Children and teens with Diamond-Blackfan anemia (DBA) are treated at Dana-Farber/Boston Children's through our Bone Marrow Failure Program , recognized as one of the nation’s best pediatric treatment and research programs for bone marrow failure and related conditions. Our patients have access to advanced treatments and diagnosis, including DNA mutation identification and ongoing clinical trials investigating new treatments. Dana-Farber/Boston Children's is also home to one of the largest and most experienced pediatric stem cell transplant centers in the world. Stem cell (bone marrow) transplant is currently the only cure for DBA.
A common complication of DBA is iron overload, which can affect the heart and liver. It results from the transfusions required for treatment. Treatment may include iron chelation drugs that remove iron from the blood to prevent complications. Steroids can have significant side effects, such as osteoporosis, weight gain, high blood pressure, and diabetes mellitus , all of which need to be monitored no matter the age of the patient. Younger patients are at particular risk for growth retardation. They may need a change in their treatment plan to ensure adequate growth in the first year of life.