The Rare Anemia and Iron Disorder Program at Dana-Farber/Boston Children's Cancer and Blood Disorders Center provides comprehensive medical evaluation and treatment for children with all types of rare anemias and iron disorders, including:
- congenital sideroblastic anemia
- congenital dyserythropoietic anemias (CDA)
- rare hemoglobinopathies
- iron-refractory iron deficiency anemia (IRIDA)
- hereditary hemochromatosis
- red cell enzyme deficiencies
- red cell membrane disorders (e.g. hereditary spherocytosis, elliptocytosis, and xerocytosis)
- pyruvate kinase deficiency
We also provide ongoing medical management, counseling and support, and access to new treatment approaches through clinical research.
How we diagnose and treat rare anemias and iron disorders
Rare anemias are diagnosed through careful review of blood and urine tests. These tests may involve a microscopic review of the peripheral blood and a marrow exam. Iron disorders are diagnosed through laboratory testing, iron challenge, and MRI or iron burden assessment. In some cases, a patient may have no known diagnosis, yet the symptoms are present in multiple family members. These families are often invited to participate in research studies aimed at establishing the diagnosis though genetic discovery. Findings that come out of this research can then be translated into innovative new therapies.
The strength of our expertise and facilities enables us to prescribe the most appropriate treatment for each and every diagnosis. A treatment plan may include red blood cell transfusions, intravenous immune globulin to strengthen the immune system, an iron-rich diet or iron supplements, exchange transfusions to replace damaged blood with fresh blood, surgery, immunosuppressive therapy, or partial exchange transfusions to slowly remove and replace a large portion of a patients blood volume.
Our treatment team
Dana-Farber/Boston Children's patients have access to the broadest set of blood disorder expertise and pediatric subspecialties available. The breadth of our expertise allows us to assemble a team of specialists to meet the specific needs of the patient.
Our treatment teams are led by pediatric hematologists who specializes in inherited iron deficiency disorders, sideroblastic anemia, and iron overload. We also have nurse practitioners and physician assistants who specialize in treating anemias, hemoglobinopathies, and red blood cell disorders and a designated hematology patient coordinator. When necessary, we can augment our team with pediatric hematologist/oncologists and pediatric hematopathologists.