Patient demographics and baseline characteristics
| . | Pevonedistat+ azacitidine n = 227 . | Azacitidine alone n = 227 . |
|---|---|---|
| Higher-risk MDS | n = 161 | n = 163 |
| Median age, years (range) | 73 (36-87) | 74 (41-92) |
| Male/female, n (%) | 93 (58)/68 (42) | 103 (63)/60 (37) |
| Disease type, n (%) | ||
| De novo | 150 (93) | 154 (94) |
| Secondary | 9 (6) | 9 (6) |
| Missing/unknown | 2 (1) | 0 |
| WHO tumor classification, n (%) | ||
| Refractory anemia with excess blasts-1 | 50 (31) | 52 (32) |
| Refractory anemia with excess blasts-2 | 63 (39) | 77 (47) |
| CMML-2 | 1 (<1) | 2 (1) |
| Not available | 2 (1) | 1 (<1) |
| Missing data | 45 (28) | 31 (19) |
| IPSS-R category, n (%) | ||
| Intermediate risk | 38 (24) | 41 (25) |
| High risk | 60 (37) | 64 (39) |
| Very high risk | 63 (39) | 58 (36) |
| ECOG PS, n (%) | ||
| 0-1 | 145 (90) | 143 (88) |
| 2 | 14 (9) | 20 (12) |
| Missing data | 2 (1) | 0 |
| TP53 mutation yes/no, n (%) | 39 (24)/122 (76) | 35 (21)/128 (79) |
| Median modified Charlson Comorbidity Index, (range) | 1 (0-8) | 1 (0-9) |
| Median time from initial diagnosis, months (range) | 1.31 (0-98.7) | 1.61 (0.1-149.7) |
| Higher-risk CMML | n = 16 | n = 11 |
| Median age, years (range) | 69.5 (60-86) | 70 (61-85) |
| Male/female, n (%) | 10 (63)/6 (38) | 8 (73)/3 (27) |
| Disease type, n (%) | ||
| De novo | 15 (94) | 9 (82) |
| Secondary | 1 (6) | 2 (18) |
| WHO tumor classification, n (%) | ||
| CMML-1 | 12 (75) | 2 (18) |
| CMML-2 | 1 (6) | 6 (55) |
| Refractory anemia with excess blasts-1 | 1 (6) | 0 |
| Missing data | 2 (13) | 1 (9) |
| IPSS-R category, n (%) | ||
| Intermediate risk | 7 (44) | 3 (27) |
| High risk | 8 (50) | 4 (36) |
| Very high risk | 1 (6) | 4 (36) |
| ECOG PS, n (%) | ||
| 0-1 | 15 (94) | 9 (82) |
| 2 | 1 (6) | 2 (18) |
| TP53 mutation yes/no, n (%) | 2 (13)/14 (88) | 1 (9)/10 (91) |
| Median modified Charlson Comorbidity Index, (range) | 0 (0-6) | 2 (0-5) |
| Median time from initial diagnosis, months (range) | 4.04 (0.4-25.5) | 1.71 (0.3-55.7) |
| AML 20-30% blasts | n = 50 | n = 53 |
| Median age, years (range) | 75 (36-92) | 75 (54-92) |
| Male/female, n (%) | 29 (58)/21 (42) | 31 (58)/22 (42) |
| Disease type, n (%) | ||
| De novo | 31 (62) | 26 (49) |
| Secondary | 18 (36) | 27 (51) |
| Unknown | 1 (2) | 0 |
| Revised WHO classification, n (%) | ||
| AML with recurrent genetic abnormalities | 0 | 3 (6) |
| AML with myelodysplasia-related changes | 33 (66) | 38 (72) |
| Therapy-related AML | 1 (2) | 0 |
| AML not otherwise specified | 14 (28) | 11 (21) |
| Other | 2 (4) | 1 (2) |
| European LeukemiaNet risk classification, n (%) | ||
| Adverse | 30 (60) | 37 (70) |
| Intermediate | 1 (2) | 2 (4) |
| Indeterminate | 10 (20) | 8 (15) |
| Missing data | 9 (18) | 6 (11) |
| ECOG PS, n (%) | ||
| 0-1 | 43 (86) | 39 (74) |
| 2 | 7 (14) | 13 (25) |
| Missing data | 0 | 1 (2) |
| TP53 mutation yes/no, n (%) | 7 (14)/43 (86) | 11 (21)/42 (79) |
| Median time from initial diagnosis, months (range) | 0.72 (0.1-5.3) | 0.76 (0.1-10.7) |
