Current Management of Newly Diagnosed Acute Promyelocytic Leukemia

Ann Oncol. 2016 Aug;27(8):1474-81. doi: 10.1093/annonc/mdw171. Epub 2016 Apr 15.


The management of acute promyelocytic leukemia (APL) has considerably evolved during the past two decades. The advent of all-trans retinoic acid (ATRA) and its inclusion in combinatorial regimens with anthracycline chemotherapy has provided cure rates exceeding 80%; however, this widely adopted approach also conveys significant toxicity including severe myelosuppression and rare occurrence of secondary leukemias. More recently, the advent of arsenic trioxide (ATO) and its use in association with ATRA with or without chemotherapy has further improved patient outcome by allowing to minimize the intensity of chemotherapy, thus reducing serious toxicity while maintaining high anti-leukemic efficacy. The advantage of ATRA-ATO over ATRA chemotherapy has been recently demonstrated in two large randomized trials and this option has now become the new standard of care in low-risk (i.e. non-hyperleukocytic) patients. In light of its rarity, abrupt onset and high risk of early death and due to specific treatment requirements, APL remains a challenging condition that needs to be managed in highly experienced centers. We review here the results of large clinical studies conducted in newly diagnosed APL as well as the recommendations for appropriate diagnosis, prevention and management of the main complications associated with modern treatment of the disease.

Keywords: PML-RARA; acute promyelocytic leukemia; all-trans retinoic acid; arsenic trioxide.

Publication types

  • Review

MeSH terms

  • Anthracyclines / adverse effects
  • Anthracyclines / therapeutic use*
  • Antineoplastic Combined Chemotherapy Protocols / adverse effects
  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use*
  • Arsenic Trioxide
  • Arsenicals / adverse effects
  • Arsenicals / therapeutic use
  • Drug-Related Side Effects and Adverse Reactions / drug therapy
  • Drug-Related Side Effects and Adverse Reactions / pathology
  • Humans
  • Leukemia, Promyelocytic, Acute / drug therapy*
  • Leukemia, Promyelocytic, Acute / epidemiology
  • Leukemia, Promyelocytic, Acute / pathology
  • Oxides / adverse effects
  • Oxides / therapeutic use
  • Randomized Controlled Trials as Topic
  • Tretinoin / adverse effects
  • Tretinoin / therapeutic use*


  • Anthracyclines
  • Arsenicals
  • Oxides
  • Tretinoin
  • Arsenic Trioxide