Aplastic anemia is a
condition that occurs when your body stops producing enough new blood
cells. Aplastic anemia leaves you feeling fatigued and at higher risk of
infections and uncontrolled bleeding.
A rare and serious condition, aplastic anemia can develop at any age,
though it's most common in younger people. Treatment for aplastic anemia
may include medications, blood transfusions or a bone marrow
transplant. Once considered nearly always fatal, aplastic anemia has a
much better prognosis today, thanks to advances in treatment.
Aplastic anemia symptoms result from a shortage of one or more types of blood cells. Signs and symptoms may include:
* Shortness of breath with exertion
* Rapid or irregular heart rate
* Pale skin
* Frequent or prolonged infections
* Unexplained or easy bruising
* Nosebleeds and bleeding gums
* Prolonged bleeding from cuts
* Skin rash
Aplastic anemia can progress slowly over weeks or months, or it may come
on suddenly. The illness may be brief, or it may become chronic.
Without treatment, it may progress and become fatal.
Aplastic anemia develops when damage occurs to your bone marrow, slowing
or shutting down the production of new blood cells. Bone marrow is a
red, spongy material found within the cavities of many of your bones.
Bone marrow produces stem cells, which give rise to other cells. Stem
cells in the bone marrow produce blood cells — red cells, white cells
and platelets. Stem cells also make more stem cells.
Normally, your body continually replaces blood cells. Red blood cells
live about 120 days, platelets about seven days and most white blood
cells a day or less before they're used and absorbed by your body.
Factors that can temporarily or permanently injure bone marrow and affect blood cell production include:
* High-dose 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. Exposure to toxic chemicals, such as
some used in pesticides and insecticides, may cause aplastic anemia.
Exposure to benzene — an ingredient in gasoline — also has been linked
to aplastic anemia. This type of anemia sometimes gets better on its own
if you avoid repeated exposure to the chemicals that caused your
* Use of certain drugs. Some medications, such as those used to
treat rheumatoid arthritis and some antibiotics, can cause aplastic
* Autoimmune disorders. An autoimmune disorder, such as lupus, in
which your immune system begins attacking healthy cells, may involve
stem cells in your bone marrow.
* A viral infection. Viral infections that affect bone marrow may
play a role in the development of aplastic anemia in some people.
Viruses that have been linked to the development of aplastic anemia
include hepatitis, Epstein-Barr, cytomegalovirus, parvovirus B-19 and
* Pregnancy. Aplastic anemia that occurs in pregnancy may be related
to an autoimmune problem — your immune system may attack your bone
marrow during pregnancy.
* Unknown factors. In about half the cases, doctors aren't able to
identify the cause of aplastic anemia. This is called idiopathic
In aplastic anemia, the bone marrow is described in medical terms as
aplastic or hypoplastic — meaning that it's empty, or contains very few
Confusion with myelodysplastic syndrome
Aplastic anemia can be mistaken for a condition called myelodysplastic
syndrome. In this group of disorders, the bone marrow produces new blood
cells, but they're deformed and underdeveloped. The bone marrow in
myelodysplastic syndrome is sometimes called hyperplastic — meaning that
it's packed with blood cells. But some people with myelodysplastic
syndrome have empty marrow that's difficult to distinguish from aplastic
Connections with other rare disorders
Additionally, about 30 percent of people with aplastic anemia also have a
rare disorder known as paroxysmal nocturnal hemoglobinuria. Marrow
cells become overly sensitive to the immune system in this disorder,
destroying red blood cells and causing defective platelets to form.
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 may increase your risk include:
* Treatment with high-dose radiation or chemotherapy for cancer
* Exposure to toxic chemicals
* Use of some prescription drugs — such as chloramphenicol, which is
used to treat bacterial infections, and gold compounds used to treat
rheumatoid arthritis — that are known to rarely induce aplastic anemia
* Certain blood diseases, autoimmune disorders and serious infections
* Pregnancy, rarely
Preparing for your appointment
You may start by first seeing your family doctor or a general
practitioner, but you'll likely be quickly referred to a doctor who
specializes in treating blood disorders (hematologist). If aplastic
anemia comes on suddenly, you may begin treatment in the emergency room.
Here's some information to help you get ready for your appointment, and what to expect from your doctor.
What you can do
* Write down any symptoms you're experiencing, including any that
may seem unrelated to the reason for which you scheduled the
* Write down key personal information, including any recent life
changes, such as a new job, particularly one that exposes you to
* Make a list of all medications, as well as any vitamins or supplements, that you're taking.
