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300 Longwood Avenue
Boston, MA 02115
(617) 355-6000
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My Child Has:
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Myelodysplastic Syndrome
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Myelodysplastic syndrome (MDS) is a disease of the blood. It develops in the bone marrow, the soft, spongy center of the long bones that produces the three major blood cells: white blood cells to fight infection; red blood cells that carry oxygen; and platelets that help blood clot and stop bleeding.
MDS occurs when the bone marrow does not properly produce enough red blood cells, white blood cells and platelets. With this disease, the blood cells lose their ability to mature and function properly. Red and white blood cells may mature, but not normally. The number of immature blood cells, called blasts, increases, and these blasts invade the bone marrow, making it impossible to work effectively.
As you read further below, you will find general information about MDS. If you would like to view summary information about cancer first, see the cancer overview.
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Some cases of MDS arise by chance, meaning there is no identifiable cause. Many people with MDS have a chromosomal abnormality associated with the disease, however these changes are not inherited from someone in the family. Instead, these abnormalities are changes in the chromosomes that occur as a result of the disease. The most common abnormalities occur in chromosomes 5 and 7. Certain chromosomal abnormalities like Down syndrome are associated with an increased risk for the development of childhood MDS.
MDS is sometimes found in patients who have had chemotherapy or radiation therapy for another malignancy.
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Physicians have identified several types of myelodysplastic syndrome, based on how blood and marrow cells appear under the microscope. Types are defined by what kind and what percentage of blasts (immature blood cells) are found in the marrow and blood. The types are:
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Because MDS is a disease of the blood-forming tissue called the bone marrow, the initial symptoms are often related to irregular bone marrow function. The bone marrow is responsible for producing the body's blood cells, including the red blood cells, white blood cells, and platelets.
The following are the most common symptoms of MDS, however, each child may experience symptoms differently. Often, MDS is discovered when a child is having a routine blood test for other reasons. Symptoms may include:
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- anemia - when red blood cells are unable to be produced because of the crowding in the marrow, anemia is present. With anemia, the child may appear tired, pale, and may breathe faster to compensate for the decrease in oxygen carrying capacity. The number of red blood cells on a blood count will be below normal.
- recurrent infections - although there may be an unusually high number of white blood cells on a blood count of a child with leukemia, these white blood cells are immature and do not fight infection. The child may have had repetitive viral or bacterial infections over the past few weeks.
- bone and joint pain - pain in bones and joints is another common symptom of leukemia. This pain is usually a result of the bone marrow being overcrowded and "full."
- abdominal distress - abdominal pain may also be a symptom of leukemia. Leukemia cells can collect in the kidney, liver, and spleen, causing enlargement of these organs. Pain in the abdomen may cause a child to have loss of appetite and weight loss.
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It is important to understand that the symptoms of MDS may resemble other blood disorders or medical problems. These are common symptoms of the disease, but do not include all possible symptoms. Children may experience symptoms differently. Always consult your child's physician if you have concerns.
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In addition to a complete medical history and physical examination, diagnostic procedures for leukemia may include:
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- bone marrow aspiration and biopsy - marrow may be removed by aspiration or a needle biopsy under local anesthesia. In aspiration biopsy, a fluid specimen is removed from the bone marrow. In a needle biopsy, a core sample of bone and bone marrow is removed. These methods are often used together.
- complete blood count (CBC) - a measurement of size, number, and maturity of different blood cells in blood
- additional blood tests - may include blood chemistries, evaluation of liver and kidney functions, and genetic studies
- chromosomal analysis - additional tests that can help distinguish MDS from other leukemias and help physicians tailor treatment to the specific disease
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Specific treatment for MDS will be determined by your child's physician based on:
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- your child's age, overall health, and medical history
- how your child's physician expects the disease to progress
- your opinion or preference
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Treatment usually begins by addressing the presenting symptoms such as anemia, bleeding, and/or infection. In addition, treatment for MDS may include (alone or in combination) the following:
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- chemotherapy - a drug treatment that works by interfering with the cancer cell's ability to grow or reproduce. Chemotherapy is generally used to treat the more aggressive types of MDS, including RAEB and RAEB-t.
Different groups of drugs work in different ways to fight cancer cells and shrink tumors. Chemotherapy may be used alone for some types of cancer or in conjunction with other therapy such as radiation. Often, a combination of chemotherapy drugs is used to fight a specific cancer. Certain chemotherapy drugs may be given in a specific order depending on the type of cancer it is being used to treat. While chemotherapy can be quite effective in treating certain cancers, the agents do not differentiate some normal healthy cells from cancer cells. Because of this, there can be many adverse side effects during treatment. Being able to anticipate these side effects can help the care team, parents, and child prepare, and, in some cases, prevent these symptoms from occurring, if possible.
Chemotherapy is systemic treatment, meaning it is introduced to the bloodstream and travels throughout the body to kill cancer cells. Chemotherapy can be given:
- as an injection into the muscle or fat tissue
- intravenously (directly to the bloodstream; also called IV)
- intrathecally - chemotherapy given directly into the spinal column with a needle
- stem cell transplant - offering the best option for overcoming MDS, this treatment involves stem cells, a specific type of cell from which all blood cells develop. Stem cells develop into red blood cells to carry oxygen, white blood cells to fight disease and infection, and platelets to aid in blood clotting. Stem cells are found primarily in the bone marrow, but some also circulate in the blood stream.
Treatment begins with high-dose chemotherapy (perhaps combined with radiation therapy) to destroy all of the bone marrow, stem cells and cancerous cells, in the body. Then, healthy stem cells from another person is used to help restore normal blood production in the patient with MDS. The use of stem cells from another individual whose tissue is the same as, or almost the same as, the patient's is called allogeneic transplantation.
- biological therapy - a wide range of substances that may be able to involve the body's own immune system to fight cancer or lessen harmful side effects of some treatments. Among the treatments for MDS are growth factors, substances that encourage bone marrow to produce healthy blood cells and platelets.
- other medications - to prevent or treat damage to other systems of the body caused by treatment, for example, to prevent or treat nausea
- blood transfusions - red blood cells, platelets
- antibiotics - to prevent/treat infections
- continuous follow-up care - to determine response to treatment, detect recurrent disease, and manage late effects of treatment
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Prognosis greatly depends on:
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- disease response to treatment.
- age and overall health of the child.
- your child's tolerance of specific medications, procedures, or therapies.
- new developments in treatment.
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As with any cancer, prognosis and long-term survival can vary greatly from child to child. Prompt medical attention and aggressive therapy are important for the best prognosis. Continuous follow-up care is essential for the child diagnosed with MDS. Side effects of chemotherapy, as well as second malignancies, can occur in survivors of MDS. New methods are continually being discovered to improve treatment and to decrease side effects of the treatment for the disease.
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In the area of stem cell transplantation, the Dana-Farber/Children's Transplant Program is one of only eight institutions around the country that are investigating the use of umbilical cord transplantation. Also under study is a novel method for preventing graft versus host disease, a serious complication that occurs when transplanted cells do not recognize the tissues and organs of the recipient's body and react against the recipient's tissue. The result of this treatment approach, if it continues to be as successful, will be that the degree of match between donor and the recipient will not need to be particularly close, greatly increasing the pool of potential donors for each patient. This could also eliminate the need for long-term drug therapy traditionally needed to treat graft versus host disease.
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The information on this website should not be taken as medical advice,
which can only be given to you by your personal health care professional. |
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