Most patients with less severe forms of alpha thalassemia may not require specific treatment. As needed, treatment for decreased hemoglobin concentrations may be required, including supplementation with iron or folic acid in some cases. In addition, the subset of patients with more severe forms of alpha thalassemia may also require transfusion therapy.
Iron and/or Folic Acid Supplementation
Although iron supplementation may help in some cases of alpha thalassemia, there are concerns with iron oveload as well. Iron overload is a particular concern in patients with hemoglobin H (HbH) disease.
Folic acid supplementation can be beneficial in cases where hemolysis rates, caused by alpha thalassemia, require the body to utllize more folic acid due to increased erythropoiesis. Recall that the red blood cell lifespan is often decreased in alpha thalassemia patients, causing the body to compensate with higher red blood cell production rates and hyperplastic bone marrow.
Transfusion Therapy
General supportive care in more severe forms of alpha thalassemia may include transfusion of red blood cells. In these cases, transfusion standards for alpha thalassemia are carefully followed. In general, however, blood transfusions are administered only when necessary. Care is taken to ensure that patients with lifetime transfusion therapy requirements are evaluated for iron overload and recieve chelation therapy when needed.
Stem Cell Transplants
In very severe cases of alpha thalassemia, allogeneic hematopoietic stem cell transplantation may become a treatment option. It is rare to find a suitable donor for this procedure, as a sibling who is fully matched for human leukocyte antigen (HLA) and who is missing, or only a carrier for, the alpha thalassemia gene deletion is the ideal donor. The risks involved in stem cell transplants often outweigh the potential therapeutic benefits in alpha thalassemia patients, and is therefore only utilized in the most severe cases.
Future Therapy Options
Both gene therapy and triggering the adult production of fetal hemoglobin are considerations for the next generation of alpha thalassemia treatment.
