How Is Aplastic Anemia Treated?
Treatments for aplastic anemia include:
- Blood transfusions
- Bone marrow transplants
- Several medicines used to suppress the immune
system, stimulate the bone marrow, and prevent and treat infections
In rare cases, no treatment is needed.
In general, treatments for aplastic anemia can limit
and prevent complications, relieve symptoms, and improve quality of life. For
some, a cure may be possible. Bone marrow transplants can be a cure for those
people who are eligible for a transplant. Removing a known cause of aplastic
anemia (such as exposure to a toxic chemical) also can cure the disorder. Other
treatments can restore blood cell counts to levels high enough so that a person
can live a normal life.
Who Needs Treatment
People with mild or moderate aplastic anemia may not
need treatment as long as the condition does not get worse. People with severe
aplastic anemia need immediate medical treatment to prevent complications from
the very low levels of blood cells in their bodies. People with very severe
aplastic anemia need emergency medical care and hospitalization or the anemia
can be fatal in a short time.
Blood Transfusions
Blood transfusions are used to keep blood counts
high enough in people with aplastic anemia. Healthy blood is taken from matched
donors and then given to the person with aplastic anemia through an injection
in a vein. Blood transfusions help relieve symptoms. They are not an effective
long-term treatment.
Red Blood Cells
Red blood cell transfusions help relieve lack of
energy, tiredness, and shortness of breath. However, if red blood cells are
given often, the immune system in the body can learn to recognize these new
cells, develop antibodies against them, and destroy these newly transfused
cells. Also, if red blood cells are given often, the body can build up too much
iron. Excess iron from transfusions can damage the heart, liver, and other
organs. If this happens, treatment may be needed to remove excess iron from the
body.
White Blood Cells
White blood cells are not routinely transfused
because they live in the bloodstream for less than 1 day. However, they may be
used for severe infections that are not being helped by antibiotics.
Platelets
Platelet transfusions reduce the risk of fatal
bleeding. Several transfusions a month are often needed because platelets live
only a few days. However, in time, the immune system will learn to recognize
and destroy newly transfused platelets by making antibodies, just as it does
with red blood cells.
Bone Marrow Transplantation
A bone marrow transplant replaces damaged stem cells
in bone marrow with healthy stem cells from a donors bone marrow. It is
the best treatment choice for eligible people because it usually cures aplastic
anemia.
A bone marrow transplant works best in children and
young adults with severe aplastic anemia who are in good health and who have
matched donors. Older people are less able to tolerate the treatments needed to
prepare the body for the transplant. They also are more likely to have
complications after the transplant. There are age limits for who can receive a
bone marrow transplant, but they vary among hospitals.
Finding a Donor
For best results, the donors bone marrow must
be a close match with the bone marrow of the person with aplastic anemia. A
matched donor is found by means of a blood test that looks at proteins on the
surface of body cells. These proteins are called human leukocyte antigens
(HLA). The donor and the person with aplastic anemia are both tested. The HLA
antigens on their cells are compared.
Bone marrow transplants work best if the donated
marrow is an exact match and comes from a family member, such as a brother or
sister. A person receiving bone marrow that is an exact match usually has fewer
complications. Only about 2035 percent of people with severe aplastic
anemia have an HLA-matched family member to donate bone marrow.
If no matching family member is found, the search
for bone marrow widens. Millions of volunteer donors are registered with the
National Marrow Donor Program. Doctors will look for:
- Donors who are HLA matches but are not family
members
- Family members who are not exact HLA matches
- Unrelated donors who are not exact HLA
matches
- Umbilical cord blood that is an HLA match
A person who is going to have a bone marrow
transplant should not receive blood transfusions from close family members
before the transplant. When a person receives blood transfusions, their bodies
can develop antibodies against the donated blood. If a person develops
antibodies against the blood of a close family member, the person would not be
able to receive a bone marrow transplant from that family member.
Receiving the Transplant
Bone marrow transplants are done in special units in
hospitals to try to protect the person with aplastic anemia from any
infections. Before the transplant, the person receives high doses of
chemotherapy, radiation therapy, or both. These treatments destroy the abnormal
bone marrow that is not producing blood cells.
A sample of bone marrow is taken from the donor and
is given to the recipient through a vein. The donated bone marrow stem cells
travel to the recipients bone marrow and start to make healthy blood
cells. If the transplant is successful, the new stem cells multiply and begin
making new, healthy blood cells within 3 to 4 weeks.
Possible Complications
After a bone marrow transplant, the recipient is in
the hospital for weeks or months. During this time, he or she is isolated and
closely watched for infections and other complications. Complications are most
likely to occur in the first 100 days after a transplant. Complications include
rejection of the graft and what is called graft-versus-host disease (GVHD).
Graft rejection. Sometimes, the
recipients immune system destroys the new bone marrow cells. This occurs
in 5 to 10 percent of recipients, and it is more likely to happen in people who
have had several bone marrow transplants.
GVHD. GVHD happens when the new
immune system cells, created by the donated bone marrow, attack the
recipients body. Signs and symptoms are skin inflammation, diarrhea, and
liver disease. GVHD can occur soon after transplant, or it can develop slowly
and can last for months or years. GVHD most often happens in older people and
those who receive unmatched transplants.
To help prevent GVHD, donor marrow is sometimes
treated or people may be given medicines that suppress the immune system after
the transplant. Those who develop mild but acute GVHD are treated with
medicines such as steroids.
Medicines To Stimulate Bone Marrow
Manmade versions of substances that occur naturally
in the body are used to stimulate the bone marrow to produce more blood cells.
Bone marrow that can produce more blood cells can help a person with aplastic
anemia avoid blood transfusions. An increased number of white cells can help
protect a person from infection. Examples of bone marrow stimulation medicines
used to treat aplastic anemia are:
- Erythropoietin (e-RITH-ro-PO-e-tin)
- Colony-stimulating factors
These medicines have some risks. Based on your
situation, you doctor will decide whether the benefits of the medicines
outweigh the risks.
Medicines To Suppress the Immune System
Since research suggests that aplastic anemia may
occur because the bodys immune system attacks its own cells by mistake,
certain medicines may be used to suppress the bodys immune system. They
allow the bone marrow to start making blood cells again, and they help avoid
the need for blood transfusions. Medicines that suppress the immune system do
not cure aplastic anemia, but they can relieve its symptoms and reduce its
complications.
These medicines are often used for people who are
not good candidates for a bone marrow transplant or who are waiting for a bone
marrow transplant.
There are three medicinesoften given
togetherthat will suppress the bodys immune system:
- Antithymocyte globulin (ATG)
- Cyclosporine
- Methylprednisolone (a corticosteroid)
If this treatment is successful, it may take a few
months to notice the effects of these medicines. Most often, as blood counts
rise, symptoms lessen. Blood counts in people who respond well to these drugs
do not usually reach normal levels, but they are often high enough to allow
people to take part in their normal activities. People with aplastic anemia may
need to take the medicines indefinitely to keep their blood counts high enough
to resume normal daily life.
Medicines that suppress the immune system can have
side effects. They also may increase the risk of developing leukemia
(loo-KE-me-a) or myelodysplasia (MI-e-lo-dis-PLA-ze-a; MDS). Leukemia is a type
of cancer in which the number of white blood cells increases. MDS is a
condition in which the bone marrow produces too many defective blood cells.
Antibiotics
Antibiotics and antivirals are given to people with
aplastic anemia to prevent and treat infections. |