treatment

Last reviewed 01/2018

Treatment is mainly supportive involving transfusions of red packed cells and platelets to maintain peripheral blood counts and antibiotics for infections. Frequent transfusions over many years however, may lead to secondary haemochromatosis while allo-immunization may cause refractoriness to platelet transfusions and to multiple red cell allo - antibodies.

• Supportive care
  • supportive care consists of transfusions to correct anemia and thrombocytopenia, antibiotics, and haematopoietic growth factors such as erythropoietin (Epo) and G-CSF
    • supportive care is now the choice of treatment for indolent, low-risk MDS
    • Epo increases hematocrit approximately 20%, and together with low-dose G-CSF can improve anemia and reduce transfusion requirements in up to 40% of low-risk patients
• Curative therapy
  • stem cell transplant (SCT)
    • at present, the only chance of a cure is an allogeneic bone marrow transplant and all patients under 50years of age should be referred to a cancer centre offering bone marrow transplant. Currently the transplant related mortality (within 100 days post-transplant) is high at 30-40 per cent for sibling allografts and 60 per cent for unrelated donor transplants
    • disease free survival rates are approximately 30-50%, with the greatest benefit for those who may least need treatment -younger patients with low-risk MDS treated within one year of diagnosis
    • non-myeloablative allogeneic transplant may become an alternative for older patients or those at high risk for complications after SCT and relies on the graft-versus-leukemia effect of an allogeneic graft.
  • intensive chemotherapy
    • cytotoxic chemotherapy is poorly tolerated in the elderly with prolonged periods of pancytopenia associated with considerable morbidity and mortality. Chemotherapy is often used in younger patients with excess blasts since transformation to overt AML is imminent and complete remission may be obtained
    • cytotoxic chemotherapy includes AML-type regimens such as idarubicin and/or fludarabine plus cytarabine, as well as topotecan plus cytarabine with or without cyclophosphamide. These regimens produce remission rates of 40-60%
      • however, the median duration of response is only 10-12 months and only 10-15% of patients can be cured by this approach
      • limited to those who can tolerate intensive chemotherapy
        • factors that favorably affect outcome include age <55, normal karyotype
    • low-dose cytarabine offers brief and partial remission but is a potent myelosuppressant requiring careful haematologic monitoring

Less established therapies include growth factors such as recombinant granulocyte-macrophage colony stimulating factor (GM-CSF) to increase circulating granulocytes and so reduce infections. However, theis may accelerate transformation to AML since GM-CSF receptors are present on leukaemic blasts.