Bone marrow transplantation and stem cell transplantation are currently the only available cures for sickle cell disease (SCD). However, these procedures are risky, and to be successful they require a close match between the donor and recipient. Finding a suitable donor, even among relatives, can be challenging, and as a result, a bone marrow transplant is typically considered and offered only to children with severe SCD.[1]
Common medications prescribed for SCD include:[2]
- Antibiotics (e.g., penicillin), which are given daily to prevent infections, especially in young children. The treatment is usually continued up until the age of 5, but sometimes it may be prescribed for longer.
- Hydroxyurea reduces the likelihood of complications from severe cases of SCD. However, it is associated with a high risk of side effects and is therefore not suitable for milder cases.
- Voxelotor is an oral medication which reduces the likelihood of sickling of red blood cells. It is approved for patients 4 years or older.
- Crizanlizumab-tmc injections can be helpful in patients 16 years or older to reduce episodes of pain. Safety for younger patients has not been determined.
- L-glutamine powder and painkillers are used to reduce pain associated with sickle cell crises.[3]
Additionally, in severe cases of anemia, or in patients who have had a stroke or are at risk of developing one, blood transfusions and red blood cell transfusions may be recommended.[2]