Aplastic Anemia

Aplastic anemia is an uncommon but serious condition that occurs when the body fails to produce enough new blood cells. This happens when the bone marrow’s healthy blood-forming stem cells are damaged or destroyed, leaving the marrow empty (aplastic) or partially depleted (hypoplastic).

Although aplastic anemia is not classified as a cancer, it is sometimes linked to leukemia. The condition may also develop as a side effect of certain cancer treatments—particularly high-dose chemotherapy or radiation therapy—which can harm healthy stem cells and disrupt normal blood cell production.

What are the causes and risk factors for aplastic anemia?

The exact cause of aplastic anemia is often unknown (idiopathic). Although the condition can affect anyone, several factors may increase the risk. These include:

• Autoimmune disorders – Conditions such as lupus and rheumatoid arthritis can trigger an immune system attack on the bone marrow, destroying its stem cells and reducing blood cell production.
• Long-term exposure to toxins – Chemicals such as benzene, pesticides and heavy metals can impair bone marrow function over time.
• Certain medications – Some antibiotics, anti-inflammatories and chemotherapy drugs may interfere with bone marrow activity.
• Viral infections – Infections such as hepatitis, Epstein-Barr virus (EBV), cytomegalovirus and human immunodeficiency virus (HIV) can weaken bone marrow function.
• Radiation therapy and chemotherapy – Cancer treatments involving high-energy X-rays or cytotoxic drugs can suppress the production of new blood cells.
• Genetic predisposition – Some rare inherited disorders, such as Fanconi anemia, may increase susceptibility to aplastic anemia.

Is aplastic anemia preventable?

Aplastic anemia cannot always be prevented, especially when it develops due to genetic factors or has no identifiable cause. However, reducing exposure to known risk factors may help lower the likelihood of developing the condition. Steps to support prevention include:

• Avoiding harmful substances – Limit exposure to toxic chemicals and environmental pollutants, particularly in occupational settings.
• Using medications safely – Take prescription and over-the-counter drugs only under medical supervision and discuss any potential effects on bone marrow health with a physician.
• Preventing infections – Practice consistent hand hygiene, avoid contact with sick individuals when possible and stay current on recommended vaccinations.
• Seeking early medical care – Contact a healthcare provider promptly for symptoms such as unexplained fatigue, frequent infections or unusual bleeding. An early diagnosis and treatment can significantly improve the outcome.

What are the signs and symptoms of aplastic anemia?

Aplastic anemia develops when the bone marrow fails to produce enough of the three essential blood cell types—red blood cells, white blood cells and platelets—that are vital to maintaining overall health. Red blood cells deliver oxygen throughout the body, white blood cells defend against infection and platelets enable proper blood clotting.

Because aplastic anemia affects all three types of blood cells, it can produce a wide range of symptoms that may appear gradually or develop suddenly. Recognizing the early warning signs can allow for a timely diagnosis and treatment, which can ultimately improve the outcome.

What are the early warning signs of aplastic anemia?

Initially, aplastic anemia may cause mild symptoms that can be easily overlooked or mistaken for general tiredness or a minor illness, such as the common cold. Warning signs include:

• Unusual fatigue or weakness
• Pale or sallow complexion
• Shortness of breath during normal activities
• Frequent or lingering infections
• Small red or purple spots on the skin (petechiae)

These early warning signs may develop before the condition becomes severe, highlighting the importance of seeking a prompt medical evaluation.

What are the most common symptoms of aplastic anemia?

As aplastic anemia progresses, symptoms may become more noticeable and begin to interfere with everyday life. Some people may experience:

• Chronic fatigue and dizziness
• Rapid or irregular heartbeat
• Frequent nosebleeds or bleeding gums
• Prolonged bleeding from minor cuts and injuries
• Cold hands or feet
• Recurrent infections, such as respiratory and urinary tract infections (UTIs)

These symptoms reflect the inability of the bone marrow to produce new blood cells quickly enough to meet the body’s needs.

