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Rare Diseases: Aplastic Anemia
Alternative Names: None
Definition
Aplastic anemia is a rare, non-contagious and often life-threatening
disorder that results from the unexplained failure of the bone marrow to
produce red blood cells, white blood cells and platelets. A decrease in the
production of blood cells means that patients are more susceptible to
bleeding, fatigue and infections.
Incidence
Aplastic anemia affects two to six people per million worldwide. The disease
is more prevalent in Asia than in the Western world. Most cases occur in
older adults, but a significant number of children are affected.
Influencing Factors
a.. The cause of the disease in an individual patient is usually unknown,
although the disease has been linked to environmental toxins such as benzene
derivatives, some drugs, pesticides and chemicals.
b.. Aplastic anemia can be caused by a virus in some individuals.
c.. There is evidence that parts of the immune system play a role in the
disease process.
Treatment Strategies
a.. Patients with severe aplastic anemia require immediate hospital
treatment.
b.. Initial treatment often includes blood and platelet transfusions and
intravenous antibiotics.
c.. Bone marrow transplantation is recommended for patients who are good
candidates and who have matched donors. Most young patients who have matched
donors are successfully treated by bone marrow transplantation.
d.. Drug therapy, which suppresses the immune system, is another treatment
choice that is very successful.
Current Research
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 bone marrow transplant regimen for
patients who do not have a matched sibling donor and who do not respond to
the drug therapy.
Alternative Names: None
Definition
Aplastic anemia is a rare, non-contagious and often life-threatening
disorder that results from the unexplained failure of the bone marrow to
produce red blood cells, white blood cells and platelets. A decrease in the
production of blood cells means that patients are more susceptible to
bleeding, fatigue and infections.
Incidence
Aplastic anemia affects two to six people per million worldwide. The disease
is more prevalent in Asia than in the Western world. Most cases occur in
older adults, but a significant number of children are affected.
Influencing Factors
a.. The cause of the disease in an individual patient is usually unknown,
although the disease has been linked to environmental toxins such as benzene
derivatives, some drugs, pesticides and chemicals.
b.. Aplastic anemia can be caused by a virus in some individuals.
c.. There is evidence that parts of the immune system play a role in the
disease process.
Treatment Strategies
a.. Patients with severe aplastic anemia require immediate hospital
treatment.
b.. Initial treatment often includes blood and platelet transfusions and
intravenous antibiotics.
c.. Bone marrow transplantation is recommended for patients who are good
candidates and who have matched donors. Most young patients who have matched
donors are successfully treated by bone marrow transplantation.
d.. Drug therapy, which suppresses the immune system, is another treatment
choice that is very successful.
Current Research
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 bone marrow transplant regimen for
patients who do not have a matched sibling donor and who do not respond to
the drug therapy.
