Table of Contents

What is MDS?

Myelodysplastic syndromes (MDS) are a group of diseases that affect the bone marrow and blood, typically developing from a problem that occurs during blood cell production. MDS can develop in any person at any age but is most common in older adults. While MDS may not cause symptoms in some people, others may experience fatigue, shortness of breath, easy bruising or bleeding, and frequent infections. If left untreated, the condition can progress to a cancer of the blood or bone marrow called acute myelogenous leukemia (AML).

Although treatments for MDS vary depending on the type and severity of the disease, most people with MDS respond well to treatment. Treatment options include medication therapy, blood transfusions, and stem cell transplantation. Advances in research have led to more effective treatment options for patients with MDS and improvement in their quality of life.

Your MDS treatment depends on:

  • Your type of MDS
  • How severe or aggressive it is
  • Whether your bone marrow has less than 5 percent of healthy cells (that’s called “very low bone marrow blast count”)
  • Whether you have other conditions, like other types of cancer or anemia
  • Your overall health

What are the treatments for MDS?

MDS can’t usually be cured, but there are treatments that can help control the condition. The main treatments for MDS are:

  • chemotherapy
  • stem cell transplant
  • blood transfusions
  • medicines to improve the bone marrow’s ability to make blood cells
  • drugs to prevent or treat infections, anemia and bleeding.

What are MDS clinical trials?

MDS clinical trials are tests of new treatments or drugs that haven’t been approved by the FDA yet. These therapies are being tested to see if they are safe and effective. They might also be tested to find out if they work better than other drugs or therapies already in use.

The treatment being tested has to meet certain criteria before it can be used in a clinical trial. It is usually a drug or therapy that has shown promise in laboratory studies or animal testing. The goal of MDS clinical trials is to make sure that the new treatment works well and is safe for people with MDS.

It’s important to know that participating in a clinical trial is completely voluntary and you can leave the study at any time if you aren’t comfortable with it, if it’s not working, or if you experience any side effects.

What kinds of clinical trials are there?

MDS clinical trials fall into two categories: interventional and observational. Interventional trials test the effectiveness of a particular treatment or combination of treatments for a particular disease. Participants will be assigned to either the treatment being tested or to a standard treatment regimen based on their condition. Observational trials collect data from people who have been diagnosed with a specific disease and follow them over time. The results of these studies can provide clues about how lifestyles, environmental factors, genetics, and other important factors affect the course of a disease and its symptoms.

Disease-modifying therapy

Disease-modifying therapy is treatment that aims to change the natural course of MDS. Treatment is given for the following reasons:

Relieve symptoms and decrease the chance of having complications from MDS

Improve blood cell counts

Reduce the risk of acute myeloid leukemia (AML) developing from MDS

Blood transfusions are used to treat low red blood cell counts (anemia). Erythropoiesis-stimulating agents (ESAs) are drugs that may be used to improve blood cell counts in some people with MDS. ESAs may increase the number of red blood cells and decrease the need for transfusions. They don’t work in everyone, especially in those whose anemia is caused by a lack of iron or vitamin B12 or who have excess iron stores. The drug Procrit® (epoetin alfa) is an ESA commonly used in MDS.

Medications such as azacitidine and decitabine can help control symptoms of MDS by stimulating production of more healthy blood cells. These medications also reduce the risk of developing AML. These treatments are given as pills or injected into a vein (intravenously) over several days.

Chemotherapy

Chemotherapy uses drugs to destroy cancer cells. It is the most common form of treatment for MDS. The type of chemotherapy you receive depends on the severity of your disease, how well your organs are functioning, and other factors. In some cases, your doctor may prescribe a combination of different types of chemotherapy drugs.

In general, the goal of chemotherapy is to eliminate as many abnormal blood-forming cells as possible while trying to keep normal cells safe from harm. Chemotherapy works best when it is given in cycles. Each period of treatment is followed by a rest period to give your bone marrow time to recover and produce new blood cells.

