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Acute Promyelocytic Leukaemia

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Molecularly Targeted Therapy for Childhood Cancer

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Abstract

Since the time of its first description (Degos 2003), Acute Promyelocytic Leukaemia (APL) has drawn the attention of clinicians and scientists for its clinical and biological features (Degos 2003). APL is notable for distinctive clinical and biological characteristics, including its particular morphological features, the presence of potentially devastating hemorrhagic syndrome that is related to disseminated intravascular coagulopathy and abnormal fibrinolysis, and finally, the sensitivity to anthracycline-containing chemotherapy (Bernard et al. 1973). The availability of differentation therapy with All-Trans Retinoic Acid (ATRA), and more recently, the discovery of the beneficial effect of arsenic trioxide (ATO), together with the molecular characterization of the t(15;17) specific translocation (Grignani et al. 1994) have produced a remarkable improvement in patient outcome in the last decade. Most patients with APL are now cured (Sanz 2006). APL is a paradigm of a malignant disease that can be treated by cell modulation, using agents that act specifically on oncogenic, molecular events. This chapter reviews its impact in children APL.

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  1. Centro M. Tettamanti, Clinica Pediatrica Università Milano-Bicocca, Ospedale San Gerardo, Via Pergolesi, 33, 20052, Monza, Italy

    Andrea Biondi

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  2. Anna Maria Testi
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  3. Brenda E. S. Gibson
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Correspondence to Andrea Biondi .

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  1. Director, Center for Childhood Cancer, Nationwide Children’s Hospital, The Research Institute, 700 Children’s Drive 332, Columbus, 43205, Ohio, USA

    Peter J. Houghton

  2. Johns Hopkins The Bunting & Blaustein Ca, Medicine Kimmel Comprehensive Cancer Cen, Orleans Street 1650, Baltimore, 21231, Maryland, USA

    Robert J. Arceci

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Biondi, A., Testi, A.M., Gibson, B.E.S. (2010). Acute Promyelocytic Leukaemia. In: Houghton, P., Arceci, R. (eds) Molecularly Targeted Therapy for Childhood Cancer. Springer, New York, NY. https://doi.org/10.1007/978-0-387-69062-9_5

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