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Aplastic anemia

Medically reviewed by Drugs.com. Last updated on Feb 1, 2024.

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

Aplastic anemia can have no symptoms. When present, signs and symptoms can include:

Aplastic anemia can be short-lived, or it can become chronic. It can be severe and even fatal.

Causes

Stem cells in the bone marrow produce blood cells — red cells, white cells and platelets. In aplastic anemia, stem cells are damaged. As a result, the bone marrow is either empty (aplastic) or contains few blood cells (hypoplastic).

The most common cause of aplastic anemia is from your immune system attacking the stem cells in your bone marrow. Other factors that can injure bone marrow and affect blood cell production include:

Connections with other rare disorders

Some people with aplastic anemia also have a rare disorder known as paroxysmal nocturnal hemoglobinuria, which causes red blood cells to break down too soon. This condition can lead to aplastic anemia, or aplastic anemia can evolve into paroxysmal nocturnal hemoglobinuria.

Fanconi's anemia is a rare, inherited disease that leads to aplastic anemia. Children born with it tend to be smaller than average and have birth defects, such as underdeveloped limbs. The disease is diagnosed with the help of blood tests.

Bone marrow

Bone marrow is a red, spongy material inside your bones that produces blood cells.

Risk factors

Aplastic anemia is rare. Factors that can increase risk include:

Prevention

There's no prevention for most cases of aplastic anemia. Avoiding exposure to insecticides, herbicides, organic solvents, paint removers and other toxic chemicals might lower your risk of the disease.

Diagnosis

The following tests can help diagnose aplastic anemia:

Once you've received a diagnosis of aplastic anemia, you might need other tests to determine the cause.

Bone marrow exam

In a bone marrow aspiration, a health care provider uses a thin needle to remove a small amount of liquid bone marrow, usually from a spot in the back of your hipbone (pelvis). A bone marrow biopsy is often done at the same time. This second procedure removes a small piece of bone tissue and the enclosed marrow.

Treatment

Treatments for aplastic anemia, which will depend on the severity of your condition and your age, might include observation, blood transfusions, medications, or bone marrow transplantation. Severe aplastic anemia, in which your blood cell counts are extremely low, is life-threatening and requires immediate hospitalization.

Blood transfusions

Although not a cure for aplastic anemia, blood transfusions can control bleeding and relieve symptoms by providing blood cells your bone marrow isn't producing. You might receive:

While there's generally no limit to the number of blood transfusions you can have, complications can sometimes arise with multiple transfusions. Transfused red blood cells contain iron that can accumulate in your body and can damage vital organs if an iron overload isn't treated. Medications can help rid your body of excess iron.

Over time your body can develop antibodies to transfused blood cells, making them less effective at relieving symptoms. The use of immunosuppressant medication makes this complication less likely.

Stem cell transplant

A stem cell transplant to rebuild the bone marrow with stem cells from a donor might be the only successful treatment option for people with severe aplastic anemia. A stem cell transplant, also called a bone marrow transplant, is generally the treatment of choice for people who are younger and have a matching donor — most often a sibling.

If a donor is found, your diseased bone marrow is first depleted with radiation or chemotherapy. Healthy stem cells from the donor are filtered from the blood. The healthy stem cells are injected intravenously into your bloodstream, where they migrate to the bone marrow cavities and begin creating new blood cells.

The procedure requires a lengthy hospital stay. After the transplant, you'll receive drugs to help prevent rejection of the donated stem cells.

A stem cell transplant carries risks. Your body may reject the transplant, leading to life-threatening complications. In addition, not everyone is a candidate for transplantation or can find a suitable donor.

Immunosuppressants

For people who can't undergo a bone marrow transplant or for those whose aplastic anemia is due to an autoimmune disorder, treatment can involve drugs that alter or suppress the immune system (immunosuppressants).

Drugs such as cyclosporine (Gengraf, Neoral, Sandimmune) and anti-thymocyte globulin suppress the activity of immune cells that are damaging your bone marrow. This helps your bone marrow recover and generate new blood cells. Cyclosporine and anti-thymocyte globulin are often used together.

Corticosteroids, such as methylprednisolone (Medrol, Solu-Medrol), are often used with these drugs.

Although effective, these drugs further weaken your immune system. It's also possible for anemia to return after you stop these drugs.

Bone marrow stimulants

Certain drugs — including colony-stimulating factors, such as sargramostim (Leukine), filgrastim (Neupogen) and pegfilgrastim (Neulasta), epoetin alfa (Epogen/Procrit), and eltrombopag (Promacta) — help stimulate the bone marrow to produce new blood cells. Growth factors are often used with immune-suppressing drugs.

Antibiotics, antivirals

Having aplastic anemia weakens your immune system, which leaves you more prone to infections.

If you have aplastic anemia, see your doctor at the first sign of infection, such as a fever. You don't want the infection to get worse, because it could prove life-threatening. If you have severe aplastic anemia, your doctor might prescribe antibiotics or antiviral medications to help prevent infections.

Other treatments

Aplastic anemia caused by radiation and chemotherapy treatments for cancer usually improves after those treatments stop. The same is true for most other drugs that induce aplastic anemia.

Pregnant women with aplastic anemia are treated with blood transfusions. For many women, pregnancy-related aplastic anemia improves once the pregnancy ends. If that doesn't happen, treatment is still necessary.

Self care

If you have aplastic anemia, take care of yourself by:

Coping and support

Tips to help you and your family better cope with your illness include:

Preparing for your appointment

Start by making an appointment with your primary care doctor. He or she might then refer you to a doctor who specializes in treating blood disorders (hematologist). If aplastic anemia comes on suddenly, your treatment might begin in the emergency room.

Here's some information to help you get ready for your appointment.

What you can do

Make a list of:

Take a family member or a friend with you to your doctor, if possible, to help you remember the information you're given.

For aplastic anemia, questions to ask your doctor include:

What to expect from your doctor

Your doctor is likely to ask you questions, such as:

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