* Ask a family member or a friend to stay with you while you talk to
your doctor. You may be tired or overwhelmed by all the information. A
friend or a family member can take notes for you, or bring up questions
you may forget to ask.
* Write down questions you want to ask your doctor.
Preparing a list of questions ahead of time can help you make the most
of your time together. For aplastic anemia, some basic questions to ask
your doctor include:
* What's the most likely cause of my symptoms?
* Are there other possible causes for my symptoms?
* What's my prognosis?
* What treatments are available, and which do you recommend?
* Are there any alternatives to the primary approach that you're suggesting?
* I have another health condition. How can I best manage them together?
* Are there any brochures or other printed material that I can take with me? What Web sites do you recommend?
In addition to the questions that you've prepared to ask your doctor,
don't hesitate to ask questions during your appointment any time that
you don't understand something.
What to expect from your doctor
Your doctor is likely to ask you a number of questions, such as:
* Have you had any infections?
* Have you had any unexpected bleeding?
* Have you felt more tired than usual?
* When did you first begin experiencing symptoms?
* Is there anything new in your life, such as a new job?
* Does anything seem to improve your symptoms?
* Does anything appear to worsen your symptoms?
Tests and diagnosis
Doctors diagnose aplastic anemia using blood tests and bone marrow biopsy.
* Blood tests. Normally, red blood cell, white blood cell and
platelet levels stay within a certain range. Your doctor may suspect
aplastic anemia when all three of these blood cell levels are very low.
Many conditions can cause low blood cell counts, but usually of just one
type of blood cell. For example, other types of anemia cause a decrease
in red blood cells. If you have an infection, your white blood cell
count alone may be low.
* Bone marrow biopsy. To confirm a diagnosis, you'll need to undergo
a bone marrow biopsy. In this procedure, a doctor uses a needle to
remove a small sample of bone marrow from a large bone in your body,
such as your hipbone. The bone marrow sample is examined under a
microscope to rule out other blood-related diseases. In aplastic anemia,
bone marrow contains fewer blood cells than normal. The few cells that
are present, however, are normal. In certain diseases such as leukemia
and myelodysplastic syndrome, the bone marrow is full of abnormal blood
Once you've received a diagnosis of aplastic anemia, you may need additional tests to determine an underlying cause.
Treatments and drugs
Aplastic anemia can be mild, moderate or severe. Severe aplastic anemia,
in which your blood cell counts are extremely low, is life-threatening
and requires immediate hospitalization for treatment. Mild or moderate
aplastic anemia is still serious, but usually doesn't require
hospitalization to treat.
Treatments for aplastic anemia may include observation for mild cases,
blood transfusions, medications and, in severe cases, bone marrow
Most people with aplastic anemia require multiple blood transfusions —
transfusions of red blood cells or platelets, or both. Blood
transfusions aren't a cure for aplastic anemia. But they do relieve
signs and symptoms by providing blood cells that your bone marrow isn't
* Red blood cells. Transfusions of red blood cells raise red blood
cell counts. This helps relieve anemia and fatigue. Red blood cells are
easiest to replace by transfusion.
* Platelets. Transfusions of platelets help prevent excessive bleeding.
* White blood cells. Because they're so short-lived, white blood cells can't be easily transfused.
While there's generally no limit to the number of blood cell
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. Another possible complication is that over time, your
body may develop antibodies to transfused blood cells, making them less
effective at relieving symptoms.
Bone marrow transplantation
Bone marrow transplantation — replacing diseased bone marrow with
healthy bone marrow from a donor — may offer the only successful
treatment option for people with severe aplastic anemia. This is
generally the treatment of choice for people who are younger and have a
matching sibling donor.
Bone marrow transplantation from a related, matched donor can treat
aplastic anemia — without recurrence — in about four out of five cases.
Up to about half the people with matched, but unrelated, donors can
expect to have successful bone marrow transplants.
This procedure carries risk. There's a chance that 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
If a donor is found, the diseased bone marrow in the person with
aplastic anemia is first depleted with radiation or chemotherapy.
Healthy bone marrow from the donor is extracted through a surgical
technique. The healthy marrow is injected intravenously into the
bloodstream of the person with aplastic anemia, where it migrates to the
bone marrow cavities and may begin generating new blood cells in about
three to four weeks. The procedure requires a lengthy hospital stay.
After the transplant, you'll receive drugs to help prevent rejection of
the donated marrow.
