Response Criteria

In the past, the treatment for the vast majority of patients with MDS consisted of supportive care to mitigate cytopenia-related complications, which involved mainly red blood cell (RBC) transfusions and growth factor use to avoid symptomatic anemia. Only patients who progressed to AML were treated more aggressively, usually with chemotherapy similar to that used to treat AML; these patients were then assessed using the same criteria as those to assess the response to therapy for patients with AML.

The algorithm used to treat patients with MDS has become much more complex recently for several reasons. First, reliable prognostic classification systems have been developed to best determine the natural history of MDS (discussed further below) and to identify different prognostic subgroups. Second, several therapies have been developed that influence outcome and QOL in each identifiable prognostic subgroup. Third, the responses are now evaluated according to the goals of each therapeutic approach. Thus, clinicians and investigators encounter greater variability in the definitions of the responses to MDS than in the past, making it difficult to interpret and compare results between clinical trials.

To resolve this variability in the response criteria, an international working group of investigators with expertise in MDS has achieved a consensus to establish standardized response criteria for clinical trials involving patients with MDS1-3 (Table 43.1). This working group looked for clinically relevant, practical, and

Table 43.1 Measurement of response/treatment effect in MDS

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