Stem-cell Transplantation

This procedure is the only way to cure MDS. Your doctor will give you high doses of chemotherapy to destroy the abnormal cells in your bone marrow. After chemotherapy, you will be given blood-forming stem cells from a disease-free, matched donor to help your body form new, healthy blood cells. Most younger patients will be given allogeneic stem-cell transplants because their bodies can tolerate the harsh side effects. Older patients will usually only be given stem-cell transplants once their disease becomes advanced. If a stem-cell transplant is not an option, your doctor will focus your treatment on relieving symptoms and side effects as well as trying to reduce and delay the risk of transformation to leukemia with other available treatments. It is important to know that deciding whether a transplant is the best option for you really depends on each individual patient, and many factors have to be taken into account, including the characteristics of the disease, your age, and other medical conditions as well as how good the disease is controlled prior to transplant. You should always address this issue in depth with your doctor.

Chemotherapy

Chemotherapy is the use of drugs to treat rapidly growing cancer cells in an effort to destroy them. Chemotherapy destroys your abnormal stem cells and lets your healthy blood cells return to normal levels. Chemotherapy medications may be given through the vein (intravenously) or by mouth (orally). You may be given the same combination of chemotherapy commonly given for AML to treat MDS.

Hypomethylating Agents

Hypomethylating agents are a form of chemotherapy that specifically targets your genes. These drugs destroy malignant cells and prevent your genes from stimulating cell growth. Hypomethylating agents can improve blood counts to the point where you no longer need blood transfusions, and reduce the risk of the disease progressing to acute leukemia. They are also the only approved treatment for high-risk MDS and can improve the survival if compared to other forms of therapy. They are given intravenously or as a subcutaneous injection during 5-7 days a month. Treatment with these agents, unlike with intensive chemotherapy, must be administered continuously as long as they are able of controlling the disease and being effective. It is important to take into account that their effectiveness may take 4-6 months to be able to be observed.

Immunomodulators

Immunomodulators stimulate your body's immune system to help it fight off cancer cells. These drugs can prevent the progression of low-risk MDS with certain chromosome changes to acute leukemia. These drugs can correct chromosome abnormalities and reduce the need for blood transfusions.

Growth Factors

Hematopoietic growth factors are naturally occurring, hormone-like substances that cause your blood cells to grow and mature. Doctors give these medications to increase blood cell production in your bone marrow. These substances are made in a laboratory and given to patients in higher-than-normal amounts. Growth factors can help your blood counts return to normal and can also relieve symptoms. Your doctor may give you a combination of growth factors, or combine growth factors with other treatments. Growth factors are usually given as a subcutaneous injection under the skin and are generally used in lower risk MDS. New therapies for developed for patients who failed growth factors including luspatercept and new agents like imetelestat, a telomerase inhibitor. Luspatercept was recently approved as frontline therapy for patients with low-risk disease.

Blood Transfusions

Often, the primary goal of treatment for MDS is to relieve the symptoms of low blood cell counts. Blood transfusions can replenish your levels of red blood cells, white blood cells, and platelets. Too many blood transfusions can cause excess iron to build up in your blood, which can cause organ and tissue damage. Your doctor may then give you chelating agents to remove the extra iron in your blood.

Clinical Trials

Clinical trials allow patients to try a new treatment before it is available to the general public. In some cases, this may be a new drug that has not been used in humans before, or it may be a drug or drug combination that is not currently used for that specific type of cancer. Early phase clinical trials are often used to test side effects of a drug or drug combination, while later phase clinical trials are used to see how effective a new treatment might be for a certain type of cancer. Clinical trials allow doctors and researchers to improve the treatment of cancers with possibly more effective therapies. A clinical trial may be a new, groundbreaking drug or it may have no effect. It is important to talk with your doctor about the pros and cons of clinical trials for your particular situation.

In MDS, clinical trials are always advisable, even in newly diagnosed patients, due to the relatively poor outcome of hypomethylating agents (HMA) and the diversity of the disease, which may allow tailored therapy within clinical trials for specific disease features (such as specific mutations or WHO subtypes). Clinical trials are also advisable in patients who failed HMA therapy.