What are the symptoms of advanced aplastic anemia?

As blood cell levels continue to decline, advanced aplastic anemia may cause severe and sometimes life-threatening complications, such as:

• Extreme fatigue and shortness of breath, even at rest
• Severe, treatment-resistant infections
• Spontaneous bleeding in the skin, gums or internal organs
• Skin changes, such as widespread bruising or purplish patches
• Heart palpitations or chest pain
• Signs of organ stress or failure, such as swelling in the legs or abdomen

Severe or advanced aplastic anemia is a medical emergency that often requires immediate intervention, which may include blood transfusions, medications or a bone marrow transplant.

What are the signs that aplastic anemia has spread?

Because aplastic anemia is not a metastatic or contagious condition, it does not “spread” in the same way that cancer or an infection might. However, if left untreated, the effects of severe bone marrow failure can impact multiple organs throughout the body. Possible signs include:

• Widespread infection that may enter the bloodstream (sepsis)
• Severe bleeding in the gastrointestinal tract or brain
• Heart strain or heart failure from prolonged anemia
• Liver or kidney complications due to reduced oxygen and nutrient delivery

Rather than indicating that the condition has “spread,” these symptoms indicate systemic consequences of untreated or advanced aplastic anemia. Prompt medical treatment is essential to manage any complications and help prevent organ damage.

Florida's Top Choice for Cancer Care

Moffitt is Florida's top cancer hospital by expert opinion in America's Best Cancer Hospitals by U.S. News & World Report.

Schedule an Appointment

How is aplastic anemia diagnosed?

Aplastic anemia is diagnosed through a combination of medical history review, physical examination and diagnostic testing. Because its symptoms—such as fatigue, easy bruising and frequent infections—can resemble those of other blood disorders, multiple tests are usually needed to confirm the diagnosis. The primary goal is to determine whether the bone marrow is producing insufficient blood cells and, if so, identify the underlying cause.

During the diagnostic process, the physician will ask the patient about any exposures to toxins, recent infections, medications and family history of blood disorders. A series of lab tests, procedures and imaging scans can help the physician confirm the condition and assess its severity.

What laboratory tests are used to diagnose aplastic anemia?

Lab testing plays a key role in diagnosing aplastic anemia. The results can help the physician evaluate blood cell counts, rule out other conditions and assess bone marrow function. Common lab tests include:

• Complete blood count (CBC) – This blood test measures the levels of red blood cells, white blood cells and platelets in the bloodstream. Low counts across all three types suggest bone marrow suppression.
• Reticulocyte count – This blood test measures young red blood cells (reticulocytes) to determine how actively the bone marrow is producing new cells.
• Peripheral blood smear – A drop of blood is microscopically examined by a pathologist, who can assess the shape, size and number of blood cells present.
• Liver and kidney function tests – The results can help the physician rule out other causes of anemia and evaluate the patient’s overall health.
• Viral and autoimmune screening – Tests for hepatitis, EBV, HIV and autoimmune markers can help the physician identify possible causes of bone marrow failure.

What procedures are used to diagnose aplastic anemia?

The most definitive diagnostic test for aplastic anemia is a bone marrow aspiration and biopsy. This procedure can provide the physician with direct insight into how well the bone marrow is functioning.

Using a thin needle under local anesthesia, the physician will take a small sample of bone marrow, usually from the hip bone. The specimen will be sent to a lab for microscopic examination by a pathologist, who will assess the number and type of blood-forming cells present. Aplastic anemia usually causes the bone marrow to appear less cellular than normal, showing a marked decrease in the stem cells responsible for blood production.

What imaging tests are used to diagnose aplastic anemia?

