Abstract
Acute promyelocytic leukemia (APL), a distinct subtype of acute myeloid leukemia (AML), is characterized by a unique molecular aberration, the presence of circulating promyelocytes, and, frequently, a life-threatening coagulopathy. The outcome of the patients with APL has improved dramatically over the last 40 years. Discovery that in APL the (15;17) translocation is associated with rearrangement and fusion of retinoic acid receptor-α (RARα) and promyelocytic leukemia (PML) genes and the clinical observation that APL is uniquely sensitive to all-trans-retinoic acid (ATRA) provided a foundation for the further breakthroughs in understanding the mechanism of the disease. Development of sophisticated molecular techniques led to improved diagnosis and monitoring of patients with APL and provides an invaluable tool in guiding therapy. Multiple clinical trials have explored the optimal doses, schedules, and duration of chemo- and targeted therapy, with the goals of minimizing toxicity without sacrificing the outstanding outcomes now readily achieved in most patients. In this chapter we have summarized the basic mechanisms implicated in the pathogenesis of APL as well as the targeted therapeutic strategies that transformed APL from one of the most lethal acute leukemias to one that is highly curable.
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