TY - JOUR
T1 - Acute promyelocytic leukemia in children
T2 - A model of precision medicine and chemotherapy-free therapy
AU - Gurnari, Carmelo
AU - Voso, Maria Teresa
AU - Girardi, Katia
AU - Mastronuzzi, Angela
AU - Strocchio, Luisa
N1 - Funding Information:
We thank the ?American Italian Cancer Foundation? Fellowship program (to C.G.).
Publisher Copyright:
© 2021 by the authors. Licensee MDPI, Basel, Switzerland.
Copyright:
Copyright 2021 Elsevier B.V., All rights reserved.
PY - 2021/1/2
Y1 - 2021/1/2
N2 - 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.
AB - 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.
KW - Acute promyelocytic leukemia
KW - All-trans retinoic acid
KW - Arsenic trioxide
KW - Children
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U2 - 10.3390/ijms22020642
DO - 10.3390/ijms22020642
M3 - Review article
AN - SCOPUS:85099344294
VL - 22
SP - 1
EP - 13
JO - International Journal of Molecular Sciences
JF - International Journal of Molecular Sciences
SN - 1661-6596
IS - 2
M1 - 642
ER -