Table 1. Selected agents that may be... | American Society of Hematology

Table 1.

Selected agents that may be beneficial in sickle cell disease.

Drug	Mechanism of action	Possible SCD-related indications	Ongoing studies	N
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

Drug	Mechanism of action	Possible SCD-related indications	Ongoing studies	N
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