Studies reporting clinical outcome of karyotypically normal AML patients according to the CEBPA mutational status
| Prognostic significance . | Independent prognostic factor on MVA . | AML type . | No. of pts (no. with/no. without alteration)* . | Age range, y (median) . | Median follow-up . | Differences in pretreatment features . | References . |
|---|---|---|---|---|---|---|---|
| CEBPA+ versus CEBPA− | |||||||
| Rates of CR, resistant disease, and early or hypoplastic death: no significant differences | — | De novo and secondary | 236 (36/200) | 16-60 (47/47)† | 30 mo | Higher hemoglobin level, lower platelet counts, higher percentage of PB blasts, less lymphadenopathy, less extramedullary involvement | Fröhling et al19 |
| OS: significantly longer for CEBPA+ pts (median, not reached vs 19 mo; P = .05) | Yes | 236 (36/200) | |||||
| CRD: significantly longer for CEBPA+ pts (median, not reached vs 26 mo; P = .01) | Yes | 187 (31/156) | |||||
| CR rate: no significant difference (100% vs 82%) | — | De novo (84%), secondary (16%) | 67 (12/55) | 18-71 (46/49)† | 38 mo | Lower LDH, higher CD7 | Bienz et al46 |
| OS: significantly longer for CEBPA+ pts (median, 45.5 vs 12 mo; P < .001) | Yes | 67 (12/55) | |||||
| DFS: significantly longer for CEBPA+ pts (median, 33.5 vs 10 mo; P = .002) | Yes | 57 (12/45) | |||||
| CR rate: no significant difference | — | De novo | 106 (20/86) | 17-65 (44) | NR | NR | Boissel et al37 |
| OS: no significant difference (6-year OS rates, 53% vs 38%) | — | 106 (20/86) | |||||
| EFS: significantly longer for CEBPA+ pts (P = .01) | No | 106 (20/86) | |||||
| CEBPA N-terminal nonsense mutations versus other CEBPA mutations versus CEBPA wild-type | |||||||
| CRD: the longest for pts with CEBPA N-terminal nonsense mutations, the shortest for pts with CEBPA-WT (median, not reached vs 52 mo vs 26 mo; P = .04) | ND | De novo (88%), secondary (12%) | 187 | 16-60 | 30 mo | FAB M1/M2 more common in pts with N-terminal mutations than in those with other mutations | Fröhling et al19 |
| Prognostic significance . | Independent prognostic factor on MVA . | AML type . | No. of pts (no. with/no. without alteration)* . | Age range, y (median) . | Median follow-up . | Differences in pretreatment features . | References . |
|---|---|---|---|---|---|---|---|
| CEBPA+ versus CEBPA− | |||||||
| Rates of CR, resistant disease, and early or hypoplastic death: no significant differences | — | De novo and secondary | 236 (36/200) | 16-60 (47/47)† | 30 mo | Higher hemoglobin level, lower platelet counts, higher percentage of PB blasts, less lymphadenopathy, less extramedullary involvement | Fröhling et al19 |
| OS: significantly longer for CEBPA+ pts (median, not reached vs 19 mo; P = .05) | Yes | 236 (36/200) | |||||
| CRD: significantly longer for CEBPA+ pts (median, not reached vs 26 mo; P = .01) | Yes | 187 (31/156) | |||||
| CR rate: no significant difference (100% vs 82%) | — | De novo (84%), secondary (16%) | 67 (12/55) | 18-71 (46/49)† | 38 mo | Lower LDH, higher CD7 | Bienz et al46 |
| OS: significantly longer for CEBPA+ pts (median, 45.5 vs 12 mo; P < .001) | Yes | 67 (12/55) | |||||
| DFS: significantly longer for CEBPA+ pts (median, 33.5 vs 10 mo; P = .002) | Yes | 57 (12/45) | |||||
| CR rate: no significant difference | — | De novo | 106 (20/86) | 17-65 (44) | NR | NR | Boissel et al37 |
| OS: no significant difference (6-year OS rates, 53% vs 38%) | — | 106 (20/86) | |||||
| EFS: significantly longer for CEBPA+ pts (P = .01) | No | 106 (20/86) | |||||
| CEBPA N-terminal nonsense mutations versus other CEBPA mutations versus CEBPA wild-type | |||||||
| CRD: the longest for pts with CEBPA N-terminal nonsense mutations, the shortest for pts with CEBPA-WT (median, not reached vs 52 mo vs 26 mo; P = .04) | ND | De novo (88%), secondary (12%) | 187 | 16-60 | 30 mo | FAB M1/M2 more common in pts with N-terminal mutations than in those with other mutations | Fröhling et al19 |
MVA indicates multivariable analysis; pts, patients; CEBPA+, patients with mutations of the CEBPA gene; CEBPA−, patients without mutations of the CEBPA gene; CR, complete remission; OS, overall survival; CRD, CR duration; DFS, disease-free survival; EFS, event-free survival; —, no significant difference in univariable analysis (MVA not performed); PB, blood; LDH, serum lactate dehydrogenase level; NR, not reported; ND, not done; and FAB, French-American-British.
Numbers of patients for whom clinical data were available.
Median age for patients with CEBPA mutations/patients without CEBPA mutations.