Serendipity: decitabine monotherapy induced complete molecular response in a 77-year-old patient with acute promyelocytic leukemia

Abstract

Acute promyelocytic leukemia (APL) is an uncommon but very aggressive subtype of acute myeloid leukemia (AML), accounting for 10-15% of adult AML with 600-800 new cases per year in the United States. 1,2 Over 95% of cases involve translocation t(15;17)(q22;q21), which results in the promyelocytic leukemia retinoic acid receptor alpha (PML-RARa) rearrangement and fusion protein. 3 Most of the remaining APL cases harbor a fusion protein involving RARa and another partner. 4 The PML-RARa fusion gene initiates APL by blocking differentiation and increasing self-renewal and proliferation of leukemic progenitor cells.

Clinical Response to Venetoclax and Decitabine in Acute Promyelocytic Leukemia With a Novel RARA-THRAP3 Fusion: A Case Report

“…Interestingly, Liu et al reported a novel HNRNPC-RARA fusion in APL lacking PML-RARA rearrangement, sensitive to venetoclax-based therapy. Moreover, Abboud et al demonstrated that decitabine may be an efficacious and more tolerable alternative to elderly patient with low-risk AP therapy (13). The results give us some hints; combination therapy with venetoclax and decitabine may show a promising clinical efficacy in variant APLs.…”