First case of acute promyelocytic leukemia with TFG-RARA achieved complete remission treated with venetoclax and all-trans retinoic acid.

Abstract

Variant acute promyelocytic leukemia (vAPL) represents a certain type of APL case whose specific fusion proteins, which are relevant but atypical variants, may fail to be identified by polymerase chain reaction (PCR) and fluorescence in situ hybridization (FISH) and requires identification through next-generation sequencing (NGS) or RNA sequencing (RNA-seq). These patients often show insensitivity to arsenic trioxide (ATO) or all trans-retinoic acid (ATRA) and therefore exhibit unclear prognosis. Venetoclax (VEN), an oral small-molecule B-cell lymphoma 2 (BCL-2) inhibitor, demonstrates effectiveness and safety as a cytoreduction therapy for pediatric APL and has shown some promising effect on relapsed or refractory APL. However, only a few cases have been reported on the treatment of vAPL with a single drug or multiple drugs combined with VEN. Therefore, this study reported the first vAPL case with the TFG-RARA fusion gene, who achieved complete remission (CR) with oral administration of VEN and ATRA, and remained CR till submission. Our study indicated that VEN may have a good therapeutic effect and contribute to a better prognosis of vAPL and warranted further application among APL patients.