Treatment options for anemia include:
Corticosteroids. About 80 percent of patients respond to corticosteroids. Side effects from this treatment, particularly at high doses, may lower the body's ability to fight off infections or make them harder to treat, and may cause appetite changes, nervousness, restlessness, sleep problems, and indigestion, high blood pressure, high blood sugar, low bone density and cataracts. For some patients the side effects will not resolve and the patient has to discontinue this therapy. For others, remission may allow them to also discontinue the therapy.
Red cell transfusion support. This treatment begins after the initial diagnosis and again later if the patient doesn’t respond well to corticosteroid treatment. Iron is released from the breakdown of red blood cells and is not recycled into new red cells in people with DBA. With no way for the body to excrete this additional iron, patients run the risk iron overload, which can damage the heart, liver, pancreas, thyroid, and other organs. Iron levels will need to be monitored when receiving regular transfusions and chelation therapy may be required to handle the excess iron. Chelation therapy must be individualized for the needs of each patient.
Bone marrow transplant (hematopoietic stem cell transplant) is curative for DBA. HLA-matched sibling donor transplants appear to have better outcomes than unmatched donor transplants. However, at Seattle Cancer Care Alliance, the unrelated donor transplant results for bone marrow failure diseases are similar to those obtained using matched sibling donors.
Hematopoietic cell transplantation is a complicated procedure that is best performed in large transplant centers, such as Fred Hutchinson Cancer Research Center/Seattle Cancer Care Alliance and Seattle Children’s, where there is an interest and long standing experience with performance of this type of transplant.
SCCA and Seattle Children’s hematologists work in partnership to care for adults with this disease.
Regular monitoring by a hematologist experienced in this disease is important, as anemia can improve or worsen over time. Regular monitoring also assists the early detection of a malignancy should it occur.