Acute promyelocytic leukemia in children: A model of precision medicine and chemotherapy-free therapy

Research output: Contribution to journalReview articlepeer-review

Abstract

Acute promyelocytic leukemia (APL) represents a paradigm of precision medicine. Indeed, in the last decades, the introduction of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) completely revolutionized the therapeutic approach to this previously highly fatal disorder. This entirely chemotherapy-free treatment, which provided excellent survival rates, has been initially validated in adults and, recently, translated in the pediatric setting. This review summarizes currently available data on the use of ATRA and ATO combination in pediatric APL, providing a particular focus on peculiar issues and challenges, such as the occurrence of pseudotumor cerebri and death during induction (early death), as well as the advantage offered by the ATO/ATRA combination in sparing long-term sequelae.

Original languageEnglish
Article number642
Pages (from-to)1-13
Number of pages13
JournalInternational Journal of Molecular Sciences
Volume22
Issue number2
DOIs
Publication statusPublished - Jan 2 2021

Keywords

  • Acute promyelocytic leukemia
  • All-trans retinoic acid
  • Arsenic trioxide
  • Children

ASJC Scopus subject areas

  • Catalysis
  • Molecular Biology
  • Spectroscopy
  • Computer Science Applications
  • Physical and Theoretical Chemistry
  • Organic Chemistry
  • Inorganic Chemistry

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