Acute Promyelocytic Leukemia in Children: A Model of Precision Medicine and Chemotherapy-Free Therapy

International Journal of Molecular Sciences

MariaTeresa Voso¹  Angela Mastronuzzi²  Luisa Strocchio²  Carmelo Gurnari²  Katia Girardi² 

[1] Department of Biomedicine and Prevention, University of Rome Tor Vergata, 00133 Rome, Italy;Department of Pediatric Hemato-Oncology and Cell and Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico Bambino Gesù Children’s Hospital, 00165 Rome, Italy;

关键词: acute promyelocytic leukemia;    children;    arsenic trioxide;    all-trans retinoic acid;   

DOI  :  10.3390/ijms22020642

来源: DOAJ

【 摘 要 】

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.

【 授权许可】

Unknown