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Severe aplastic anemia (SAA)

Aplastic anemia is a form of bone marrow failure: the bone marrow does not produce enough red and white blood cells and platelets. Every year, 5-10 children in the Netherlands are diagnosed with aplastic anemia.

The brochure entitled Bone marrow failure and myelodysplastic syndrome contains comprehensive information about this disease. The most important information is provided below.

Causes

Aplastic anemia can be hereditary, but it can also be the result of a virus infection or medication. Usually, the cause is unknown, in which case it is probably an autoimmune process: the child's own immune cells (T lymphocytes) impede the production of blood cells.

Symptoms

Children with aplastic anemia can be pale, tired, dizzy and short of breath. Other symptoms include small, pointed blood spots, bruises, recurring infections, fever and sometimes abdominal pain, bone pain and an enlarged spleen.

How is aplastic anemia diagnosed?

Blood tests are done and bone marrow is extracted under sedation. Genetic testing is also carried out. Sometimes a small piece of bone is removed under anesthesia.

Treatment

Children with aplastic anemia are treated according to a protocol drawn up by national and international experts.
When blood cells are no longer produced, your child is dependent on transfusions. Your child is regularly given red blood cells and platelets and daily (low dose) antibiotics and sometimes antifungal medicines to prevent infections.

If your child has autoimmune aplastic anemia and a suitable donor is found, your child will get an allogeneic stem cell transplant. If no donor is found, your child will receive medication to suppress the T lymphocytes. If that medication helps, it will eventually no longer be needed and your child is cured. If the medication is not effective enough, your child will remain dependent on those drugs and an allogeneic stem cell transplant may still be necessary in the long term.

Survival rate

The survival rate after an allogeneic stem cell transplant is very favorable, but children can also be cured with drugs that suppress T lymphocytes.

Questions?

Children with aplastic anemia are treated in the hematology-oncology department.

Telephone 088-1111111