Human blood is composed of three types of cells (red blood cells, white blood cells, and platelets) that circulate throughout the body. Red blood cells contain hemoglobin (Hb) that carries oxygen from the lungs to all of the body’s muscles and organs. Oxygen provides the energy the body needs for all of its normal activities. Anemia occurs when the number of red blood cells falls below normal and the body gets less oxygen and therefore has less energy than it needs to function properly.
Aplastic anemia occurs when the bone marrow stops making enough blood cells. When this happens, the bone marrow is almost empty of blood forming cells and is described as hypoplastic or aplastic (meaning low- or no growth). Anemia results from reduced red cell production. It is estimated that there are approximately 1,000 new cases of aplastic anemia each year in the United States.
Commonly Prescribed (On-Label) Drugs: Lymphocyte Immune Globulin, Oxymetholone
Off-Label Prescription Drugs Breakthrough Options: Antithymocyte Globulin, Cyclosporine, Epoetin Alpha, Sargramostin
Autoimmune hemolytic anemia (AIHA) is a group of disorders characterized by a malfunction of the immune system that produces autoantibodies (proteins that attack other proteins or substances found in organs of the body), which attack red blood cells as if they were substances foreign to the body. Autoimmune hemolytic anemia is an uncommon group of disorders that can occur at any age. These disorders affect women more often than men. About half of the time, the cause of AIHA cannot be determined, which is referred to as idiopathic autoimmune hemolytic anemia. AIHA can also be caused by or occur with another disease, such as lupus.
Commonly Prescribed (On-Label) Drugs: Betamethasone, Cortisone, Dexamethasone, Fludarabine, Methylprednisolone, Prednisolone, Prednisone
Off-Label Prescription Drugs Breakthrough Options: Cyclophosphamide, Cyclosporine, Danazol, Rituximab
Therapy-resistant anemia is a subgroup in the myelodysplastic syndromes (MDS), also called pre-leukemia or “smoldering” leukemia. These are disorders in which the bone marrow - the spongy tissue inside the large bones - does not function normally. Bone marrow cells called “blast” develop or mature into several different types of blood cells including red blood cells that carry oxygen and other materials to all tissues of the body. The marrow cells that produce red cells appear abnormal. The white cell and producing cells may also appear abnormal. The proportion of blast cells is near normal levels. Therapy-resistant anemia accounts for about 20 to 30 percent of MDS cases. This form of the disease rarely transforms to acute leukemia.
Commonly Prescribed (On-Label) Drugs: None
Off-Label Prescription Drugs Breakthrough Options: Epoetin Alpha, Sargramostim
Thalassemia includes a number of different forms of anemia (red blood cell deficiency). The two main types are called alpha and beta thalassemias, depending on which part of an oxygen-carrying protein (called hemoglobin) is lacking in the red blood cells. Thalassemia consists of a group of inherited blood diseases. About 100,000 babies worldwide are born with severe forms of the disease each year. Thalassemia occurs most frequently in people of Italian, Greek, Middle Eastern, Southern Asian, and African ancestry.
Commonly Prescribed (On-Label) Drugs: None
Off-Label Prescription Drugs Breakthrough Options: Hydroxyurea
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