Note: This is for informational purposes only. Doctors cannot provide a diagnosis or individual treatment advice via e-mail. Please consult your physician about your specific health care concerns.
In this group of diseases, those at the chronic and stable end of the
spectrum are often not treated.
In patients with more troublesome decreases in blood cell counts, drugs
that can stimulate blood cell production may be useful.
There are treatments for all patients with myelodysplastic syndromes. The choice
of treatment depends on the type of myelodysplastic syndrome, and the patient's
age and general health.
Often the main treatment is giving red blood cells or platelets by a needle
in a vein (transfusion) to control anemia or bleeding. Vitamins or other drugs
may also be given to treat anemia.
Various treatment options include:
Chemotherapy and biological therapies are being tested in clinical
trials. Chemotherapy uses drugs to treat disease. Chemotherapy may be taken
by pill, or it may be put into the body by a needle in the vein or muscle.
Chemotherapy enters the bloodstream, travels through the body, and affects
cells throughout the body. Biological therapy tries to get the body to fight
disease. It uses materials made by the body or made in a laboratory to boost,
direct, or restore the body's natural defenses against disease.
Stem cell/bone marrow transplantation is a newer type of treatment
that uses high doses of chemotherapy and/or radiation therapy (high doses
of X-rays or other high-energy rays) to destroy all of the bone marrow in
the body, then transplants healthy bone marrow back into the body. Healthy
marrow is then taken from another person (a donor) whose tissue is the same
or almost the same as the patient's. The donor may be a twin (the best match),
a brother, sister, or other relative, or another person not related. The healthy
marrow is given to the patient through a needle in the vein, and the marrow
replaces the marrow that was destroyed. A bone marrow transplant using marrow
from a relative or person not related to the patient is called an allogenic
bone marrow transplant.
In an autologous stem cell transplant, a patient's own stem cells are
removed from his or her bone marrow or bloodstream. This type of transplant
is used in treating some types of cancer but is not used for patients with
MDS because it is not possible to get normal stem cells from the patient that
are not MDS cells.
Growth factors: Erythropoietin, a growth factor that promotes red
blood cell production, can help avoid red blood cell transfusions in some
patients. Recently it has been found that combining erythropoietin with G-CSF
improves the patient's response to the erythropoietin. Research on a new growth
factor called thrombopoietin, which promotes platelet production, is in progress.
Differentiation agents: Differentiation agents cause bone marrow blasts to
develop into mature blood cells. These drugs have shown promising results
in studies of bone marrow cells grown in laboratory dishes; however, their
value in patients with MDS is less clear. Several differentiation agents,
including retinoids (chemicals related to vitamin A) and vitamin D, are being
tested in clinical trials to find the best way of combining these agents together
or with other treatments.
Supportive therapy: For many patients with forms of MDS such as RA
(refractory anemia) and RARS (refractory anemia with ringed sideroblasts),
as well as other patients with favorable prognostic factors, the risk of progression
to acute leukemia is low. The main goal of treatment is to prevent symptoms
and complications of low blood cell counts. For example, low red blood cell
counts (anemia) can cause severe fatigue. Patients with MDS and anemia often
benefit from receiving red blood cell transfusions.
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