Drug treatment options for acute promyelocytic leukemia

Expert Opin Pharmacother. 2022 Jan;23(1):117-127. doi: 10.1080/14656566.2021.1961744. Epub 2021 Aug 5.

Abstract

Introduction: Until the late 1980s, acute promyelocytic leukemia (APL) was the most rapidly fatal leukemia; however, nowadays, it is a curable disease with survival rates exceeding 90-95%. The improvement of APL outcome is mainly due to two agents, which target the typical translocation t(15;17) and its fusion transcript PML-RARα responsible for initiating and maintaining the disease: all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). The story of APL represents a pioneering model for the development of precision medicine and curative chemotherapy-free approaches for acute leukemia.

Area covered: The authors examine the major advances in the treatment of patients with APL focusing on three different eras: 1) the pre-ATRA era; 2) the ATRA era; 3) the ATO era.

Expert opinion: The combination of ATRA and ATO is effective and curative for the majority of APL patients. It has been approved for low/intermediate risk cases while an experimental trial with a minimal addition of chemotherapy for high-risk ones is ongoing. Disease relapse is infrequent and can be cured with ATRA-ATO rechallenging, with or without subsequent transplantation depending on the interval between complete remission and relapse. New therapeutic landscapes contemplate the use of an oral chemo-free ATRA-ATO combination, implementing treatment as outpatient care, thus increasing quality of life and decreasing medical costs.

Keywords: Acute promyelocytic leukemia; arsenic trioxide; chemo-free approach; oral arsenic derivatives; risk-adapted strategies.

MeSH terms

  • Antineoplastic Combined Chemotherapy Protocols / therapeutic use
  • Arsenicals* / therapeutic use
  • Humans
  • Leukemia, Promyelocytic, Acute* / drug therapy
  • Leukemia, Promyelocytic, Acute* / genetics
  • Oxides / therapeutic use
  • Pharmaceutical Preparations*
  • Quality of Life
  • Treatment Outcome

Substances

  • Arsenicals
  • Oxides
  • Pharmaceutical Preparations