Current Treatment Options

There are a variety of therapeutic options for treating myelodysplastic syndromes (MDS). These options can be classified as supportive care (discussed in Managing Myelodysplastic Syndromes), low-intensity treatments or high-intensity treatments.1

Low-intensity treatments include hematopoietic cytokines, growth factors and immunosuppressive therapy.1 These usually are administered to patients with low-risk disease.1

High-intensity treatment is chemotherapy or hematopoietic stem-cell transplantation (HSCT). It is used only for patients with advanced MDS who have an appropriate health status.1

Treatment plans are designed in response to the patient’s specific needs and profile. The criteria used to create the treatment plan include the patient’s MDS type, age, International Prognostic Scoring System (IPSS) score, and the stage of the disease. From this information, doctors can create a therapy regimen that supplements ongoing supportive care and best addresses the disease.1

Growth Factors

Recombinant human erythropoietin (EPO) has been shown to be effective in treating symptomatic anemia.1 In a small number of patients, EPO has even eliminated the need for transfusion.2 High doses of EPO administered subcutaneously daily or 3 times a week have been proven effective in 20%-30% of patients, but some patients do not respond. For this nonresponsive group, EPO is supplemented with granulocyte colony-stimulating factor (G-CSF) or granulocyte-macrophage colony-stimulating factor (GM-CSF). This supplementation can double the response rate.1

Combining G-CSF, GM-CSF and EPO has shown to be very effective in bolstering neutrophil and red cell production, and other combinations of hematopoietic hormones may produce a synergistic effect.1,3 Although G-CSF and GM-CSF have been shown to improve neutrophil production rates, they have not been shown to decrease infection or improve infection treatment.1

Immunosuppressive Therapy

Although immunosuppressive therapy has shown some promise in treating MDS, particularly in patients with the hypoplastic form, it has not been validated.1 Hypoplastic MDS resembles aplastic anemia (AA), which has been successfully treated with antithymocyte globulin (ATG) and cyclosporine. For this reason, ATG and cyclosporine have been administered as MDS treatment, primarily in hypoplastic patients but also in patients with the RA subtype.2

These treatments provide fewer benefits for MDS patients, however, than they do for their AA counterparts. Also, the beneficial effects are limited and short-lived, and they are accompanied by significant side effects.2

Chemotherapy

Induction chemotherapy is used to treat patients in the high-risk or second intermediate-risk category. Studies of several chemotherapy strategies have not shown consistent benefits for MDS patients. Although these approaches have a greater chance of changing the natural history of the disease, they also have an increased risk of regimen-related morbidity and mortality.1

About 30% of the MDS patients treated with chemotherapy may go into remission, but the disease usually returns within a year.4 Still, for patients younger than 60 years who have high-risk disease and a good performance status, chemotherapy is recommended.1

MDS chemotherapy involves two strategies, one to address indolent MDS and another for the proliferative subtypes. Agents for indolent MDS include thalidomide, lenalidomide, arsenic trioxide, the retinoids, interferons, farnesyl transferase inhibitors, infliximab, amifostine, valproic acid, and vitamin D. These agents also are used to address proliferative MDS, along with agents used to treat acute myeloid leukemia.2 It should be noted, however, that the only drug approved for MDS by the FDA is 5-azacytidine.2

HSCT

HSCT is the only proven cure for MDS, but it rarely is used for patients who are older than 40 years.4 This is true for two reasons. First, HSCT generally relies on marrow donation of a sibling, which is harder to achieve in an elderly patient group due to the frequently reduced number of available donors. Second, the treatment is rigorous, requiring an intensive chemotherapy conditioning regimen that is only appropriate for patients with good performance status, of which there are few in the typical patient group.1

HSCT has been reported to cure MDS in up to one-third of recipients who are at high risk of developing leukemia, although these patients also have a higher post-HSCT relapse rate. The survival rate for patients who are not at risk of leukemia is about 50%.2

Given the physical demands of HSCT and the limited availability of donors, alternative methods are under investigation. Peripheral blood stem-cell transplants collect healthy blood cells from the donor’s blood rather than the marrow and then transfuse them into the MDS patients. This approach is an attractive alternative for older patients who may not have available donors.2

Another new approach for older patients is the nonmyeloablative transplant, also called the “mini” transplant, which uses a less toxic chemotherapy conditioning regimen to reduce side effects and make it more appropriate for older patients.2

Visit the Treatment Guidelines and Latest Developments pages for more information on treating MDS.