Aplastic Anemia
Aplastic anemia is a condition that happens when your bone marrow stops making enough new blood cells. The condition leaves you tired and more prone to infections and uncontrolled bleeding.
A rare and serious condition, aplastic anemia can develop at any age. It can happen suddenly, or it can come on slowly and worsen over time. It can be mild or severe.
Treatment for aplastic anemia might include medicines, blood transfusions or a stem cell transplant, also known as a bone marrow transplant.
Aplastic anemia can have no symptoms. When present, symptoms can include:
- Fatigue.
- Shortness of breath.
- Rapid or irregular heart rate.
- Frequent or prolonged infections.
- Unexplained or easy bruising.
- Nosebleeds and bleeding gums.
- Prolonged bleeding from cuts.
- Pale skin.
- Skin rash.
- Fever.
Aplastic anemia can be short-lived, or it can become chronic. It can be serious and even fatal.
Bone marrow
Bone marrow is a red, spongy material inside your bones that produces blood cells.
Bone marrow is a red, spongy material inside your bones. Stem cells in the bone marrow make blood cells — red cells, white cells and platelets. In aplastic anemia, stem cells are damaged. As a result, the bone marrow makes fewer new blood cells.
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:
- Radiation and chemotherapy treatments. While these cancer-fighting therapies kill cancer cells, they can also damage healthy cells, including stem cells in bone marrow. Aplastic anemia can be a temporary side effect of these treatments.
- Exposure to toxic chemicals. Toxic chemicals, such as some used in pesticides and insecticides, and benzene, an ingredient in gasoline, have been linked to aplastic anemia. This type of anemia might improve if you avoid repeated exposure to the chemicals that caused your illness.
- Use of certain drugs. Some medicines, such as those used to treat rheumatoid arthritis and some antibiotics, can cause aplastic anemia.
- Autoimmune disorders. An autoimmune disorder, in which your immune system attacks healthy cells, might involve stem cells in your bone marrow.
- A viral infection. Viral infections that affect bone marrow can play a role in the development of aplastic anemia. Viruses that have been linked to aplastic anemia include hepatitis, Epstein-Barr, cytomegalovirus, parvovirus B19 and HIV.
- Pregnancy. Your immune system might attack your bone marrow during pregnancy.
- Unknown factors. In many cases, doctors aren’t able to identify the cause of aplastic anemia. This is called idiopathic aplastic anemia.
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.
Aplastic anemia is rare. Factors that can increase risk include:
- Treatment with high-dose radiation or chemotherapy for cancer.
- Exposure to toxic chemicals.
- The use of some prescription drugs — such as chloramphenicol, which is used to treat bacterial infections, and gold compounds used to treat rheumatoid arthritis.
- Certain blood diseases, autoimmune disorders and serious infections.
- Pregnancy, rarely.
There’s no known 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.
The following tests can help diagnose aplastic anemia:
- Blood tests. Normally, red blood cell, white blood cell and platelet levels stay within certain ranges. In aplastic anemia, all three of these blood cell levels are low.
- Bone marrow biopsy. A needle is used to remove a small sample of bone marrow from a large bone, such as your hipbone. The sample is examined under a microscope to rule out other blood-related diseases. In aplastic anemia, bone marrow contains fewer blood cells than normal. Confirming a diagnosis of aplastic anemia requires a bone marrow biopsy.
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 healthcare professional uses a thin needle to remove a small amount of liquid bone marrow. The marrow often is taken from a spot in the back of the hipbone. A bone marrow biopsy removes a small piece of bone tissue and its marrow. The procedures often are done at the same time.
Once you’ve received a diagnosis of aplastic anemia, you might need other tests to determine the cause.
Treatments for aplastic anemia depend on the symptoms and your age and overall health. Treatment options might include observation, blood transfusions, medicines 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 provide blood cells your bone marrow isn’t producing. This can help control bleeding and relieve symptoms. You might receive:
- Red blood cells. These raise red blood cell counts and help relieve anemia and fatigue.
- Platelets. These help prevent excessive bleeding.
While there’s generally no limit to the number of blood transfusions you can have, complications sometimes arise with multiple transfusions. Transfused red blood cells contain iron that can accumulate in your body and damage vital organs if an iron overload isn’t treated. Medicines 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 medicine 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 through a vein into your bloodstream. The healthy cells 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, called immunosuppressants.
Drugs such as cyclosporine (Neoral, Sandimmune) and antithymocyte 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 antithymocyte globulin are often used together.
Corticosteroids, such as methylprednisolone (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
Your hematologist may prescribe medicines to stimulate your bone marrow to make new blood cells. These drugs are called growth factors or colony-stimulating factors. Each drug stimulates production of a different type of blood cell. Sargramostim (Leukine), filgrastim (Neupogen), pegfilgrastim (Neulasta), epoetin alfa (Epogen/Procrit) and eltrombopag (Promacta) help stimulate the bone marrow to produce new red blood cells, white blood cells and platelets. 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 healthcare professional 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 medicines 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 can cause 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.
References
- Ferri FF. Anemia, aplastic. In: Ferri’s Clinical Advisor 2020. Elsevier; 2020. https://www.clinicalkey.com. Accessed Aug. 4, 2023.
- AskMayoExpert. Aplastic anemia (adult). Mayo Clinic; 2023.
- Hoffman R, et al. Aplastic anemia. In: Hematology: Basic Principles and Practice. 8th ed. Elsevier; 2023. https://www.clinicalkey.com. Accessed Aug. 4, 2023.
- Olson TS. Aplastic anemia: Pathogenesis; clinical manifestations; and diagnosis. https://www.uptodate.com/contents/search. Accessed Aug. 4, 2023.
- Aplastic anemia. National Heart, Lung, and Blood Institute. https://www.nhlbi.nih.gov/health-topics/aplastic-anemia. Accessed Aug. 4, 2023.
- Olson TS. Treatment of aplastic anemia in adults. https://www.uptodate.com/contents/search. Accessed Aug. 4, 2023.
- Aplastic anemia. Aplastic Anemia and MDS International Foundation. https://www.aamds.org/diseases/aplastic-anemia. Accessed Aug. 4, 2023.
- Kaushansky K, et al., eds. Aplastic anemia: Acquired and inherited. In: Williams Hematology. 10th ed. McGraw Hill; 2021. https://accessmedicine.mhmedical.com. Accessed Aug. 4, 2023.
- Larson RA. Use of granulocyte colony stimulating factors in adult patients with chemotherapy-induced neutropenia and condition other than acute leukemia, myelodysplastic syndrome, and hematopoietic cell transplantation. https://www.uptodate.com/contents/search. Accessed Aug. 8, 2023.
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