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RESEARCH PRODUCT

Prognostic Factors in APL

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The prognosis of acute promyelocytic leukemia (APL) has significantly improved since the introduction of anthracyclines, but especially after the advent of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). The identification of prognostic factors is a key process in clinical investigation, since their recognition allows the stratification of disease risks and promotes refined therapeutic adjustments. In fact, most treatments are now designed following risk-adapted strategies in order to optimize the therapeutic efficacy and minimizing side effects. The efficacy of treatment is one major determinant in prognosis. Therefore, the impact of a number of disease and patient characteristics on outcome should be analyzed according to their predictive value with the two main therapeutic approaches currently used for APL, i.e., ATRA plus chemotherapy-based and ATRA plus ATO-based therapy. The prognostic impact of WBC counts on induction response and the risk of relapse are universally accepted, regardless of the type of treatment used. A score defined by the GIMEMA and PETHEMA groups, based on the presenting WBC and platelet counts, is the most widely used for risk stratification in clinical trials and routine practice, because of its simplicity and accuracy. Other prognostic factors, including clinical and molecular markers, have been associated with outcome in some studies, but in general they have not been incorporated into decision-making algorithms, with the exception of age. This chapter includes a comprehensive review of patient- and disease-related prognostic factors, with special emphasis on those reported with state-of-the-art treatments.