Abstract
Acute promyelocytic leukemia (APL) is a rare disease accounting for only 5%–10% of pediatric acute myeloid leukemia (AML) and fewer than 1000 cases occur annually in the United States across all age groups. Characterized by t (15; 17), with a resultant PML-RARA gene fusion driving leukemia development, advances in therapy have improved outcomes for APL significantly in the past several decades, now making APL the most curable form of AML in both children and adults. Cure rates in APL are now comparable to pediatric B-lymphoid leukemias. The success of APL treatment is due, in part, to the breadth of understanding of the driver PML-RARA mutation as well as collaborative efforts to quickly introduce and maximize the benefit of new therapies. Here, we review the presentation, clinical features, pathogenesis, and treatment advances in pediatric APL.
Highlights
Acute promyelocytic leukemia (APL) is a unique entity in acute myeloid malignancies typically characterized by the balanced translocation t (15; 17)(q24.1;q21.2) and resultant Promyelocytic leukemia (PML)-retinoic acid receptor alpha (RARA) fusion gene [1,2]
Due to the characterization of the PML-RARA gene caused by t (15; 17) and its effect on leukemia development, patients with APL benefit from a targeted therapeutic approach primarily utilizing differentiation therapy
This has allowed for significant advances in overall survival (OS) and event-free survival (EFS), making APL the most curable form of acute myeloid leukemia (AML) today
Summary
Acute promyelocytic leukemia (APL) is a unique entity in acute myeloid malignancies typically characterized by the balanced translocation t (15; 17)(q24.1;q21.2) and resultant PML-RARA fusion gene [1,2]. Some patients with APL still experience complications in this disease and treatment, with early death prior to or shortly after the initiation of therapy accounting for the majority of fatalities, in high-risk patients [5,6,7]. These early deaths are not typically included in EFS and OS rates as many patients die before they can be enrolled on a clinical trial, and, the actual survival rates of patients diagnosed with APL is lower than reported [8]. We will review the presentation, pathophysiology, and current treatment approaches to pediatric APL
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