Current Clinical Practice: Current Therapeutic Options in Myelodysplastic Syndromes

Abstract

Myelodysplastic syndromes (MDS) are characterized initially by ineffective hematopoiesis and subsequently the frequent development of acute myelogenous leukemias (AML). During the last 15 years, important progress has been made in the understanding of the biology and prognosis of myelodysplastic syndromes. Risk-adapted treatment strategies were established due to the high median age (60–75 years) of MDS-patients and the individual history of the disease (number of cytopenias, cytogenetical changes, transfusion requirements). The use of allogeneic bone marrow transplantation for MDS patients currently offers the only potentially curative treatment, but this treatment modality is not available for the most of the “typical” MDS-patients aged >60 years. Based on in-vitro findings analyzing the potential of several agents to differentiate or to stimulate hematopoietic progenitor cells a number of therapeutic options were evaluated in clinical trials: hematopoietic growth factors (e.g. erythropoietin, G-CSF), differentiation inducers (e.g. retinoids), or cytoprotective substances (amifostine). The role of immunsuppressive agents (antithymocyte globulin, cyclosporine A) either alone or in combination is being actively investigated. Using intensive cytotoxic treatment in patients with advanced MDS or AML after MDS complete remission rates comparable with those known from the treatment of de novo AML were reported. The therapy related toxicity (early death rate <10%) was reduced by using G-CSF given prior (“Priming”) and/or after the cytotoxic treatment.

Keywords:

• Myelodysplastic syndrome
• immunotherapy
• differentiation induction
• hematopoietic growth factors
• chemotherapy
• bone marrow transplantation