Heart and Blood Conditions

Diamond Blackfan Anemia Treatment

How is Diamond Blackfan anemia treated?

There are several different treatment options your doctor may recommend.

Steroids (corticosteroids)

  • Steroids are medicine that can help produce more red blood cells. Steroids are often the first course of treatment.
  • Potential side effects may include decreased ability to fight infections, appetite changes, upset stomach, sleep problems, high blood sugar, high blood pressure, low bone density and cataracts. To avoid these side effects, the steroid dose is tapered to the minimum dose necessary.
  • 80% of patients will respond to steroid treatment.

Red blood cell transfusion

A blood transfusion (adding blood into your child's body) is another way to increase red blood cells in the body. Transfusions are most often used when anemia is severe and steroid therapy is not effective. How often your child varies.

One side effect of blood transfusions is that iron can build up after multiple transfusions and damage vital organs. Iron chelation therapy is used to treat iron overload in children who receive frequent red blood cell transfusions. Iron levels are checked regularly with blood tests and specialized magnetic resonance imaging (MRI) exams.

Bone marrow transplant

A hematopoietic cell transplant (also called a bone marrow transplant or stem cell transplant) may cure bone marrow failure, including DBA. Hematopoietic (him-at-oh-poy-EH-tik) cells are blood-forming cells inside the bone marrow. They are a type of stem cell or immature cell. These immature blood-forming cells are taken from a donor's bone and infused into your child's vein, with the potential for curing the marrow failure.

Hematopoietic cell transplantation is a complicated procedure that is best performed at a center skilled in treating patients with DBA. We work closely with the Fred Hutchinson Cancer Research Center to provide care for children who need a bone marrow or stem cell transplant. We specialize in transplant regimens that result in reduced toxicity and side effects. Learn more about pediatric bone marrow and stem cell transplants offered through the Pediatric Blood and Marrow Transplant Program.

Your medical team will discuss treatment options as needed. Not every child will need these treatments. Instead, your provider might recommend waiting and watching your child's counts closely.

Our hematology nurse and nonmalignant transplant coordinator can assist with answering questions, coordination and follow-up care.