From the Guidelines
Sickle cell patients should take hydroxyurea as the primary chronic medication, typically starting at 15-20 mg/kg/day with potential increases to the maximum tolerated dose, as it has been shown to increase fetal hemoglobin production, reduce sickling, and decrease vaso-occlusive complications 1.
Medication Overview
The goal of chronic medication in sickle cell patients is to reduce morbidity, mortality, and improve quality of life.
- Hydroxyurea is the most well-established medication for this purpose, with a long history of use and a strong evidence base supporting its efficacy 1.
- Folic acid supplementation at 1 mg daily is also recommended to support red blood cell production.
- Other medications, such as L-glutamine (Endari), crizanlizumab (Adakveo), and voxelotor (Oxbryta), may be beneficial for specific patients, but the evidence for these medications is not as strong as it is for hydroxyurea 1.
Key Considerations
- Regular monitoring of blood counts is essential while on these medications, and treatment should be individualized based on disease severity, complications, and patient response.
- The choice of medication and dosage should be guided by the most recent and highest-quality evidence, with a focus on minimizing morbidity, mortality, and improving quality of life.
- Patients with specific complications, such as delayed hemolytic transfusion reaction and ongoing hyperhemolysis, may benefit from immunosuppressive therapy, such as IVIg, steroids, rituximab, and/or eculizumab 1.
From the FDA Drug Label
The efficacy of ADAKVEO was evaluated in patients with sickle cell disease in SUSTAIN [NCT01895361], a 52-week, randomized, multicenter, placebo-controlled, double-blind study Patients received ADAKVEO (with or without hydroxyurea) and were allowed to receive occasional transfusions and pain medications [i.e., acetaminophen, NSAIDs, and opioids] on an as needed basis.
Medications for Sickle Cell Patients:
- Hydroxyurea: may be used in combination with ADAKVEO
- Acetaminophen: for pain management
- NSAIDs: for pain management
- Opioids: for pain management
- ADAKVEO (crizanlizumab): for reducing the frequency of vaso-occlusive crises 2
From the Research
Medications for Sickle Cell Patients
Sickle cell patients may be prescribed several medications to manage their condition. These include:
- Hydroxyurea: a disease-modifying therapy that increases fetal hemoglobin and reduces red blood cell sickling 3, 4, 5
- L-glutamine: an adjunctive therapy that reduces hospitalization rates and mean length of stay 3, 6, 7
- Crizanlizumab: a P-selectin inhibitor that reduces sickle cell-endothelial interactions and vaso-occlusive events 3, 4, 6, 7
- Voxelotor: a hemoglobin-oxygen affinity-shifting agent that decreases sickle hemoglobin polymerization 3, 4, 6, 7
Treatment Approach
The goal of pharmacotherapy for sickle cell disease is to reduce morbidity and mortality by diminishing both hemolytic anemia and vaso-occlusive events 4. This can be achieved by:
- Starting patients on maximal doses of hydroxyurea 4
- Adding voxelotor or crizanlizumab to hydroxyurea therapy if the clinical and hematologic response is insufficient 4
- Considering combination therapy with all three medications if the response to monotherapy or dual-drug therapy is poor 4
Benefits and Barriers
These medications have been shown to have several benefits, including:
- Reduced pain crises and hospitalizations 3, 6, 7
- Increased hemoglobin and decreased markers of hemolysis 3, 7
- Reduced need for opioid use 7 However, there are also barriers to access, including:
- Insurance denial and high deductibles 7
- Intolerability and side effects, such as abdominal pain, headache, and nausea 7