Prolonged Response of a Patient with Relapsed Acute Myeloid Leukemia to a Novel Oral Bromodomain Extraterminal Inhibitor (BETi)

Table 1

Clinical course, including bone marrow, molecular, and peripheral blood responses.

Day	Circulating blasts (%)	BM blast morphology (%)	MLFS achieved	MRD flow blasts (%)	Cytogenetics	Molecular	ANC (K/μL)	Hgb (g/dL)	Plt (K/μL)	ELN response
Screening mono (M)	6	21	N/A	Not done	46,XX [20]	STAG2 R213Efster12 (VAF 18%)	0.12	10.6	95	N/A
C2D1M	3	40	No	Not done	46,XX [18]	Not done	0.12	8	13	Resistant disease
C3D1M	0	11	No	Not done	Failed	Not done	0.76	7.5	23	Resistant disease
C5D1M	0	8	No	Not done	46,XX [20]	Not done	1.2	9	12	Resistant disease
C6D1M	0	4	Yes	20.1	46,XX [20]	Not done	1.34	8.9	4	CRi
C7D1M	0	4	Yes	10.8	46,XX [20]	Not done	3.3	8.2	10	CRi
C9D1M	0	8	No	12.1	46,XX [20]	Not done	2.5	10.9	28	Morphologic relapse
C10D1M	0	4	Yes	Not done	46,XX [20]	Not done	3.1	8.6	43	CRi
C11D1M	0	8	No	13.5	46,XX [20]	STAG2 R213Efster12 (VAF 6.6%), WT1 K467Ter (VAF 7.4%)	2.7	11.4	33	Morphologic relapse
C13D1M	0	14	No	26.3	46,XX [20]	Not done	1.9	10.6	24	Resistant disease
Screening combo (C)	Not reported	8	N/A	Not done	46,XX [20]	Not done	>1.0	9.3	29	N/A
C2D1C	0	2	Yes	0.99	46,XX [20]	Not done	1.1	11.9	51	CRi
C3D1C	0	2	Yes	0.64	46,XX [20]	Not done	1.8	12.8	68	CRi
C5D1C	0	1	Yes	0.35	46,XX [20]	None detected	1.3	12	20	CRi
C7D1C	0	2	Yes	0.14	46,XX [20]	Not done	1.5	12.2	30	CRi
C9D1C	0	1	Yes	0.34	46,XX [20]	Not done	3.5	12.9	104	CR
C11D1C	0	N/A	N/A	0.9	46,XX [20]	None detected	3	13.7	51	CRi
C13D1C	0	6	No	5.3	46,XX [20]	Not done	4.4	13.3	42	Morphologic relapse
C14D1C	0	4	Yes	5.5	46,XX [20]	KMT2A I3423V (VUS, VAF 50.6%)	2.9	10.2	24	CRi
C15D1C	0	10	No	10.7	46,XX [20]	Not done	1.4	11.5	50	Morphologic relapse
EOT marrow deferred							2.6	11.7	75

BM, bone marrow; MLFS, morphologic leukemia-free state; MRD, measurable residual disease; VAF, variant allele frequency; ANC, absolute neutrophil count; Hgb, hemoglobin; Plt, platelet; ELN, European LeukemiaNet; CRi, complete remission with incomplete count recovery; CR, complete remission; N/A, not applicable; EOT, end of treatment; VUS, variant of uncertain significance.