If no suitable bone marrow donor is available, treatment of severe
aplastic anemia is with immune-suppressing drugs. If you're a candidate
for bone marrow transplantation, your doctor may limit the number of
blood transfusions you have before the procedure because they can
increase the risks associated with bone marrow transplantation.
Aplastic anemia may be due to an autoimmune disorder that's causing your
immune system to attack and damage cells in your bone marrow. To
prevent this from continuing, doctors may treat aplastic anemia with
drugs that alter or suppress your immune system.
Drugs such as cyclosporine (Gengraf, Neoral, Sandimmune) and
anti-thymocyte globulin (Thymoglobulin) are examples. These drugs
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 in
combination. This option is usually the treatment of choice for older
people with aplastic anemia and for those without a matching donor for
bone marrow transplant or those with additional health conditions.
Corticosteroids, such as methylprednisolone (Medrol, Solu-Medrol), are often given at the same time as these drugs.
Immune-suppressing drugs can be very effective at treating aplastic
anemia. The downside is that these drugs further weaken your immune
system. It's also possible that after you stop taking these drugs,
aplastic anemia may return.
Bone marrow stimulants
Certain drugs — including granulocyte-macrophage colony-stimulating
factor, such as sargramostim (Leukine); granulocyte colony-stimulating
factor, such as filgrastim (Neupogen); and epoetin alfa (Epogen,
Procrit) — may help stimulate the bone marrow to produce new blood
cells. Growth factors aren't considered effective on their own, but may
be beneficial when used in combination with immune-suppressing drugs.
Having aplastic anemia weakens your immune system. You have fewer white
blood cells in circulation to fight off germs. This leaves you
susceptible to all kinds of infections — everything from colds to
At the first sign of infection, such as a fever, see your doctor. You
don't want the infection to become severe, because it could prove
life-threatening. If you have severe aplastic anemia, your doctor may
give you antibiotics or antiviral medications to help prevent
Aplastic anemia caused by radiation and chemotherapy treatments for
cancer usually improves once you complete those treatments. The same is
true for most other drugs that induce aplastic anemia. You usually can
expect a full recovery once you stop taking the drug.
Some cases of pregnancy-related aplastic anemia improve once the
pregnancy ends. If that doesn't happen, treatment is still necessary.
Some women who have aplastic anemia that responds to immune-suppressing
drugs before pregnancy may find the disorder returns when they stop
taking the drugs — for safety reasons — during pregnancy.
If all treatments fail, aplastic anemia can be fatal. Ask your doctor
about what experimental treatments may be available if standard
treatments aren't working.
Lifestyle and home remedies
If you have aplastic anemia, it's important to:
* Avoid excessive exercise. Anemia can cause fatigue and shortness
of breath with even mild exertion. It may be best to conserve your
* Avoid contact sports. Because of the risk of bleeding associated
with a low platelet count, avoid activities that may result in a major
cut or fall.
* Avoid infections. Protect yourself from infections by frequent
hand washing and by avoiding sick people. If you develop a fever or
other indicators of an infection, see your doctor for treatment.
Herbal treatments and vitamins aren't effective treatments for aplastic
anemia and may actually worsen the condition. Talk to your doctor before
trying any such therapy.
Coping and support
As with any serious and chronic disease, you may experience many
emotional side effects from living with aplastic anemia. Tips to help
you and your family better cope with your illness include:
* Research your disease. The more you know, the better prepared you'll be to make treatment decisions.
* Ask questions. Be sure to ask your doctor about anything related
to your disease or treatment that you don't understand. It may help you
to record or write down what your doctor tells you.
* Be vocal. Don't be afraid to express any concerns you have to your
doctor or any other health care professional treating you.
* Seek support. Ask family and friends for emotional support. Ask
them to consider becoming blood donors or bone marrow donors. Consider
joining an aplastic anemia support group. It may be helpful to talk to
others coping with the disease. Ask your doctor if he or she knows of
any local support groups, or contact the Aplastic Anemia & MDS
International Foundation. They offer a peer support network and can be
reached at 800-747-2820.
* Take care of yourself. Proper nutrition and sleep are important to optimize blood production.
There's no prevention for most cases of aplastic anemia. However,
avoiding exposure to insecticides, herbicides, organic solvents, paint
removers and other toxic chemicals may lower your risk of the disease.
This is especially important if you've already had aplastic anemia that
was caused by toxic chemicals. Exposure to the same compound a second
time may cause the condition to return.