It's no longer news that bone marrow transplants aka stem cell transplant offers the only potential cure for sickle cell anemia. Treatment is therefore only aimed at managing/avoiding crisis as much as possible, preventing complications and making living with the disease more bearable.
However, in recent years there have been improvements with care through medication some of which are still undergoing clinical trials while others have been approved for treatment of the disease by the FDA.
Below are 5 drugs for the treatment of sickle cell anemia that you should ask your doctor about if you were not already aware of them:
1. Endari - Approved by the FDA to decrease the pain complications in sickle cell patients, it was approved in July 2017 for use in patients aged 5 and older making it the first approved pediatric treatment for sickle cell disease and the first new treatment for adults in almost 20 years.
2. Oral THU-decitabine - This drug has shown in clinical studies, increased hemoglobin F and improved biomarkers of hemolysis and inflammation. It is currently still being studied to potentially find a lasting way to change the natural history of sickle cell by addressing its root cause pathophysiology aka the disordered physiological processes associated with the disease.
3. Voxelotor - This drug was discussed in my post last week and it looks to reduce the risk of stroke and organ failure in patients if eventually approved by the FDA.
4. Hydroxyurea & Glutamine - These are the only 2 drugs currently approved by the FDA that help manage crisis and complications from the disease.
5. Crizanlizumab - The FDA has given priority review to an approval application of this drug to treat scd. It is to be used as treatment to prevent vaso-occlusive crises in patients which are unpredictable and extremely painful events that can be life threatening. They are the most common cause of emergency room visits.
I think it is safe to say that a lot is being done to alleviate the painful episodes and life threatening complications SC patients suffer through with the introduction of possible new medication. However, more could be done to hopefully have a complete cure one day asides bone marrow transplants that require a donor from within the immediate family. Let's hope that this happens sooner rather than later.
Will you be asking your doctor about any of these potential drugs or are you already aware of their existence? Comment below..
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