Eltrombopag for SAA - Clinical Trial

We are doing this study to understand how the body absorbs, distributes, breaks down, and eliminates eltrombopag (also known as Promacta and Revolade) in children with severe aplastic anemia.

Severe Aplastic Anemia (SAA)

Children ages 1-17 years who:
- Have Severe Aplastic Anemia
- Don't have congenital/inherited bone marrow failure syndromes
- Have refused or are not eligible for hematopoietic stem cell transplantation (HSCT)

If you decide that you would like for your child to be part of this study, your child will:  
- Get the study medicine, eltrombopag by mouth.
- Be asked to give blood and urine samples
- Be asked to come to the clinic once a week for the first 8 weeks, then once every 2 weeks for the next 6 weeks, then visit once a month for a total 18 months
- Come for visits once every 3 months for the following 3 years.