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Aplastic Anemia

Overview

Aplastic anemia is a condition that occurs when your body stops producing enough new blood cells. The condition leaves you fatigued and more prone to infections and uncontrolled bleeding.

A rare and serious condition, aplastic anemia can develop at any age. It can occur suddenly, or it can come on slowly and worsen over time. It can be mild or severe.

Treatment for aplastic anemia might include medications, blood transfusions or a stem cell transplant, also known as a bone marrow transplant.

Symptoms

Each type of blood cell has a different role:

  • Red cells carry oxygen around the body.
  • White cells fight infections.
  • Platelets prevent bleeding.

Your symptoms depend on what type of blood cells you're low on, but you may be low on all three. These are common symptoms for each:

Low red blood cell count:

  • Tiredness
  • Shortness of breath
  • Dizziness
  • Pale skin
  • Headaches
  • Chest pain
  • Irregular heartbeat
  • Low white blood cell count:
  • Infections
  • Fever
  • Low platelet count:
  • Easy bruising and bleeding
  • Nosebleeds

If you have any of these symptoms, your doctor may do a test called a complete blood count. They may also take a biopsy of your bone marrow to check you for this disorder.

Diagnosis

Severe aplastic anemia is a serious disorder requiring prompt medical attention. To diagnose aplastic anemia, physicians must examine the cells of the bone marrow and blood under a microscope. To do this, they will likely perform blood and laboratory tests as well as a bone marrow aspiration and biopsy. During this test, a sample of bone marrow is removed from the hipbone with a special needle under local anesthetic. The physician will check the number of blood cells, including white cells known as neutrophils, clotting cells known as platelets, and young, regenerating red cells known as reticulocytes.  Your doctor may also study kidney function or order other associated tests. HLA-typing, a laboratory blood test, is usually performed to ensure that patients receive immune system-compatible blood and marrow products during treatment.

Ideally, the diagnosis should be made by an experienced physician at a bone marrow failure specialty center.

Classification

Classifying aplastic anemia depends on the numbers of blood cells present in the blood tests and bone marrow biopsy. Aplastic anemia may be classified as moderate (MAA), severe (SAA), or very severe (VSAA). SAA and VSAA are usually treated aggressively while MAA can be observed in appropriate situations.

MANAGEMENT AND TREATMENT

Your treatment plan depends on several factors, such as your age, overall health and symptoms. Aplastic anemia treatment may include:

Immunosuppressants: These medicines suppress your immune system. Aplastic anemia symptoms improve in about 2 out of 3 people who take immunosuppressive medications.

Bone marrow stimulants: Certain types of drugs can stimulate your bone marrow to produce more blood cells. You may take sargramostim (Leukine®), filgrastim (Neupogen®) or epoetin alfa (Epogen®).

Blood transfusion: Transfusions replace red blood cells and platelets. Transfusions are not a cure for aplastic anemia, but they can treat symptoms like fatigue or bruising easily.

Supportive antibiotics depending on the severity of your blood counts.

A bone marrow transplant is the only cure for aplastic anemia. Bone marrow transplants are also called stem cell transplants. A transplant is the preferred treatment for severe aplastic anemia.

Bone marrow transplants replace damaged stem cells with healthy ones. The healthy stem cells may come from:

Donated bone marrow.

Peripheral stem cells (stem cells that providers remove and treat before transplanting).

Umbilical cord blood.

OUTLOOK / PROGNOSIS

The prognosis (outlook) for people with aplastic anemia is typically good. Most people who receive treatment can live a high-quality life. A bone marrow transplant may cure aplastic anemia.

Many people with aplastic anemia also have other blood disorders, such as:

Fanconi anemia, a rare hereditary condition that causes birth defects (congenital disabilities) such as undeveloped arms or legs.

Paroxysmal nocturnal hemoglobinuria, an acquired disorder when your red blood cells break down too quickly.

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