|Nydia Chien, MSN|
|Jude Jonassaint, RN|
1 United States site
> 18 Years
– Age 18 years or older
– Diagnosis of SCD: homozygous sickle cell disease, hemoglobin-SC, Sβ-thalassemia,
hemoglobin-SO or hemoglobin-SD.
– Patients not on a chronic exchange transfusion program for at least 2 months.
– If patients are on hydroxyurea, glutamin, or selectin inhibitors the doses must be
stable for at least 2 months prior to randomization.
– Any one of the following vasculopathy biomarker results (a, b, or c) that indicates a
high-risk patient patient measured in the 12 months before randomization:
1. Both a TRV 2.5-2.9 m/sec and NT-proBNP plasma level ≥ 160 pg/mL
2. TRV ≥ 3.0 m/sec
3. Chronic kidney disease (CKD) due to SCD with macroalbuminuria (>300 mg albumin
per gram creatinine) confirmed by 2 repeat measurements, or proteinuria (protein
creatinine ratio >30 mg/mmol) confirmed by 2 positive results, or estimated
glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 calculated on 2 occasions Biomarkers used for eligibility are based on local clinical site readings. TRV, NT-proBNP, albumin to creatinine ratio, protein creatinine ratio, or eGFR values must be measured in a steady state (defined as measured ≥ 14 days since an acute care pain event) within 12 months prior to randomization. - Written informed consent obtained from patient to participate in the trial.
– RBC alloimmunization resulting in inability of blood bank to obtain compatible
components for chronic exchange transfusions
– Previous history of hyper-hemolysis syndrome
– Previous history of severe transfusion reaction resulting in renal failure or due to
serious complications such as hypotension or respiratory distress
– Religious objection to receiving blood transfusion
– Diagnosis of ischemic stroke within the past 6 months
– Clinical evidence of liver failure or advanced cirrhosis or any co-existing medical
condition that in the Investigator’s judgement will substantially increase the risk
associated with the patient’s participation in the trial
– Women of childbearing potential who have a positive pregnancy test at Screening