Selection of therapy: rational decisions based on molecular events.

This article reviews to what extent molecular data can be used to rationalize therapeutic choices in the treatment of chronic myeloid leukemia. Two categories of data are discussed: markers that globally measure risk but do not provide a molecular rationale for therapy selection; and biomarkers with a causal link to a clinical phenotype, such as certain mutations of the BCR-ABL kinase domain. As therapy selection is still mainly based on clinical criteria, molecular biomarkers are discussed in the context of available clinical prognostication tools, focusing on biomarkers that do not reflect disease burden as a surrogate of responsiveness to treatment.