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.

Gurnari, C., Voso, M.t., Girardi, K., Mastronuzzi, A., Strocchio, L. (2021). Acute Promyelocytic Leukemia in Children: A Model of Precision Medicine and Chemotherapy-Free Therapy. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 22(2), 1-13 [10.3390/ijms22020642].

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

Gurnari, Carmelo;Voso, Maria Teresa;
2021-01-11

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.
11-gen-2021
Pubblicato
Rilevanza internazionale
Articolo
Esperti anonimi
Settore MED/15 - MALATTIE DEL SANGUE
English
acute promyelocytic leukemia
all-trans retinoic acid
arsenic trioxide
children
Gurnari, C., Voso, M.t., Girardi, K., Mastronuzzi, A., Strocchio, L. (2021). Acute Promyelocytic Leukemia in Children: A Model of Precision Medicine and Chemotherapy-Free Therapy. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 22(2), 1-13 [10.3390/ijms22020642].
Gurnari, C; Voso, Mt; Girardi, K; Mastronuzzi, A; Strocchio, L
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2108/266791
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