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Myelodysplasia – therapeutic response to novel therapy and the need for new diagnostic groups

Leukemia volume 17, pages 1183–1185 (2003)Cite this article

The rapid development of new treatment strategies directed against the varied clinical entities encompassed by the term myelodysplasia calls for new clinical and biologic descriptors. The term ‘myelodysplasia’ itself refers to a heterogeneous group of clonal hematopoietic disorders characterized by variable clinical features of hematologic insufficiency coupled with morphologic and cytogenetic bone marrow findings.1,2 Originally descriptive, diagnostic categories of refractory anemia and preleukemia were classified according to five subtypes based on morphologic criteria of peripheral blood and bone marrow.3 A specific criterion, dysplasia of the bone marrow, has been described as ‘disturbed differentiation of hematopoiesis’.2 The complex clinical heterogeneity of presentation, the presence or absence of erythropoietin-responsive anemia, neutropenia, and often severe thrombocytopenia were submitted to a classification system dependent on the assessment of blast percentage and abnormal localization of progenitors in the bone marrow with distinct findings in the peripheral blood. More recent classification systems have taken cytopenias and complex cytogenetic findings into consideration when assessing prognostic risk for progression to marrow failure or acute leukemia.4

A classification system based on marrow topography and blast percentage may never have been satisfactory to describe a complex set of clinical features ranging from megaloblastic hematopoiesis to paraneoplastic autoimmune abnormalities to overt leukemia.5 A system based on clinical risk assessment may also prove insufficient to identify patients likely to respond to a variety of exciting novel therapies, which may supplant allogeneic or autologous progenitor cell transplantation, treatments limited to a subset of young patients.6,7,8 New agents include differentiation agents, hematopoietins, and hypomethylation agents such as decitabine, and 5′-azacytidine.9,10,11,12,13,14 Like 5′-azacytidine, most clinical trials of active agents demonstrate a consistent response rate across histopathologic categories, thus speaking to the need for a new system to anticipate clinical response in biologically identified groups of patients.

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  1. Division of Hematology and Oncology, Department of Medicine, Los Angeles, CA, USA

    G J Schiller

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Schiller, G. Myelodysplasia – therapeutic response to novel therapy and the need for new diagnostic groups. Leukemia 17, 1183–1185 (2003). https://doi.org/10.1038/sj.leu.2402939

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