Hematologic Disorders: Aplastic Anemia
Alternate Names: anemia-secondary aplastic, acquired aplastic anemia
What is aplastic anemia?
In aplastic anemia, the bone marrow’s stem cells do not make enough new blood cells. Most often, all three types of blood cells are affected:
- Red blood cells, which carry oxygen
- White blood cells, which fight infection
- Platelets, which help the blood to clot and stop bleeding
Rarely, just one of the cell types is affected.
Although aplastic anemia affects how many blood cells the body produces, the few that do mature and enter the bloodstream are normal.
Aplastic anemia is associated with some cancers and cancer treatments, but it is not considered a type of cancer.
How common is aplastic anemia?
Aplastic anemia is a rare disease that occurs in only one to two people per million each year. That’s 300 to 600 new cases across all age groups in the U.S.
Risk of aplastic anemia is higher among those who are:
- Exposed to toxic chemicals such as pesticides, arsenic and benzene
- Treated for cancer using radiation and chemotherapy
- Taking certain medicines such as chloramphenicol, used to treat bacterial infections, or gold compounds, used to treat rheumatoid arthritis
- Diagnosed with infections such as hepatitis, Epstein-Barr virus or human immunodeficiency virus (HIV)
- Diagnosed with certain inherited conditions including Fanconi anemia, Shwachman-Diamond syndrome, dyskeratosis congenita and Diamond-Blackfan anemia
What are the symptoms of aplastic anemia?
Symptoms of aplastic anemia include:
- Feeling tired
- Shortness of breath
- Rapid or irregular heartbeat
- Pale skin
- Frequent fevers
- Frequent infections that last a long time
- Unexplained or easy bruising
- Nosebleeds and bleeding gums
- Bleeding that isn’t easily stopped, even when the cut is small
- Skin rash
- Feeling dizzy
How is aplastic anemia treated?
Treatment for aplastic anemia may include blood transfusion, stem cell/bone marrow transplant and medicines to treat the associated cancer and the side effects of those medicines.
- Blood transfusion - helps keep blood cell counts at desired levels. Blood matched with your child’s blood is given through a tube inserted into a vein. The transfused cells replace diseased ones but do not help make new, healthy cells. Transfusions can relieve symptoms but do not treat causes of the disease.
Man-made versions of substances occurring naturally in the body may be used to stimulate the bone marrow to make more blood cells. If this treatment works, blood transfusion may not be needed.
- Stem cell / bone marrow transplant - replaces damaged blood-forming stem cells with normal cells from a donor. Given through a tube inserted into a chest vein, the stem cells travel to the bone marrow and begin making new, healthy blood cells. This treatment works best in children and young adults with severe aplastic anemia who are in good health and have blood-matched donors.
- Medicines - may be used for different reasons according to your child’s specific needs. For example:
- To suppress the body’s immune system when it is attacking its own cells by mistake. The medicine restores the bone marrow’s ability to make blood cells, so blood transfusions may not be needed. These drugs only treat symptoms; they cannot cure aplastic anemia.
- To treat or reduce the risk of infection due to low white blood cell counts. Antibiotic and antiviral medicines that kill bacteria, viruses and other germs may be used.
What are the survival rates for aplastic anemia?
Based on cancer registry data, the overall five-year survival rate is about 80% for patients younger than age 20 who have a stem cell / bone marrow transplant.
Why choose St. Jude for your child’s aplastic anemia treatment?
- St. Jude is the only National Cancer Institute-designated Comprehensive Cancer Center devoted solely to children.
- The nurse-to-patient ratio at St. Jude is unmatched— averaging 1:3 in hematology and oncology, and 1:1 in the Intensive Care Unit.
- In a recent study, the event-free survival rate was 100% in children who received a transplant at St. Jude.
- St. Jude is preparing for a study for patients without a matched donor and in whom drug therapy was only partially successful. Investigators are planning to use a therapy that modifies the immune system in a different way than drug therapy. This therapy is expected to interrupt the disease process and allow the bone marrow to recover and produce blood cells again.
- St. Jude is also developing a new stem cell/bone marrow transplant regimen for patients who do not have a matched sibling donor and who do not respond to the drug therapy.
- St. Jude has one of the world’s largest hematopoietic stem cell transplant programs dedicated to caring for children, teens and young adults. Since the first hematopoietic stem cell transplant at St. Jude was performed in 1982, the Transplant Program has performed more than 2,500 transplants.
- St. Jude is accredited by the Foundation for the Accreditation of Cellular Therapy (FACT) for autologous and allogeneic peripheral blood and marrow transplantation in children and adults.
- The Transplant Program at St. Jude has been a part of the National Marrow Donor Program (NMDP) since 1990. Our center also provides data to the International Bone Marrow Transplant Registry and Autologous Bone Marrow Transplant Registry.
- St. Jude was the first institution to perform an allogeneic stem cell / bone marrow transplant in a patient with sickle cell anemia.
- To improve outcomes and reduce side effects associated with transplant, doctors in the St. Jude Transplant Program work closely with laboratory scientists to rapidly move discoveries from the lab to the clinic.
- St. Jude support staff members have been specially trained to care for children receiving hematopoietic stem cell transplants.
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