Treatment

Treatments

Treatments for MDS fall under three categories: supportive care, drug therapy, and transplantation. At Siteman, your doctors will thoroughly evaluate your test results, your genetic profile, your overall health, and your desired lifestyle to decide which types of treatment will be best for you.

Supportive care

The goal of supportive care is to relieve the symptoms caused by myelodysplastic syndromes. Often, this involves improving blood cell counts through either medications or transfusions. Depending on your age, the score of your MDS, and your health, this may be all that is needed to manage your symptoms and ensure that you regain your quality of life.

To get your blood cells back up to healthy levels, you may be treated with “growth factor” drugs (also known as “erythropoiesis-stimulating agents,” or ESAs) that encourage your bone marrow to produce more cells. The drugs are tailored to different types of blood cells: drugs such as epoetin alfa help to raise red blood cell levels, while filgrastim raises the level of white blood cells.

Transfusions are another, quicker way to replenish your body’s supply of red blood cells and platelets. You will receive a transfusion if your doctor feels that your blood cell counts have become unacceptably low and need to be improved immediately. Donated cells and platelets will be introduced into your body through an IV line. Drugs such as aminocaproic acid can help decrease bleeding and bruising if platelet transfusions do not improve your condition.

Drug therapy

The drugs azacitidine, decitabine, and lenalidomide are used to actually treat MDS, rather than just improve the symptoms it causes.

Azacitidine and decitabine are technically chemotherapy drugs. However, they are unlike other chemo drugs in that they are thought to help cells to mature normally as well as attacking cells that are dividing too quickly. Consequently, the side effects you experience from these drugs may not be as severe as the side effects caused by conventional chemotherapies: you’re unlikely to lose your hair, for instance. Azacitidine and decitabine can be given as out-patient treatments and are typically well-tolerated, especially by older or frailer patients.

Lenalidomide is often prescribed for patients who have MDS with isolated del(5q). This drug overrides the defective del(5q) chromosome so that the bone marrow can produce more red blood cells. Patients with low-risk MDS are also given lenalidomide.

Patients may also be treated with immunosuppressive drugs that can sometimes prevent the body from attacking healthy blood cells by mistake.

Depending on the type of MDS you have and your risk level, you may be treated with the same chemotherapy regimen that is used to treat patients who have already progressed to AML.

Many patients with MDS choose to participate in clinical trials. Clinical trials are good for patients in two primary ways. First, trials allow them to receive more advanced treatments that often turn out to be better than the existing standard of care. There are not many MDS drugs to choose from, and although azacitidine and decitabine can be very effective, they may not work for everyone. Second, clinical trials ensure increased access to physicians and staff, who must carefully monitor the health and progress of the patients taking part in the trial process.

Washington University physicians at the Siteman Cancer Center are currently involved in a number of clinical trials for MDS.

Stem cell transplant

Also commonly described as “bone marrow transplants,” hematopoietic stem cell transplants (HSCT) are the only known way to cure myelodysplastic syndromes. In an HSCT, the abnormal stem cells in the bone marrow are replaced with healthy stem cells from a donor. The procedure is most effective when the donor’s cells closely match your own. Your immediate, biological family members, such as your parents, children, and siblings, are likely to be the best possible match and may be able to donate stem cells for you. However, you may also be able to find a good match through a donor registry.

There are several steps to an HSCT. First, the donor’s stem cells are collected. This process used to involve extracting stem cells directly from his or her bone marrow in an operating room – hence the name “bone marrow transplant.” Today, the donor is given a medication that releases the stem cells from the bone marrow into the blood stream. This makes it easier to collect the cells.

Meanwhile, the patient undergoes chemotherapy in order to kill all of the defective cells in the bone marrow. When the course of chemotherapy is complete, he or she then receives the healthy stem cells from the donor through an IV infusion. These new stem cells will begin to multiply in the bone marrow and start producing new, normal blood cells. If the procedure is successful, the patient’s myelodysplastic syndrome may be completely cured.

Though HSCT offers the possibility of a cure, the process carries serious risks. The chemotherapy temporarily leaves the patient without any functioning bone marrow, which means that he or she becomes susceptible to severe infections and excessive bleeding. There’s also a chance that the donor stem cells will be rejected by the patient’s body, a dangerous complication known as “graft versus host disease.” Undergoing a stem cell transplant requires a hospital stay of several weeks so you can be treated and monitored in a safe environment with precautions in place to protect you.

Siteman Cancer Center has a dynamic, nationally-renowned stem cell and bone marrow transplantation program. Patients who come to Siteman for a transplant are cared for by expert doctors and nurses in facilities that have been designed to meet their needs. Their safety and comfort are of the utmost priority to everyone involved in their care.

Stem cell transplants at Siteman Cancer Center

Siteman Cancer Center is a national leader in stem cell – or bone marrow – transplants. Barnes Jewish Hospital, one of our parent institutions, helped pioneer bone marrow transplantation. Washington University Physicians have performed more than 7,500 bone marrow transplants since 1982 and currently perform close to 400 transplants every year. When you come to Siteman, you will see a physician who has had countless hours of experience conducting bone marrow transplants and leading patients through the transplant journey.

Bone marrow transplants remain a major area of research for Washington University Physicians at Siteman Cancer Center. They’re studying new therapies to treat relapses in transplant patients, using genetic sequencing to determine why transplant patients relapse, and searching for better ways to prevent life-threatening complications such as graft versus host disease. Their findings will go on to impact the way that stem cell transplants are performed, making them safer and more effective.

For more information, visit our Bone Marrow and Stem Cell Transplant Program page.