Aplastic Anemia

Overview

What is aplastic anemia?

Aplastic anemia is a rare blood disorder. This serious condition is a type of bone marrow failure syndrome. If you have aplastic anemia, the springy tissue inside your bones (bone marrow) does not produce enough white blood cells (leukopenia or neutropenia), red blood cells (anemia) or platelets (thrombocytopenia).

What does bone marrow do?

In healthy bone marrow, stem cells produce three types of blood cells. When these blood cells are fully formed, they leave your bone marrow and enter your bloodstream.

  • Red blood cells transport oxygen by getting it from the lungs and circulating it throughout your body.
  • White blood cells (leukocytes) fight and destroy bacteria and germs.
  • Platelets stop bleeding by helping your blood to clot.

How does aplastic anemia affect my body?

When you have aplastic anemia, your body doesn’t create enough blood cells because you don’t have adequate amounts of stem cells. Usually, acquired aplastic anemia occurs because an immune trigger (disease where the body attacks itself) damages your stem cells.

What are the types of aplastic anemia?

There are two types of aplastic anemia:

  • Inherited aplastic anemia occurs because of a random gene mutation. It is most common in children and younger adults.
  • Acquired aplastic anemia occurs because of an immune system problem. It is most common in older adults, but can occur in younger adults.

Who might get aplastic anemia?

Aplastic anemia can affect anyone of any race or gender. You are more likely to have aplastic anemia if you are:

  • Asian or Asian-American.
  • A teenager or young adult.
  • Over the age of 65.

How common is aplastic anemia?

In the United States, experts don’t know exactly how many people have aplastic anemia. Some sources show that, annually, around 500 to 1,000 people receive an aplastic anemia diagnosis.

Symptoms and Causes

What causes aplastic anemia?

Often, healthcare providers cannot identify exact aplastic anemia causes. Factors that increase your risk of having the condition include:

  • Autoimmune disorders such as eosinophilic fasciitis or lupus.
  • Drugs and chemical exposures.
  • Infections such as HIV, hepatitis or Epstein-Barr virus (the virus that causes mononucleosis).
  • Medical treatments such as radiation therapy or chemotherapy.
  • Paroxysmal nocturnal hemoglobinuria, an acquired disorder when your red blood cells break down too quickly.
  • Family history of bone marrow failure syndrome.
  • Pregnancy.

What are the symptoms of aplastic anemia?

Many people with this condition present with minimal symptoms, but with low blood counts. Most common symptoms include:

  • Bleeding/bruising easily.
  • Heavy menstrual bleeding.
  • Dyspnea (shortness of breath).
  • Fatigue.
  • Frequent infections.

Aplastic anemia symptoms can affect people differently. They may come on slowly or suddenly. Some people may have manageable, mild symptoms. Others may have symptoms that indicate severe aplastic anemia. Severe aplastic anemia requires urgent medical treatment.

What are the complications of untreated bone marrow failure?

Without treatment, people may experience severe complications. These complications include:

  • Bleeding.
  • Severe infections (viral, bacterial and fungal).
  • Heart complications (arrhythmia, heart failure).
  • Disease progression to myelodysplastic syndrome or acute leukemia.

Diagnosis and Tests

What tests are used in aplastic anemia diagnosis?

Healthcare providers use a series of tests to diagnose aplastic anemia. They may order:

  • Blood tests to measure your platelet, red blood cell and white blood cell levels.
  • Bone marrow biopsy to remove a sample of your bone marrow and evaluate it under a microscope.

Management and Treatment

How is aplastic anemia treated?

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.

Is there a treatment that cures aplastic anemia?

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.

How do I find a match for a bone marrow transplant?

A successful bone marrow transplant requires a good donor match. A good donor match is someone with the same blood type or similar genetic makeup. For most people, the best match is a close relative such as a sibling or parent.

If you do not have a known donor match, you may enlist in a national registry. This registry pairs you with a compatible donor. You may need immunosuppressive therapy or other treatment while you wait for a good donor match.

Prevention

How can I prevent aplastic anemia?

There is no guaranteed way to prevent aplastic anemia.

Outlook / Prognosis

What is the outlook for people with aplastic anemia?

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.

Living With

How do I take care of myself if I have aplastic anemia?

If you have aplastic anemia, it’s important to:

  • Avoid high-contact activities: Sports like football, hockey or wrestling come with a risk of injury. Because aplastic anemia carries a risk of uncontrolled bleeding, you may need to avoid contact sports where you could get injured.
  • Protect yourself from viruses and germs: Wash your hands thoroughly and frequently. Avoid exposure to sick people, wear a mask and get all recommended vaccines, including your annual flu shot. If you do start to show signs of infection, see your healthcare provider right away.
  • Rest as needed: If you have anemia, you may feel short of breath even after mild-intensity activities. Rest when you need to, and take a lot of breaks.

What else should I ask my healthcare provider?

You may want to ask your healthcare provider:

  • What is the most likely cause of aplastic anemia?
  • What can I do to manage aplastic anemia symptoms?
  • What treatments do you recommend?
  • Are there any side effects of aplastic anemia treatments?
  • How can I best manage aplastic anemia and another health condition?

A note from Cleveland Clinic

Aplastic anemia is a rare but serious bone marrow disorder. It occurs when your stem cells don’t create enough blood cells. You may bruise easily, have fatigue or suffer from shortness of breath. Without treatment, aplastic anemia can increase the risk of serious infections, bleeding, heart problems and other complications. The only cure for aplastic anemia is a bone marrow transplant. If you need to wait for a bone marrow donor who is a good match, you may take immunosuppressive medicines such as antithymocyte globulin (ATG), cyclosporine or tacrolimus, and thrombopoietin receptor agonist eltrombopag. Most people who receive aplastic anemia treatment live a high quality of life.

Last reviewed by a Cleveland Clinic medical professional on 08/02/2021.

References

  • Aplastic Anemia & MDS International Foundation. . Accessed 9/1/2021.Aplastic Anemia (https://www.aamds.org/diseases/aplastic-anemia)
  • Medline Plus. . Accessed 9/1/2021.Aplastic Anemia (https://medlineplus.gov/aplasticanemia.html)
  • National Heart, Lung, and Blood Institute. . Accessed 9/1/2021.Aplastic Anemia (https://www.nhlbi.nih.gov/health-topics/aplastic-anemia)
  • National Organization for Rare Disorders. . Accessed 9/1/2021.Acquired Aplastic Anemia (https://rarediseases.org/rare-diseases/acquired-aplastic-anemia/)

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