|Nydia Chien, MSN|
|Jude Jonassaint, RN|
18 United States sites
1 France site
1 United Kingdom 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 clinical results (a, b, c, d, or e) measured in the 13 months before randomization that indicates a high-risk patient:
Both a TRV 2.5-2.9 m/sec and NT-proBNP plasma level ≥ 160 pg/mL,
TRV ≥ 3.0 m/sec,
Both a mean pulmonary artery pressure (PAP) by right heart catheterization 20-24 mmHg and NT-proBNP plasma level ≥ 160 pg/mL,
Mean PAP by right heart catheterization ≥ 25 mmHg,
Chronic kidney disease (CKD) due to SCD with macroalbuminuria (albumin creatinine ratio (ACR) >300 mg/g) on 2 occasions, or proteinuria (protein creatinine ratio >30 mg/mmol) on 2 occasions, or estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2 calculated on 2 occasions. 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
More than 10 vaso-occlusive episodes in the past 12 months requiring admission to a hospital to receive treatment.
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 baseline