Endari Endari®, an amino acid (also called L-glutamine), is a drug approved by the FDA in 2017 to treat sickle cell disease. Endari is a powder taken by mouth twice a day that helps to decrease the number of pain episodes in patients with sickle cell disease who are 5 years of age and older. It is important to take all medications as prescribed. Talk to your child’s hematology team to find out if this medication is right for your child. ADAKVEO (Crizanlizumab-tmca) ADAKVEO® is an infusion drug approved by the FDA in 2019 to help reduce how often pain episodes occur in patients with sickle cell disease. ADEKVEO works by making blood vessels and certain blood cells less sticky. It is approved as a once-monthly infusion to be used in patients 16 years of age and older. The infusion must be given by a healthcare provider. Talk to your hematology healthcare team to find out if ADEKVEO can be given to your child. Oxbryta (Voxelotor, GBT440) Oxbryta® is an oral drug (to be taken by mouth) approved by the FDA in 2019 to treat sickle cell disease in patients 12 years of age and older. It helps hemoglobin hold on to oxygen and helps prevent the red blood cells from becoming sickled. Oxbryta also should help reduce the risk of pain crises caused by sickle cells blocking small blood vessels. Your hematology healthcare provider can give you more information on this drug. Stem Cell Transplant Stem cell transplant is the only cure for sickle cell disease. Because stem cell transplant has the potential for very serious side effects, it most often is used for those with severe sickle cell disease. However, there is research looking closely into the benefits of stem cell transplant before significant complications from the disease are experienced. Before a stem cell transplant, the child will be given chemotherapy and/or radiation to destroy their own bone marrow (where blood cells are made). He or she then receives an infusion of a donor’s stem cells. In the weeks to months following the transplant, the donor cells take over and produce blood cells that are unaffected by sickle cell disease. Stem cell transplant does have risks, including serious infection, bleeding, and organ damage from the chemotherapy and radiation. Graft-versus-host disease, a condition in which the donor’s stem cells begin to attack the patient’s body cells, also may develop. In some cases, these complications can be fatal. Additionally, the stem cells may not “take,” which is a condition called graft failure.
n WHAT RESEARCH IS TAKING PLACE? Fortunately, there is a great deal of research being done in the area of sickle cell disease. For instance, studies are taking place to improve and limit the side effects of current treatments and additional research is looking at new treatment options.
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