cyclosporine, including tacrolimus (Prograf), daclizumab (Zenepax), and mycopheolate (Cellcept). High- dose cyclophosphamide (Cytoxan) also may be considered.
Hematopoietic Stem Cell Transplant
MATCHED SIBLING DONOR For children with an HLA matched family member, generally a matched sibling donor, the recommended treatment is a bone marrow transplant, also called a hematopoietic stem cell transplant (HSCT). Children can proceed to transplant without receiving any other treatment for their aplastic anemia. Children are admitted to the hospital for an HSCT. At the hospital, they have a central line placed (see page 7) to receive medications, IV fluids, and blood products that they will need during their HSCT. Two medications are used to destroy the malfunctioning immune system: ATG and cyclophosphamide (Cytoxan), a chemotherapy agent. After these medications have been given, new, healthy bone marrow that has been harvested (collected) from a sibling is infused into the patient. After approximately 10–20 days, the new marrow starts to grow and repopulates the bone marrow. As a result, blood counts begin to return to normal. Children usually are hospitalized for 20–50 days after an HSCT. During this time, they are monitored closely for complications as they await the growth of the new marrow. When they are transitioned to the outpatient clinic, they are seen frequently and monitored closely for engraftment (growing of the new blood cells) and complications of HSCT, including graft-versus-host disease, infections, and organ toxicity. Matched-sibling HSCT is a very effective treatment for aplastic anemia; however, very serious complica- tions can occur. The greatest risk for complications occurs during the first 2 years after HSCT; therefore, close monitoring is required during this time frame. ALTERNATIVE DONOR TRANSPLANT Children who do not have a matched sibling and do not respond to immune suppression or have recur- rence of their aplastic anemia are often referred for an alternative HSCT. Potential bone marrow donors can be an unrelated person that is identified from the National Marrow Donor Program (NMDP) or a partially matched family member, such as a parent. A member of the bone marrow transplant team will further discuss with you details of identifying a donor if this is needed for your child. This process is similar
to a matched-sibling HSCT but may require dif- ferent medications and possibly radiation for the new bone marrow to grow. Complications of HSCT Children who undergo HSCT are at risk for complications. However, the risks of transplant- related complications are higher with alternative donor transplants. Complications include the following: • Nonengraftment occurs when the new bone marrow does not grow. • Rejection occurs when the child’s own immune system reacts with the donor’s new immune system and the donor’s cells do not grow.
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