the standard recommended treatment. Anti-thymocyte globulin (ATG) combined with cyclosporine is the most common first-line therapy. ATG is made from horse or rabbit serum. It is produced by injecting the animals with human lymphocytes (a type of WBC that participates in the body’s immune response), which produces antibodies (a protein made by the immune system) against these lymphocytes that are then harvested. The ATG is then infused into the child and suppresses (lowers) his or her immune system. While receiving ATG, your child will receive antipyretics (fever reducing medication, such as Tylenol), antihistamines (such as Benadryl), and steroids. It is common for children to have fevers and chills during the infusion, and the medications are used to reduce these side effects. ATG is given very slowly through an IV, usually during a 4-day period. Because there is a risk for an adverse reaction to this treatment, children are hospitalized while receiving ATG. Serum sickness is another side effect that usually occurs 1–2 weeks after ATG is given. Symptoms include fever, rash, joint pain, and muscle aches. Serum sickness is treated with steroids and can last from several days to a couple of weeks. The other medication that is used in combination with ATG is cyclosporine. Cyclosporine is a medication that can be given orally (by mouth) or intravenously (into the blood via an IV). This medication is give twice daily and helps to suppress the immune system. It will be started at the beginning of treatment and will continue for 6 months or longer after your child has been discharged from the hospital. Your child’s healthcare team will monitor medication levels of this drug to make sure your child receives a therapeutic dose. This medication can have side effects including kidney damage, high blood pressure, low magne- sium levels in the blood, seizures, and excess hair growth. It is important to discuss any side effects your child may be experiencing with your healthcare team. In November 2018, the U.S. Federal Drug Administration (FDA) approved the medication Eltrombopag (Promacta®) for first-line treatment of severe aplastic anemia in children aged 2 years and older. Eltrom- bopag is an oral medication that is given once a day in combination with standard immunosuppressive therapy. Because data on the use of Eltrombopag in children with aplastic anemia is limited, this medica- tion is usually only offered to children as part of a clinical trial. After discharge from the hospital, children will be seen frequently in the outpatient clinic and their blood counts will be monitored for response to treatment and the need for transfusion. While on immune system suppression, children are at risk for serious infection and need to be monitored closely. They may require supportive care medications to prevent infection or treat organ toxicity. Response to immunosuppressive therapy may be slow and can take 3–6 months. About 75% of children will have improvement or complete recovery of their blood counts. About one-third of children who respond to immunosuppressive therapy will have a recurrence (relapse) of their aplastic anemia. Bone marrow transplant with a matched unrelated donor is the recommended treatment for children who do not respond to their initial course of immune suppression or for those who have a recurrence of aplastic anemia. A second course of immune suppression may be considered for children who do not have an appropriate unrelated donor. Other immunosuppressive drugs also have been tried in place of
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