Aplastic Anemia

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The blood disorders and bone marrow transplantation teams at UT Southwestern excel at diagnosing and treating children and adults with aplastic anemia and advancing care through clinical research.

Our highly specialized program has been named a national center of excellence by all major national health insurance carriers and is accredited by the Foundation for the Accreditation of Cellular Therapy, which recognizes top-quality patient care.

Expert Diagnosis and Individualized Care for Children and Adults with Aplastic Anemia

Aplastic anemia is a rare and serious blood disorder that occurs when there’s a problem with the bone marrow, which is the tissue inside bones that produces blood-forming cells.

People with aplastic anemia have low levels of:

  • Red blood cells, which move oxygen throughout the body
  • White blood cells, which help fend off infection and disease
  • Platelets, which affect the blood clotting process

Aplastic anemia is usually acquired, but it can also be inherited, meaning it’s present at birth. People may have mild, severe, or very severe forms of the disorder. People with severe forms may need aggressive treatment.

At UT Southwestern, we offer the full spectrum of diagnostic tools and treatments for aplastic anemia. Bone marrow transplantation often provides a cure for the disorder, and our providers are leaders in this medical treatment.

Part of the Harold C. Simmons Comprehensive Cancer Center, UT Southwestern’s Blood Cancers/Transplant and Cellular Therapies team has the leading survival rate for adult allogeneic stem cell (bone marrow) transplants in Texas. Allogeneic means the transplanted stem cells come from a donor.

Through our clinical research, we are advancing understanding of aplastic anemia and care for children and adults with the disorder.

Types of Aplastic Anemia

The two types of aplastic anemia are:

  • Acquired aplastic anemia, which is the more common type and arises when the immune system is not working properly
  • Inherited aplastic anemia, which is caused by a gene mutation

Causes of Aplastic Anemia

Aplastic anemia occurs when the bone marrow fails to produce an adequate number of new blood cells. Most of the time, this problem occurs because the immune system attacks bone marrow stem cells.

There are numerous reasons why this might happen. In many cases, an exact cause can’t be determined.

Risk factors for aplastic anemia include:

  • Autoimmune disorders, such as lupus or eosinophilic fasciitis
  • Having family members who have bone marrow failure syndrome
  • Serious infections, such as HIV, hepatitis, or Epstein-Barr virus
  • Undergoing radiation therapy or chemotherapy
  • Using certain medications, including chloramphenicol (for bacterial infections) and gold compounds (for rheumatoid arthritis)
  • Exposure to toxic chemicals and substances, such as arsenic, benzene, and pesticides
  • Pregnancy

People with aplastic anemia may develop another rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH) or vice versa. People with Fanconi anemia often develop aplastic anemia.

Symptoms of Aplastic Anemia

Some people with aplastic anemia have mild or no symptoms. Others have severe symptoms and need immediate care.

Typical symptoms include:

  • Easy bruising or bleeding
  • Heavy menstrual bleeding
  • Shortness of breath
  • Feeling very tired
  • Pale skin
  • Skin rash
  • Repeated or long-lasting infections
  • Fever
  • Dizziness
  • Headache
  • Rapid or irregular heartbeat

Diagnosing Aplastic Anemia

At UT Southwestern, our doctors check for aplastic anemia by examining the patient, reviewing the patient’s medical history, and ordering blood tests to check levels of red and white blood cells and platelets.

We will likely do a bone marrow aspiration and biopsy to rule out other possible causes of low blood cell levels and to confirm an aplastic anemia diagnosis. This test involves taking a bone marrow sample (often from the hipbone) with a thin needle and looking at it under a microscope.

People who are diagnosed with aplastic anemia may have additional tests to determine what’s causing the disorder.

Aplastic anemia has three classifications: moderate, severe, and very severe.

Aplastic Anemia Treatment

At UT Southwestern, we provide comprehensive care for people with aplastic anemia, as well as education on how to prevent complications. Our doctors will determine the appropriate course of treatment, depending on the severity of the disorder, the patient’s age, and the cause of the disorder, if known.

In mild cases, careful observation might be prescribed. Severe aplastic anemia, which is characterized by very low blood cell counts, needs immediate care.

A bone marrow transplant (also known as a stem cell transplant) may provide a cure for aplastic anemia.

Other treatments for aplastic anemia include:

  • Blood transfusions, which replenish red blood cells and platelets. This treatment helps relieve symptoms such as anemia, fatigue, and bleeding problems, but it is not a cure.
  • Immunosuppressants, which stop the immune system from attacking bone marrow stem cells, allowing them to make new blood cells. These medications might be an option for people who can’t have a bone marrow transplant or who developed aplastic anemia due to an autoimmune disorder. While this treatment can be effective, it is not a cure.
  • Bone marrow stimulants, which are frequently used along with immunosuppressants and help the bone marrow make new blood cells. While these medications often improve symptoms, they do not provide a cure.

If the cause of aplastic anemia is known, some people might improve by avoiding that known factor. For example, people who develop aplastic anemia during radiation therapy or chemotherapy or while using certain drugs typically get better after stopping those treatments or drugs. People who develop aplastic anemia during pregnancy will need blood transfusions during pregnancy, but the disorder often goes away when the patient is no longer pregnant.

People with aplastic anemia might also need supportive care, such as antibiotics or antiviral or antifungal drugs to treat infections and placement of a central venous catheter for delivering infused medicines.

In addition, people with aplastic anemia might avoid complications by:

  • Reducing exposure to germs by keeping hands clean, staying away from people who are sick, wearing a mask, and getting a flu shot and other vaccines as recommended
  • Taking time to rest when needed
  • Not playing contact sports

Support Services for Aplastic Anemia

At UT Southwestern, we deliver comprehensive care that extends outside our clinic. We can help you coordinate with social workers and financial counselors. When needed, we can refer you to experts in infectious disease, nephrology, and hepatology.

We also offer a range of supportive services, including support groups, for people who have had bone marrow transplants.