Table 1.

Selected agents that may be beneficial in sickle cell disease.

DrugMechanism of actionPossible SCD-related indicationsOngoing studiesN
HbF indicates fetal hemoglobin; NO, nitric oxide; RBC, red blood cell; IVIG, intravenous immunoglobulin; N/A, none available 
*Randomized, controlled trials 
Butyrate Increase HbF level Acute pain episodes; leg ulcers; pulmonary hypertension NCT00004412* 30 
Decitabine Increase HbF level Acute pain episodes; symptomatic anemia N/A  
Trichostatin A Increase HbF level; decrease cell adhesion to vessel wall Acute pain episodes N/A  
Pomalidomide Increase HbF level Acute pain episodes N/A  
Senicapoc Improve RBC hydration Symptomatic anemia; pulmonary hypertension N/A  
Nitric oxide Increase NO Acute pain episodes; acute chest syndrome NCT00094887* 150 
   NCT00142051* 20 
   NCT00748423* 240 
Sildenafil Increase NO; increase HbF level Pulmonary hypertension; priapism NCT00492531 132 
6R-BH4 Increase NO; improve endothelial function Pulmonary hypertension NCT00445978 40 
Tinzaparin Decrease P-selectin-mediated cell adherence to vessel wall; decrease coagulation activation Acute pain episodes; thromboembolic disease N/A  
IVIG Decrease number of leukocytes adherent to endothelium; decrease number of RBC interacting with leukocytes Acute pain episodes NCT00644865* 52 
Dexamethasone Decrease inflammation Acute chest syndrome N/A  
Nix-0699 Uncertain, but inhibits RBC sickling; left shift of O2 dissociation curve Acute pain episodes N/A  
Eptifibatide Decrease platelet aggregation; Acute pain episodes NCT00834899* 30 
 Decrease CD40 ligand release    
Statins Improve endothelial function; decrease coagulation activation Pulmonary hypertension; thromboembolic disease NCT00508027 36 
DrugMechanism of actionPossible SCD-related indicationsOngoing studiesN
HbF indicates fetal hemoglobin; NO, nitric oxide; RBC, red blood cell; IVIG, intravenous immunoglobulin; N/A, none available 
*Randomized, controlled trials 
Butyrate Increase HbF level Acute pain episodes; leg ulcers; pulmonary hypertension NCT00004412* 30 
Decitabine Increase HbF level Acute pain episodes; symptomatic anemia N/A  
Trichostatin A Increase HbF level; decrease cell adhesion to vessel wall Acute pain episodes N/A  
Pomalidomide Increase HbF level Acute pain episodes N/A  
Senicapoc Improve RBC hydration Symptomatic anemia; pulmonary hypertension N/A  
Nitric oxide Increase NO Acute pain episodes; acute chest syndrome NCT00094887* 150 
   NCT00142051* 20 
   NCT00748423* 240 
Sildenafil Increase NO; increase HbF level Pulmonary hypertension; priapism NCT00492531 132 
6R-BH4 Increase NO; improve endothelial function Pulmonary hypertension NCT00445978 40 
Tinzaparin Decrease P-selectin-mediated cell adherence to vessel wall; decrease coagulation activation Acute pain episodes; thromboembolic disease N/A  
IVIG Decrease number of leukocytes adherent to endothelium; decrease number of RBC interacting with leukocytes Acute pain episodes NCT00644865* 52 
Dexamethasone Decrease inflammation Acute chest syndrome N/A  
Nix-0699 Uncertain, but inhibits RBC sickling; left shift of O2 dissociation curve Acute pain episodes N/A  
Eptifibatide Decrease platelet aggregation; Acute pain episodes NCT00834899* 30 
 Decrease CD40 ligand release    
Statins Improve endothelial function; decrease coagulation activation Pulmonary hypertension; thromboembolic disease NCT00508027 36 
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