2020
DOI: 10.1159/000508658
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Sustained Complete Remission with Incomplete Hematologic Recovery (CRi) in a Patient with Relapsed AML and Concurrent BCR-ABL1 and CBFB Rearrangement Treated with a Combination of Venetoclax and 5-Azacytidine

Abstract: The co-occurrence of BCR-ABL1 fusion and core-binding factor (CBF) rearrangements is uncommonly reported in AML. Although CBF rearrangements carry a favorable prognosis, the coexistence of BCR-ABL1 is associated with aggressive disease suggesting a potential advantage of high-intensity chemotherapy in association with tyrosine kinase inhibitors. Herein, we describe a refractory AML patient harboring BCR-ABL1 fusion and CBFB rearrangement that was successfully treated with a combination of venetoclax and hypome… Show more

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Cited by 4 publications
(3 citation statements)
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“…The study VIALE-A demonstrated that association of azacitidine with BCL2 inhibitor venetoclax improves patients OS, leading to the recent approval of this combination for newly diagnosed AML in intensive chemotherapy ineligible patients [ 32 ]. Nevertheless, CBF-AML were also excluded from this study, and no clue is currently available for the efficacy of azacitidine + venetoclax combination in this AML subgroup, with the exception of one case report [ 56 ]. HMA may also be associated with other drugs whose anti-leukemic activity in CBF-AML have been suggested, such as gemtuzumab-ozogamicin [ 26 , 57 ], or tyrosine-kinase inhibitors dasatinib [ 58 , 59 ] or midostaurin [ 60 ].…”
Section: Discussionmentioning
confidence: 99%
“…The study VIALE-A demonstrated that association of azacitidine with BCL2 inhibitor venetoclax improves patients OS, leading to the recent approval of this combination for newly diagnosed AML in intensive chemotherapy ineligible patients [ 32 ]. Nevertheless, CBF-AML were also excluded from this study, and no clue is currently available for the efficacy of azacitidine + venetoclax combination in this AML subgroup, with the exception of one case report [ 56 ]. HMA may also be associated with other drugs whose anti-leukemic activity in CBF-AML have been suggested, such as gemtuzumab-ozogamicin [ 26 , 57 ], or tyrosine-kinase inhibitors dasatinib [ 58 , 59 ] or midostaurin [ 60 ].…”
Section: Discussionmentioning
confidence: 99%
“…Over the past couple of decades, significant effort has gone into understanding the biological basis of blood cancers. This accelerated the development and use of drug cocktails in clinical oncology [1][2][3][4][5][6][7][8][9]. The general guidelines for using such cocktails largely remain focused on combining (a) drugs with different mechanisms of action to counter multidrug resistance and (b) drugs with non-overlapping toxicities for administration at near-maximal dose [10][11][12][13].…”
Section: Introductionmentioning
confidence: 99%
“…The co-occurrence of CBFB and BCR-ABL1 rearrangements in AML represents a distinct subgroup with unique clinical and molecular features. To date, there have been less than 30 cases reported in the literature [1,[5][6][7][8][9][10][11][12][13][14][15][16][17][18][19][20][21][22][23][24]. This intriguing association, while rare, poses a unique diagnostic and therapeutic challenge, as the treatment strategies for CML and AML markedly differ.…”
mentioning
confidence: 99%