New Treatments and Therapies
- Crizanlizumab (Adakveo) Crizanlizumab is a newer medication approved for the treatment of sickle cell disease. It works by preventing the sickle-shaped red blood cells from sticking together and blocking blood flow, which reduces the frequency of pain crises.
- How it works: Crizanlizumab targets a molecule involved in the adhesion of sickled cells to the blood vessel walls, reducing the chance of painful blockages and crises.
- Benefits: Studies have shown that crizanlizumab can significantly reduce the number of pain crises in patients with sickle cell disease.
- Status: As of now, Crizanlizumab is not generally available on the NHS in the UK. NICE has not yet fully approved it for widespread use in SCD, although it is available through certain clinical trials or special arrangements in select hospitals.
- Cost: The cost of Crizanlizumab is approximately £75,000 to £100,000 per patient per year. The cost can vary depending on the dosage and individual circumstances.
- Voxelotor (Oxbryta) Voxelotor is a medication that works by increasing the oxygen-carrying capacity of sickle cells. It helps prevent the sickling of red blood cells by improving their ability to hold onto oxygen.
- How it works: Voxelotor binds to hemoglobin to prevent it from sickling, thereby improving the blood flow and oxygen delivery to tissues. This can reduce the frequency of pain episodes and other complications.
- Benefits: Voxelotor has been shown to increase hemoglobin levels and reduce symptoms associated with sickle cell disease.
- Status: Like Crizanlizumab, Voxelotor is also not routinely available on the NHS at this time. Its approval for use in the UK is under consideration, and it may be available through clinical trials or on a case-by-case basis.
- Cost: Voxelotor costs around £80,000 to £100,000 per year per patient.
For those with private health insurance, the coverage and access to such treatments would depend on the terms of the policy.