Rare Disease Report

Aplastic Anemia - Quick Facts

MARCH 19, 2014
RDR Staff

Aplastic anemia is a rare condition that occurs when the bone marrow stops producing enough new blood cells. The cause is unknown.

Common Symptoms

  • Fatigue
  • Shortness of breath with exertion
  • Rapid or irregular heart rate
  • Pale skin
  • Frequent or prolonged infections
  • Unexplained or easy bruising
  • Nosebleeds and bleeding gums
  • Prolonged bleeding from cuts
  • Skin rash
  • Dizziness
  • Headache

The loss of red blood cells leaves the person fatigued and other problems, the loss white blood cells leave the person at great risk for infections and the loss of platelets leaves the person at risk for uncontrolled bleeding.


Apastic anemia can develop at any age and progress very quickly or fairly slowly. In most cases, the cause of aplastic anemia is not known (idiopathic aplastic anemia). However, in some cases, the condition has been linked to:

  • Toxins, such as pesticides, arsenic, and benzene.
  • Radiation and chemotherapy (treatments for cancer).
  • Medicines, such as chloramphenicol (an antibiotic rarely used in the United States).
  • Infectious diseases, such as hepatitis, Epstein-Barr virus, cytomegalovirus, parvovirus B19, and HIV.
  • Autoimmune disorders, such as lupus and rheumatoid arthritis.
  • Pregnancy. (Aplastic anemia that occurs during pregnancy often goes away after delivery.)

Some people with aplastic anemia also have a rare disorder known as paroxysmal nocturnal hemoglobinuria. This disorder causes red blood cells to break down too soon. Paroxysmal nocturnal hemoglobinuria can lead to aplastic anemia, or aplastic anemia can evolve into paroxysmal nocturnal hemoglobinuria.  Another rare disease, Fanconi's anemia may lead to aplastic anemia.


Treatments for aplastic anemia include blood transfusions, blood and marrow stem cell transplants, and medicines. These treatments can prevent or limit complications, relieve symptoms, and improve quality of life.

At present, there are no orphan drugs approved for aplastic anemia but there are several orphan drugs designated and being investigated as a possible treatment (e.g., placental adherent stromal cells, eltrombopag).

Medications may also be give to:

  • Stimulate your bone marrow
  • Suppress your immune system
  • Prevent and treat infections

For More Information

National Heart Lung and Blood Institute.

Aplastic Anemia & MDS International Foundation

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