13. The Pathophysiology of Acute Promyelocytic Leukemia

  1. Stefan Faderl MD Associate Professor and
  2. Hagop Kantarjian MD Chairman Professor
  1. Francesco Lo-Coco MD and
  2. Syed Khizer Hasan PhD

Published Online: 4 JAN 2011

DOI: 10.1002/9781444327359.ch13

Leukemias: Principles and Practice of Therapy

Leukemias: Principles and Practice of Therapy

How to Cite

Lo-Coco, F. and Hasan, S. K. (2010) The Pathophysiology of Acute Promyelocytic Leukemia, in Leukemias: Principles and Practice of Therapy (eds S. Faderl and H. Kantarjian), Wiley-Blackwell, Oxford, UK. doi: 10.1002/9781444327359.ch13

Editor Information

  1. Department of Leukemia, University of Texas, MD Anderson Cancer Center, Houston, TX, USA

Author Information

  1. Department of Biopathology, University Tor Vergata, Rome, Italy

Publication History

  1. Published Online: 4 JAN 2011
  2. Published Print: 26 NOV 2010

ISBN Information

Print ISBN: 9781405182355

Online ISBN: 9781444327359

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Keywords:

  • acute promyelocytic leukemia;
  • PML-RARα;
  • all-trans retinoic acid (ATRA);
  • arsenic trioxide

Summary

Acute promyelocytic leukemia (APL) is a particular leukemia subset characterized by a unique genetic lesion, i.e. the PML-RARα fusion, and an exquisite response to differentiating agents. Until the late 1980s, APL was considered the most aggressive and rapidly fatal form of acute leukemia. Over the past two decades, important advances have been made into the understanding of APL pathogenesis, as well as in its treatment, such that it has nowadays been converted into the most frequently curable leukemia in adults. APL is regarded as a model disease for the innovative tailored treatment of human leukemia, including differentiation therapy and the use of chromatin remodeling agents and antibody-directed therapy. This chapter summarizes the pathophysiology of APL with special emphasis on its impact on targeted treatment.