Myelodysplastic Syndromes Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment for Myelodysplastic Syndromes
Therapies for myelodysplastic syndromes (MDS) are initiated in patients with a shorter predicted survival or in patients with clinically significant cytopenias. The impact of most MDS therapies on survival remains unproven.
Metastases at diagnosis are detected in approximately 25% of patients. The prognosis of patients with metastatic disease is poor. Current therapies for patients who present with metastatic disease achieve 6-year event-free survival (EFS) of approximately 28% and overall survival (OS) of approximately 30%.[2,3] For patients with lung/pleural metastases only, 6-year EFS is approximately 40% when utilizing bilateral lung irradiation.[2,4] In contrast, patients with bone/bone marrow metastases have...
The mainstay of treatment for MDS has traditionally been supportive care, particularly for those patients with symptomatic cytopenias or who are at high risk of infection or bleeding.[1,2] Transfusions are reserved for the treatment of active bleeding; many centers offer prophylactic platelet transfusions for patients with platelet counts lower than 10,000/mm3. Anemia should be treated with red-cell transfusions to avoid symptoms. (Refer to the PDQ summary on Fatigue for more information on anemia.)
No prospective trials have demonstrated the benefit of prophylactic use of myeloid growth factors in asymptomatic neutropenic MDS patients. Similarly, the use of prophylactic antibiotics in such patients is of uncertain benefit. While appropriate use of antibiotics in febrile patients is standard clinical practice, the benefit of myeloid growth factors in such settings is unknown.
The use of erythropoiesis-stimulating agents (ESAs) may improve anemia. The likelihood of response to exogenous erythropoietin administration is dependent on the pretreatment serum erythropoietin level and on baseline transfusion needs.
In a meta-analysis summarizing the data on erythropoietin in 205 patients with MDS from 17 studies, responses were most likely in patients who were anemic but who did not yet require a transfusion, patients who did not have ring sideroblasts, and patients who had a serum erythropoietin level lower than 200 u/L. Effective treatment requires substantially higher doses of erythropoietin than are used for other indications; the minimum effective dose studied is 60,000 IU per week. The use of high-dose darbepoetin (300 µg/dose weekly or 500 µg/dose every 2–3 weeks) has been reported to produce a major erythroid response rate of almost 50% in patients whose endogenous erythropoietin level was lower than 500 µ/mL.[5,6] Most studies discontinued ESAs in patients who failed to show hematologic improvement after 3 to 4 months of therapy. Average response duration is approximately 2 years.
One decision model found that the likelihood of responding to growth factors was higher in patients with a low serum erythropoietin level (defined as a level <500/µL) and low transfusion needs (defined as <2 units of packed red blood cells every month), but growth factors were rarely effective in patients with a high erythropoietin level and high transfusion needs. Some patients with poor response to erythropoietin alone may have improved response with the addition of low doses of granulocyte colony-stimulating factor (G-CSF) (0.5–1.0 µg/kg/day).[9,10,11] Rates of response to the combination treatment vary with classification, with responses more likely in patients with refractory anemia and ring sideroblasts (RARS) and less likely in patients with excess blasts. Patients with RARS are unlikely to respond to erythropoietin alone.