotide