The myelodysplastic syndromes (MDS) have heterogeneous clinical characteristics, morphologic features, cytogenetics, and clinical outcomes that can make management of patients with these disorders difficult. This clinical and prognostic heterogeneity can complicate the planning of therapy. The different therapeutic goals for patients with MDS vary according to age, performance status, the severity of cytopenia and its associated complications, risk of progression to acute myeloid leukemia (AML), and other factors, such as socioeconomic status.

The clinical objectives may differ according to the patient's age or other factors. For example, older patients or those with poor physical health might be unable to withstand aggressive treatment compared to younger patients in better health who can more easily overcome the complications associated with high-intensity therapies. Similarly, the therapeutic goals also differ depending on the patient's risk of progressing to AML. In general, the main objectives for low-risk patients are to improve cytopenias and their associated complications, and quality of life (QOL); in contrast, the main goal for high-risk patients, especially younger patients, is to achieve complete remission (CR) and then to eradicate the malignant clone through chemotherapy and possibly stem cell transplantation.

Because a variety of strategies can be used to treat patients with MDS according to prognosis and chance of a cure, and because the goals of treatment may vary, physicians should evaluate the responses in the context of these goals. Therefore, a consensus should be reached on the use of reliable MDS prognostic classification schemes and on standardized response criteria when designing clinical trials and evaluating different treatment options.

This chapter reviews the state of the art on the assessment of responses to treatment, prognosis, and follow-up of patients with MDS.

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