Long-term follow-up of cytogenetically normal CEBPA-mutated AML

Table 1 Therapy and outcome

	All patients (N = 88)		moCEBPA(N = 43)		biCEBPA(N = 45)		P
	N	%	N	%	N	%
Number of induction cycles
1	29	33	16	37	13	30	0.41
2	59	67	27	63	32	71
Induction therapy regimen							0.48
In study	76	86	36	84	40	89
In analogy to study	12	14	7	16	5	11
Allogeneic SCT	19	22	10	23	9	20	0.75
in 1.CR	7	8	3	7	4	9
Primary refractory	4	5	3	7	1	2
At relapse	8	9	4	9	4	9
Early death until day 30	4	5	3	7	1	2	0.28
Early death until day 60	7	8	4	9	3	7	0.65
CR/CRi*	67	76	30	70	37	82	0.17
Median OS, 95% CI, years	3.0 (0.9-5.2)		1.7 (0.7-2.7)		9.6 (NA)		0.008
Median RFS, 95% CI, years	2.3 (1.0-3.7)		1.5 (0.4-2.5)		9.4 (NA)		0.021

*62 patients have achieved a CR; 5 patients have achieved a CRi;
Abbreviations: biCEBPA biallelic mutation in the CCAAT/enhancer-binding protein alpha, CR complete remission, CRi CR with incomplete revovery, moCEBPA monoallelic mutation in the CCAAT/enhancer-binding protein alpha, N number, NA not applicable, SCT stem cell transplantation.

ISSN: 1756-8722