Alternatives to conventional or myeloablative chemotherapy in myelodysplastic

At present, the only 2 treatments that can prolong survival in patients wit h myelodysplastic syndrome (MDS) are allogeneic stem cell transplantation a nd intensive chemotherapy. Alternatives to myeloablative or conventional ch emotherapy include: (1) supportive therapy, (2) stimulation of normal resid ual hematopoietic progenitors, and (3) manipulation of myelodysplastic hema topoiesis. These alternative therapeutic strategies can be accomplished usi ng various therapeutic tools. Supportive therapy remains the mainstay in th e management of MDS patients and desferrioxamine should be administered to individuals who have a regular need for blood transfusion. The only hematop oietic growth factors that can be useful in the treatment of selected MDS p atients are recombinant human erythropoietin (rhEpo) and granulocyte colony -stimulating factor (G-CSF). Overall, 15% to 20% of patients with MDS respo nd to rhEpo treatment. Factors predicting response include serum erythropoi etin levels <100 to 200 mU/mL, low-risk MDS, and no or low need for transfu sion. G-CSF alone should be used only for short-term treatments during seve re infection episodes that do not respond to conventional therapy About 40% of MDS patients respond to a combined treatment of rhEpo plus G-CSF with a melioration of anemia. Cytoprotective anti-apoptotic agents such as amifost ine, alone or in combination, may improve blood values in occasional MDS pa tients. MDS patients with immunologically mediated myelosuppression may res pond favorably to antithymocyte globulin or cyclosporin A (CyA). Although r hEpo and CyA may be used in individual patients who appear likely to respon d, the remaining therapeutic tools must be considered strictly experimental ; phase III clinical trials are required to establish whether they can be u seful in the treatment of MDS patients. More generally, because of the curr ent uncertainties concerning MDS treatment, participation of patients in cl inical trials should be always encouraged. Int Hematol 2000;72:134-138, (C) 2000 The Japanese Society of Hematology.