Treatment for these diseases is based on the exact type of MDS or MDS/MPD as well as other factors such as the person’s age and whether they have other serious diseases. Treatment is given by a specialist such as a cancer doctor (oncologist) or someone who treats blood diseases (hematologist).

Chemotherapy

Chemotherapy (most often called "chemo") is the use of drugs, taken by mouth or injected into a vein, to treat cancer. The drugs enter the bloodstream and reach most places in the body. While chemo drugs kill cancer cells, they can also damage normal cells, causing side effects. These side effects depend on the type of drugs given, the dose, and how long they are given.

Temporary side effects can include:

• hair loss (the hair grows back once treatment ends)
• mouth sores
• loss of appetite
• nausea
• vomiting
• greater chance of infection (from a shortage of white blood cells)
• easy bruising and bleeding (from low platelet counts)
• fatigue (from a shortage of red blood cells)

Chemotherapy can also damage organs such as the kidneys, liver, brain, heart, among others. The doctor will watch carefully for all side effects and adjust treatment as needed. Your health care team often can suggest ways to lessen side effects. For example, other drugs can be given along with the chemo to prevent or reduce nausea and vomiting.

The exact way chemo is given will vary greatly with each patient. Some patients receive low doses of one or more drugs.

If a patient’s white blood cell counts are very low during treatment, the risk of infection can be reduced by taking certain measures. Patients should be very careful about washing their hands and not eating fresh, uncooked fruit and other foods that might carry germs. They might also wear a surgical mask around crowds. The doctor might also suggest giving strong antibiotics even before an infection has developed.

If platelet counts are low, patients might have platelet transfusions--or red blood cell transfusions if their red cell counts are low.

Growth Factors

Growth factors are substances that speed up the making of blood cells in the bone marrow. The body itself makes growth factors, but scientists have also learned how to make them in the lab. They can then be given to patients to help their bodies make more blood cells.

A shortage of blood cells causes most of the symptoms in people with MDS, so giving growth factors can help some patients. There are several different growth factors that might be used, depending on the patient’s situation. Male hormones (androgens) can also boost the making of blood cells for a small number of people with MDS. But male hormones can cause side effects. Many doctors only use them if there are no other good treatment options for a patient.

Many cancers seem to need new blood vessels (angiogenesis) to support their growth. Some drugs which hinder this process can slow down the rate of MDS in some patients. One of these drugs, thalidomide, has had success in treating MDS. A few patients taking this drug have been able to avoid having blood transfusions because their bodies were able to make enough normal red blood cells. Many people, though, have trouble with the drug because it causes drowsiness, fatigue, and constipation. A new drug, called lenalidomide (Revlimid^®), has fewer side effects and has worked well in treating low grade MDS. About half the patients treated with it stop needing blood transfusions.

Other treatment approaches involve using drugs that block a type of blood cell called the T-lymphocyte which can interfere with the making of normal blood cells. Some other drugs slow down the genes that promote cell growth. There are several of these drugs that are being used tested.

Supportive Treatment

For many patients with MDS, the risk of the disease changing into leukemia is low. The main goal of treatment for these patients is to prevent problems from low blood counts. These patients might have severe fatigue because of low red blood cell counts and need to get many blood ransfusions. One problem from a lot of blood transfusions is the build-up of iron in the blood. Too much iron can cause liver and heart damage in people who have had many transfusions over a period of years. There are drugs that can be given to help prevent this.

Patients with low white blood cell counts might get infections which could be very serious. These infections are treated with antibiotics.

Stem Cell Transplant

In a stem cell transplant (SCT) the patient’s bone marrow is destroyed on purpose using high doses of chemo and radiation. Once the abnormal bone marrow cells are destroyed, they are replaced with new, disease-free blood-forming stem cells. For patients with MDS or MPD, these new stem cells come from a donor—often a brother or sister. If there is no matching family member to be a donor, then a matched, unrelated donor is used.

The process works like this: Stem cells are taken from the donor’s blood. The stem cells are frozen and stored. The patient then receives high-dose chemo and, often, radiation treatment. This destroys the MDS cells, but it also kills the normal cells in the bone marrow.

The donor’s stem cells are then given to the patient as a transfusion. These cells travel through the blood to the bone marrow where they settle and start to grow. After about 14 to 21 days they begin making white blood cells, then platelets. Several weeks later they start making red blood cells.

Until the patient’s white blood cell count is above 500, he or she will need to be very careful about getting an infection. Patients are kept in “protective isolation” during this time. When the white cell count is near 1,000 the patient can usually leave the hospital. After that, there will be frequent visits to the clinic for about 6 months.

Side effects: the side effects of SCT can be divided into early and long-term effects. Early on, the side effects are the same as those caused by high-dose chemo. Those side effects that occur later can last for a long time. They include:

• radiation damage to the lungs (this is rare)

• graft vs. host disease: this can happen when the donor’s immune system cells attack the patient’s tissues. Symptoms include weakness, tiredness, dry mouth, rashes, infection, and muscle aches.

• damage to the ovaries that can result in infertility, sudden change of life (menopause), and hot flashes

• damage to the thyroid gland

• cataracts (clouding of the eye that can cause vision problems)

Right now, stem cell transplant is the only treatment that can cure some patients with MDS or MPD. About one third of patients who have SCT may be cured. But between one fourth to one third may die from complications of this treatment. The chance for a cure is higher for younger patients and for those whose MDS hasn’t begun to change into leukemia.

Most doctors consider SCT only for patients younger than 50 who are otherwise in good health. Some doctors set the age limit at 40.

There is a new type of transplant, called a mini-transplant that is now being tried. It differs from the standard approach in that low doses of chemo or radiation are used. Side effects, though, are still a major problem with this low-dose method.

Stem cell transplant is a complicated treatment. The best place to have it done is at a cancer center where the staff has experience with the treatment and with helping patients during recovery.

It is also very expensive ($100,000 or more) and requires a long hospital stay. Because it is the recognized treatment for MDS, insurance companies should pay for it.

If you would like more detailed information on SCT please see the American Cancer Society document Bone Marrow & Peripheral Blood Stem Cell Transplants. Last Revised: 01/05/2007