Abstract
Acute promyelocytic leukemia (APL) [M3 in the French-American-British (FAB) classification] is an uncommon, but distinctive, subtype of acute myeloid leukemia (AML) that has recently received increased attention. It warrants separate discussion because of its remarkable responsiveness to the vitamin A derivative, all-trans retinoic acid (ATRA). Evaluation of the role of ATRA in the treatment of patients with APL has fostered significant progress in our understanding of the molecular biology of leukemia. Rearrangement of the retinoic acid receptor α (RARα) gene locus (located on chromosome 17), as part of the balanced reciprocal translocation between chromosomes 15 and 17 characteristic of APL, represents the first direct link between a mutation in a nuclear receptor and human cancer. This suggests that abnormalities in other nuclear receptors may play a role in the pathogenesis of other human malignancies. Finally, the success of ATRA in APL represents the first example of effective differentiation therapy in human cancer and suggests that similar kinds of selective targeted treatment may be possible in other malignancies.
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Tallman, M.S. (1996). Clinical Aspects of Acute Promyelocytic Leukemia and Response to Retinoid Therapy. In: Vedeckis, W.V. (eds) Hormones and Cancer. Hormones in Health and Disease. Birkhäuser Boston. https://doi.org/10.1007/978-1-4612-4266-6_19
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