Induction therapy

Induction therapy for MDS or myelodysplastic syndrome is usually offered to patients who are suffering from severe anemia and those who have thrombocytopenia. There are several forms of induction therapies that could be used including blood transfusion, the use of cytokines and growth factors, antibiotics and intravenous immune globulin. The most common form of induction therapy for MDS is the use of antibiotics since this can help patients recover faster. It can also help in preventing infections and decreasing the number of platelets in the body.

Consolidation or intensification therapy

The role of consolidation or intensification therapy for MDS is not clear. A recent meta-analysis including seven studies and 175 patients was published. Only three studies were randomized controlled trials. In the meta-analysis, consolidation therapy appeared to significantly improve both overall survival and complete response rate but there was no significant difference in relapse rate.

In a further study, 115 patients with MDS following consolidation therapy were compared with those without consolidation therapy (non-consolidation group). A total of 68% in the consolidation group achieved complete remission, whereas 28% in the non-consolidation group did so. However, there was no significant difference in overall survival between the two groups. The median survival time of the first group was 24 months (range 10–51 months) and that of the second group was 22 months (range 8–48 months).

Blood and marrow transplantation

Blood and marrow transplantation involves replacing the blood-forming cells that are damaged or destroyed by MDS. This procedure can be used to treat MDS in some people. However, it is only an option for people with a good chance of benefiting from the procedure.

To prepare for the transplant, you will receive chemotherapy and/or radiation therapy to destroy the blood-forming cells in your bone marrow. Then, healthy blood-forming cells are put into your body through a tube that goes into your vein. These new (donor) cells travel to your bone marrow, where they begin to produce healthy blood cells.

The healthy blood-forming cells can come from:

A donor whose cells match yours (allogeneic transplant). The donor may be someone who has agreed to donate his or her bone marrow or peripheral blood stem cells as part of a standard donation process. Or, the donor may be a family member whose cells are most likely to match yours.

Allogeneic BMT

​Allogeneic BMT is a process that involves giving stem cells to the patient for treatment purposes. These stem cells are usually extracted from the bone marrow of a healthy donor and transferred to the patient. This procedure is used to treat disorders of the blood, which may include leukemia, lymphoma, multiple myeloma, and aplastic anemia. The procedure aims at replacing the dysfunctional bone marrow or stem cells in order to restore their normal function. Allogeneic transplants are also referred to as allogeneic BMT (bone marrow transplant). In this procedure, healthy stem cells are introduced into the body by intravenous injection. These stem cells travel through the bloodstream until they reach the bone marrow where they replicate and grow.

Autologous BMT

Autologous BMT is a procedure that involves collecting and storing your own stem cells, and then giving them back to you after high dose treatment. In this procedure, stem cells are collected from your blood by a machine called an apheresis machine. They are stored in a deep freezer until the time for your transplant. During the transplant, the stem cells are thawed and given back to you through a vein as a blood transfusion. The stem cells travel through your blood stream to the bone marrow, where they begin making new blood cells. This process usually takes two to four weeks after the transplant. Your doctor will be monitoring how quickly you recover by doing frequent blood tests.

What are the survival rate for MDS?

The 5-year survival rate of MDS is estimated at around 10%. The prognosis of those patients with MDS who have no or few symptoms, especially if their bone marrow biopsy shows only a few blasts in their blood (5% or less), is much better than for those whose blood has more blasts.

MDS is often considered a disease that progresses slowly and patients can live with it for many years. The median survival time ranges from 12 to 18 months, depending on the number of blasts in the bone marrow biopsy and the amount of anemia present. For those who have higher numbers of blasts present in their bone marrow, the median survival time may be as short as 6 months.

Non-blastic MDS (without an abnormal number of blast cells) has a better prognosis than blastic MDS (with more blast cells). People who have been diagnosed with non-blastic MDS are less likely to progress to acute myeloid leukemia than those with blastic MDS.