Although aplastic anemia is primarily diagnosed through blood and bone marrow tests, imaging studies can help the physician identify organ damage, internal bleeding and infections related to low blood cell counts. Options may include:

• Chest X-ray – This common medical imaging test uses a small amount of radiation to create black and white pictures of the structures inside the chest, such as the heart, lungs and ribs. These images can help the physician check for heart enlargement or a lung infection related to severe anemia.
• Ultrasound – This safe, non-invasive medical imaging technique uses high-frequency sound waves to create pictures of the body’s inner structures. These images can help the physician evaluate liver, spleen or kidney enlargement that could suggest other blood disorders.
• Computed tomography (CT) and magnetic resonance imaging (MRI) scans – In some cases, advanced imaging techniques may be used to check for internal bleeding or infections, particularly in severe or advanced stages of aplastic anemia.

Although imaging may support the diagnostic process, it cannot directly confirm aplastic anemia on its own.

How is aplastic anemia treated?

Aplastic anemia treatment focuses on restoring healthy blood cell production and addressing the underlying cause of the bone marrow failure. The best approach can vary depending on the patient’s age and overall health, the severity of the condition and whether the physician has identified a specific cause.

A mild case of aplastic anemia might improve with supportive care, but a severe case may require more intensive treatment, such as blood transfusions, medications or bone marrow transplant. An early diagnosis and prompt treatment are essential for preventing complications and improving the long-term health outcome.

How is immunotherapy used to treat aplastic anemia?

Immunotherapy, also known as immunosuppressive therapy, is a cornerstone treatment for aplastic anemia, especially when the immune system is attacking the bone marrow. Commonly used immunotherapy drugs include antithymocyte globulin (ATG) and cyclosporine, which work by calming or “resetting” the body’s immune response, allowing the bone marrow to resume normal blood cell production.

Some patients may also receive eltrombopag, a medication that stimulates the bone marrow to produce more platelets and blood cells. While immunotherapy can significantly improve blood cell counts and reduce the need for transfusions, the treatment may need to continue for several months or longer to remain effective.

How is chemotherapy used to treat aplastic anemia?

In the treatment of aplastic anemia, chemotherapy is not used to destroy cancer cells but rather to suppress the immune system. Low doses of chemotherapy drugs, such as cyclophosphamide, may be administered alongside other medications to help prevent the immune cells from attacking the bone marrow stem cells.

This treatment approach may be considered for a patient who does not respond to standard immunosuppressive therapy and is not a candidate for bone marrow transplantation. Because chemotherapy can increase the risk of infection and cause other side effects, treatment will be carefully monitored by a hematologist.

How is bone marrow transplantation used to treat aplastic anemia?

Bone marrow transplantation (BMT) is one of the most effective treatments for severe aplastic anemia. This intensive procedure aims to replace the patient’s damaged or dysfunctional bone marrow with healthy stem cells capable of producing new blood cells. BMT can restore normal bone marrow function and, in many cases, offers the best chance for a long-term cure.

Before undergoing a bone marrow transplant, the patient will typically receive conditioning therapy, which may involve high-dose chemotherapy or radiation therapy. This process will destroy the damaged bone marrow and weaken the immune system, allowing the transplanted cells to engraft more effectively.

Once conditioning therapy is complete, healthy blood-forming stem cells will be infused into the patient’s bloodstream. The transplanted cells will then travel to the bone marrow, where they will begin producing new red blood cells, white blood cells and platelets.

BMT may be a good option for a relatively young patient with a compatible donor, such as a sibling or a well-matched unrelated donor. If the patient is older or does not have a suitable match, alternative treatments such as immunosuppressive therapy may be considered instead.

Although BMT carries certain risks, including infection, graft-versus-host disease (GVHD) and transplant rejection, it remains the only treatment option with the potential to completely restore bone marrow function in a severe case of aplastic anemia.

How is surgery used to treat aplastic anemia?

Surgery is not a standard treatment for aplastic anemia, mainly because the condition affects blood cell production within the bone marrow rather than a localized area that can be addressed surgically.

Benefit from world-class care at Moffitt Cancer Center

If you have questions about aplastic anemia, you can request an appointment with a specialist in the Malignant Hematology Program at Moffitt by calling 1-888-663-3488  or completing our new patient registration form online. We do not require referrals.