Journal
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Volume 22, Issue 2, Pages -Publisher
MDPI
DOI: 10.3390/ijms22020642
Keywords
acute promyelocytic leukemia; children; arsenic trioxide; all-trans retinoic acid
Funding
- American Italian Cancer Foundation Fellowship program
- American Italian Cancer Foundation Fellowship program
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The combination therapy of ATRA and ATO has shown high survival rates in pediatric APL patients, while also reducing the occurrence of long-term sequelae. However, there are also special issues and challenges such as pseudotumor cerebri and early death during induction in the treatment process.
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.
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