Definitions

Aplastic anemia
Aplastic anemia is a condition in which the bone marrow stops producing new blood cells.

Autoimmune disorders
Autoimmune disorders are conditions in which the body’s immune system attacks its own cells, causing tissue destruction.

Fanconi anemia
This form of anemia is inherited and can lead to aplastic anemia, or bone marrow failure.

Inborn errors of metabolism
Inborn errors of metabolism include a number of inherited disorders that are caused by a deficiency of a specific substance, usually a protein, in the body. Inborn errors of metabolism may be treated with a bone marrow or stem cell transplant.

Juvenile rheumatoid arthritis
This form of arthritis affects children up to age 17 and causes joint inflammation and stiffness. One type of JRA can also affect internal organs. This is an autoimmune condition, which means the child’s immune system regards some of the body’s cells as foreign and attacks them.

Severe combined immunodeficiencies This refers to a group of rare inherited disorders characterized by little, if any, immune response.

Sickle-cell anemia
Sickle-cell anemia is an inherited disease characterized by abnormal red blood cells. The red blood cells become crescent (sickle) shaped, and as a result function abnormally.

Systemic lupus erythematosis Lupus is an autoimmune disease that can affect many tissues and cause inflammation throughout the body. Most often, lupus causes fatigue; painful, swollen joints; fever; rash; and kidney problems. It is more common in women than men, and more common in African-American women than women of other racial or ethnic backgrounds.

Systemic sclerosis (scleroderma)
In this autoimmune disease, scarring, or fibrosis occurs in the skin and other organs. This can be felt as thickening or firmness. In some cases, the effects are limited, usually to the face and fingers. In other cases, the disease is more diffuse, affecting tissues throughout the body.

Thalassemia
Thalassemia is an inherited problem with red blood cells. It can cause anemia — low levels of red blood cells. People with thalassemia do not create enough hemoglobin. This may mean: their body does not make enough red blood cells to begin with, their red blood cells break down more quickly than normal, their red blood cells are smaller than normal, or they have less hemoglobin in their red blood cells than normal.

July 2006