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.
Highlights
Acute promyelocytic leukemia (APL) represents a paradigm of precision medicine
white blood cells (WBC) count at diagnosis is the strongest predictor of outcome and relapse in APL, so that patients are stratified according to the Sanz-risk criteria as standard risk (SR) and high risk (HR), based on the presence of less or more than 10,000/μL WBC, respectively [43]
Post-remission arsenic trioxide (ATO) administration was completed over three years, and it is noteworthy that this study provided important insights into arsenic elimination kinetics [68,69]
Summary
Acute promyelocytic leukemia (APL) is a unique subtype of acute myeloid leukemia (AML) accounting for 5–10% of all pediatric AML cases [1]. A small percentage of cases presents different RARA fusion partners, such as nucleophosmin (NPM1, 5q35), signal transducer, and activator of transcription (STAT5B, 17q21), factor interacting with PAPOLA and CPSF1 (FIP1L1, 4q12), promyelocytic leukemia zinc finger (PLZF, known as ZBTB16, 11q23) and nuclear matrix-mitotic apparatus protein 1 (NUMA1, 11q13) [22,23,24,25,26]. These rare cases of “APL variant” are characterized by the same clinical picture of classic. WBC count at diagnosis is the strongest predictor of outcome and relapse in APL, so that patients are stratified according to the Sanz-risk criteria as standard risk (SR) and high risk (HR), based on the presence of less or more than 10,000/μL WBC, respectively [43]
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