MDS Treatments: More Options, Better Outcomes

The Highest Standard of Care

Our MDS team follows the National Comprehensive Cancer Network’s internationally recognized guidelines for treating cancer — the most comprehensive, most frequently updated clinical practice guidelines available in any area of medicine. Elizabeth Griffiths, MD, Director of MDS at Roswell Park, is among the national experts who serve on the NCCN committee that develops the guidelines for MDS.

The choice of treatment depends on:

  • The type of MDS you have
  • Your MDS disease history over time
  • Your age
  • Your International Prognostic Scoring System (IPSS) disease risk score and revised IPSS score.
  • Your overall health
  • Your mutational profile

Treatment for MDS can include:

  • Active surveillance (“watchful waiting”)
  • Disease-modifying therapy — such as chemotherapy or targeted therapies — which may lead to longer survival
  • Supportive care (including blood transfusions, blood-stimulating medicines and techniques to remove excess iron after multiple blood transfusions)
  • Blood and marrow transplant (BMT)
  • A combination of these treatments

Chemotherapy, disease-modifying therapy and supportive care may help relieve the symptoms of MDS, limit disease severity and extend life to some degree. However, a blood and marrow transplant (BMT) is the only potential cure for MDS.

Clinical Trials

Roswell Park offers clinical trials of promising new treatments for MDS. Learn about clinical trials and search for available clinical trials.

Disease-Modifying Therapy

Two new disease-modifying therapies known as azanucleotides are helping MDS patients achieve significant symptom relief over time. Azacitidine (Vidaza®) and decitabine (Dacogen®) are used for all MDS subtypes. Lenalidomide (Revlimid®) is used for treating only the 5q- syndrome type.

These drugs are given in low doses, making them much less toxic than standard chemotherapies. Many patients who receive them find that their blood cells begin to work more effectively, significantly improving quality of life and extending overall survival to some degree.


Chemotherapy can be an important part of treatment for many MDS patients, particularly those with high- or intermediate-2 risk MDS.

MDS cannot be cured with chemotherapy. An allogeneic bone marrow transplant (BMT) is the only potential cure for patients with MDS.

Induction Therapy

Patients whose MDS has transformed into acute myeloid leukemia sometimes require more intensive treatment, called induction. The goal of induction therapy is to cause a complete remission, or absence of abnormal cells in the bone marrow. Low-dose and high-dose treatments may be used, depending on the patient.

Because chemotherapy can also damage healthy cells, Roswell Park patients who receive induction therapy are admitted to our state-of-the art inpatient unit for approximately one month and are cared for by a specialized team of nurses.

Consolidation or Intensification Therapy

To offer the best chance of destroying lingering abnormal cells and prevent the disease from returning, additional rounds of chemotherapy drugs are given every month, on an outpatient basis.

Blood and Marrow Transplantation

Philip McCarthy, MD, Director of the Transplant & Cellular Therapy Center, explains the role of BMT in treating MDS.

Blood and marrow transplantation (BMT) offers the only chance for curing MDS. That’s why our physicians work with our BMT specialists to evaluate all our MDS patients on an ongoing basis, to determine who might benefit from the procedure. This helps ensure that everything is in place and ready to go if a transplant should be necessary.

We were among the first centers in the world to routinely offer BMT. Today we’re home to the most experienced adult BMT service in Western New York, performing 160-180 transplants every year and offering two types of BMT — including a low-dose, or “mini-transplant” — for eligible MDS patients. We are a Blue Cross/Blue Shield Blue Distinction Center for transplant and have been designated a Center of Excellence by Optum, MultiPlan, Interlink and Cigna Life Source.

Our Transplant & Cellular Therapy Center features 14 private patient rooms that are HEPA-filtered to maintain the highest level of protection against airborne pathogens. The nurses are specially trained in the management and care of BMT patients, who have special needs because of their weakened immune systems.

Allogeneic BMT

In an allogeneic transplant, your diseased bone marrow is destroyed with chemotherapy and/or radiation, and afterward you receive stem cells from a healthy donor in a procedure called a rescue.

Our experienced BMT experts also have special expertise with low-dose transplantation, or mini-transplant. This procedure requires lower doses of chemotherapy and radiation but achieves the same success rates as transplants that use high doses. As a result, many MDS patients who were once ineligible for BMT may now be able to undergo the procedure at Roswell Park.

Autologous BMT

For an autologous transplant, your stem cells are collected in advance, at a time when you have little or no evidence of disease in your blood. Then any remaining disease in your body is destroyed with chemotherapy and/or radiation. Afterward, the stem cells that were collected earlier are given back to you, in a procedure called a rescue. Autologous BMT is not generally used for treatment of MDS.

Long-Term Survival

Roswell Park was among the first centers in the world to routinely offer blood and marrow (stem cell) transplants, and we consistently have excellent patient survival results. Over a seven-year period, our patients who underwent allogeneic transplant (involving a donor) had either the same or significantly better-than-predicted survival compared with what would be expected based on patient age, other existing health problems, whether or not the patient was in remission at the time of transplant and how closely the patient was matched to the donor. We attribute this success to our highly specialized patient care team, the close working relationship among team members and our focus on laboratory research as we search continually for potential new treatment options.