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Table 3 Major FDA-approved therapies for the treatment of SCD

From: Advances in the diagnosis and treatment of sickle cell disease

Drug and FDA approval

FDA approval date and indications

Mechanism of action

Dosing

Common adverse effects

Hydroxyurea

1998:

Adults to reduce frequency of painful crises

2017:

Children ≥ 2 years to reduce frequency of painful crises and need for blood transfusions

↑ Fetal Hb via temporary arrest of hematopoiesis and stress erythropoiesis

↓ Inflammation through ↓ in WBC and platelets

↓ Adhesion molecule expression

↑ Nitric oxide production

Usual starting dose 20 mg/kg/day

Dose escalate to maximum tolerated dose (~ 30–35 mg/kg/day)

Alternatively, dose escalate to absolute neutrophil count of 1500–2000/µL

Neutropenia (13%)

Thrombocytopenia (7%)

Nausea (3%)

l-glutamine

2017:

Children and adults ≥ 5 years old to reduce severe complications (sickle cell crises and acute chest syndrome)

↑ NAD redox potential in sickle red blood cells

Protects red blood cells from oxidative stress

Dose by weight

 < 30 kg—1 packet (5 g) BID

30–65 kg—2 packets (10 g) BID

 > 65 kg—3 packets (15 g) BID

May take with or without hydroxyurea

Constipation (21%)

Nausea (19%)

Abdominal pain (17%)

Headache (18%)

Cough (16%)

Crizanlizumab

2019:

Adolescents and adults ≥ 16 years old to reduce frequency of vaso-occlusive crises

Binds to P-selectin

Blocks interactions with ligands, including P-selectin glycoprotein ligand 1

5 mg/kg/dose IV on weeks 0, 2 and every 4 weeks thereafter

May take with or without hydroxyurea

Infusion-related adverse events (< 10%)

Nausea (18%)

Arthralgia (18%)

Back pain (15%)

Fever (11%)

Voxelotor

2019:

Children and adults ≥ 12 years old to increase hemoglobin concentration

Allosteric modifier of hemoglobin to stabilize oxygenated state

↓ Sickle Hb polymerization

↓ Hemolysis

1500 mg po daily

May take with or without hydroxyurea

Headache (26%)

Diarrhea (20%)

Nausea (17%)

Abdominal pain (19%)

Skin rash (14%)

Fever (12%)

Fatigue (14%)