| . | Pevonedistat+ azacitidine n = 227 . | Azacitidine alone n = 227 . |
|---|---|---|
| Higher-risk MDS | n = 161 | n = 163 |
| Median age, years (range) | 73 (36-87) | 74 (41-92) |
| Male/female, n (%) | 93 (58)/68 (42) | 103 (63)/60 (37) |
| Disease type, n (%) | ||
| De novo | 150 (93) | 154 (94) |
| Secondary | 9 (6) | 9 (6) |
| Missing/unknown | 2 (1) | 0 |
| WHO tumor classification, n (%) | ||
| Refractory anemia with excess blasts-1 | 50 (31) | 52 (32) |
| Refractory anemia with excess blasts-2 | 63 (39) | 77 (47) |
| CMML-2 | 1 (<1) | 2 (1) |
| Not available | 2 (1) | 1 (<1) |
| Missing data | 45 (28) | 31 (19) |
| IPSS-R category, n (%) | ||
| Intermediate risk | 38 (24) | 41 (25) |
| High risk | 60 (37) | 64 (39) |
| Very high risk | 63 (39) | 58 (36) |
| ECOG PS, n (%) | ||
| 0-1 | 145 (90) | 143 (88) |
| 2 | 14 (9) | 20 (12) |
| Missing data | 2 (1) | 0 |
| TP53 mutation yes/no, n (%) | 39 (24)/122 (76) | 35 (21)/128 (79) |
| Median modified Charlson Comorbidity Index, (range) | 1 (0-8) | 1 (0-9) |
| Median time from initial diagnosis, months (range) | 1.31 (0-98.7) | 1.61 (0.1-149.7) |
| Higher-risk CMML | n = 16 | n = 11 |
| Median age, years (range) | 69.5 (60-86) | 70 (61-85) |
| Male/female, n (%) | 10 (63)/6 (38) | 8 (73)/3 (27) |
| Disease type, n (%) | ||
| De novo | 15 (94) | 9 (82) |
| Secondary | 1 (6) | 2 (18) |
| WHO tumor classification, n (%) | ||
| CMML-1 | 12 (75) | 2 (18) |
| CMML-2 | 1 (6) | 6 (55) |
| Refractory anemia with excess blasts-1 | 1 (6) | 0 |
| Missing data | 2 (13) | 1 (9) |
| IPSS-R category, n (%) | ||
| Intermediate risk | 7 (44) | 3 (27) |
| High risk | 8 (50) | 4 (36) |
| Very high risk | 1 (6) | 4 (36) |
| ECOG PS, n (%) | ||
| 0-1 | 15 (94) | 9 (82) |
| 2 | 1 (6) | 2 (18) |
| TP53 mutation yes/no, n (%) | 2 (13)/14 (88) | 1 (9)/10 (91) |
| Median modified Charlson Comorbidity Index, (range) | 0 (0-6) | 2 (0-5) |
| Median time from initial diagnosis, months (range) | 4.04 (0.4-25.5) | 1.71 (0.3-55.7) |
| AML 20-30% blasts | n = 50 | n = 53 |
| Median age, years (range) | 75 (36-92) | 75 (54-92) |
| Male/female, n (%) | 29 (58)/21 (42) | 31 (58)/22 (42) |
| Disease type, n (%) | ||
| De novo | 31 (62) | 26 (49) |
| Secondary | 18 (36) | 27 (51) |
| Unknown | 1 (2) | 0 |
| Revised WHO classification, n (%) | ||
| AML with recurrent genetic abnormalities | 0 | 3 (6) |
| AML with myelodysplasia-related changes | 33 (66) | 38 (72) |
| Therapy-related AML | 1 (2) | 0 |
| AML not otherwise specified | 14 (28) | 11 (21) |
| Other | 2 (4) | 1 (2) |
| European LeukemiaNet risk classification, n (%) | ||
| Adverse | 30 (60) | 37 (70) |
| Intermediate | 1 (2) | 2 (4) |
| Indeterminate | 10 (20) | 8 (15) |
| Missing data | 9 (18) | 6 (11) |
| ECOG PS, n (%) | ||
| 0-1 | 43 (86) | 39 (74) |
| 2 | 7 (14) | 13 (25) |
| Missing data | 0 | 1 (2) |
| TP53 mutation yes/no, n (%) | 7 (14)/43 (86) | 11 (21)/42 (79) |
| Median time from initial diagnosis, months (range) | 0.72 (0.1-5.3) | 0.76 (0.1-10.7) |
WHO, World Health Organization.