MDS Treatments: More Options, Better Outcomes

Standard of Care

Our myelodysplastic syndrome (MDS) treatment team follows the Clinical Practice Guidelines in Oncology — the National Comprehensive Cancer Network’s internationally recognized guidelines for treating different types of cancer. They are the most comprehensive, most frequently updated clinical practice guidelines available in any area of medicine.

Treatment for MDS can include

  • Active surveillance (watchful waiting)
  • Disease-modifying therapy
  • Chemotherapy
  • 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

The choice of treatment depends on the following

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

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

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. Patients who receive them find that their blood cells are coaxed into functioning more effectively. As a result, these disease-modifying drugs can significantly improve the quality of life for many MDS patients and modestly extend overall survival.


Chemotherapy can be an important part of treatment for many MDS patients, particularly those with high- or intermediate-2 risk 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 induce 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. At Roswell Park, induction treatment is conducted in a state-of-the art inpatient unit with a specialized team of nurses. Because chemotherapy can also damage healthy cells, patients receiving induction therapy at Roswell Park are admitted into our facility for approximately one month.

Consolidation or Intensification Therapy

To offer the best chance of destroying lingering abnormal cells and prevent relapse, additional rounds of chemotherapy drugs are given every month, on an outpatient basis. MDS cannot be cured with chemotherapy. An allogeneic transplant is the only potential cure for patients with MDS.

Blood and Marrow Transplantation

Roswell Park offers the most experienced adult blood and marrow transplantation (BMT) service in Western New York, performing 160-180 transplants every year. Because BMT offers the only chance for curing MDS and the best chance for curing leukemia, our physicians work closely with our BMT specialists to evaluate the status of all of our MDS and leukemia 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.

Outcomes for patients treated through the Blood and Marrow Transplant Center at Roswell Park are among the highest in the nation. The 2014 Center for International Blood and Marrow Transplant Research report shows that Roswell Park achieved higher than expected one-year survival ratings for allogeneic blood and marrow transplants.

Roswell Park’s BMT unit and its 14 private patient rooms are HEPA-filtered to maintain the most stringent protection against airborne pathogens. The nursing staff is specially trained in the management and care of BMT patients, who have special needs because of their weakened immune systems.

Roswell Park offers two types of BMT for eligible patients with MDS and leukemia.

Allogeneic BMT

For 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 have special expertise with low-dose transplantation, also called mini-transplants. These procedures require lower treatment doses but achieve the same success rates as high-dose protocols. As a result, a larger number of MDS and leukemia patients previously considered ineligible for blood and marrow transplantation may be good candidates 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 residual disease in your body is destroyed with chemotherapy and/or radiation, and afterward, the stem cells that were collected previously are given back to you, in a procedure called a rescue. Autologous BMT is rarely performed at Roswell Park for MDS or leukemia.

Long-Term Survival

Roswell Park has a higher five-year survival rate for its BMT patients than the national average and is consistent with other leading medical centers. We attribute this 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.