Cytogenetic and molecular characteristics of the treated pts
| . | Tamibarotene + azacitidine (N = 126) . | Placebo + azacitidine (N = 64) . | Total (N = 190) . |
|---|---|---|---|
| Cytogenetic risk status by IPSS-R, n (%) | |||
| Very good | 5 (3.97) | 1 (1.56) | 6 (3.16) |
| Good | 57 (45.24) | 27 (42.19) | 84 (44.21) |
| Intermediate | 17 (13.49) | 10 (15.63) | 27 (14.21) |
| Poor | 13 (10.32) | 9 (14.06) | 22 (11.58) |
| Very poor | 33 (26.19) | 16 (25.00) | 49 (25.79) |
| Missing | 1 (0.79) | 1 (1.56) | 2 (1.05) |
| Low hemoglobin (<11 g/dL) | |||
| Yes | 102 (80.95) | 53 (82.81) | 155 (81.58) |
| No | 24 (19.05) | 11 (17.19) | 35 (18.42) |
| Low platelets (<100 × 109/L) | |||
| Yes | 90 (71.43) | 46 (71.88) | 136 (71.58) |
| No | 36 (28.57) | 18 (28.13) | 54 (28.42) |
| Low absolute neutrophil count (<1.0 × 109/L) | |||
| Yes | 58 (46.03) | 26 (40.63) | 84 (44.21) |
| No | 67 (53.17) | 37 (57.81) | 104 (54.74) |
| Missing | 1 (0.79) | 1 (1.56) | 2 (1.05) |
| Molecular abnormalities for genes commonly associated with MDS∗ , n (%) | |||
| Participants with central NGS data | 76 (60.32) | 42 (65.63) | 118 (62.11) |
| ASXL1 mutated | 21 (27.63) | 14 (33.33) | 35 (29.66) |
| TET2 mutated | 21 (27.63) | 10 (23.81) | 31 (26.27) |
| TP53 mutated | 18 (23.68) | 8 (19.05) | 26 (22.03) |
| SRSF2 mutated | 11 (14.47) | 10 (23.81) | 21 (17.80) |
| RUNX1 mutated | 9 (11.84) | 8 (19.05) | 17 (14.41) |
| SF3B1 mutated | 11 (14.47) | 6 (14.29) | 17 (14.41) |
| STAG2 mutated | 7 (9.21) | 4 (9.52) | 11 (9.32) |
| DNMT3A mutated | 7 (9.21) | 3 (7.14) | 10 (8.47) |
| U2AF1 mutated | 6 (7.89) | 3 (7.14) | 9 (7.63) |
| U2AF1L5 mutated | 5 (6.58) | 3 (7.14) | 8 (6.78) |
| BCOR mutated | 6 (7.89) | 0 (0.00) | 6 (5.08) |
| CBL mutated | 3 (3.95) | 2 (4.76) | 5 (4.24) |
| IDH2 mutated | 4 (5.26) | 1 (2.38) | 5 (4.24) |
| NRAS mutated | 4 (5.26) | 1 (2.38) | 5 (4.24) |
| EZH2 mutated | 2 (2.63) | 2 (4.76) | 4 (3.39) |
| PPM1D mutated | 3 (3.95) | 1 (2.38) | 4 (3.39) |
| DDX41 mutated | 2 (2.63) | 1 (2.38) | 3 (2.54) |
| IDH1 mutated | 2 (2.63) | 1 (2.38) | 3 (2.54) |
| NF1 mutated | 2 (2.63) | 1 (2.38) | 3 (2.54) |
| SETBP1 mutated | 2 (2.63) | 1 (2.38) | 3 (2.54) |
| CEBPA mutated | 1 (1.32) | 1 (2.38) | 2 (1.69) |
| ETV6 mutated | 1 (1.32) | 1 (2.38) | 2 (1.69) |
| FLT3 mutated | 0 (0.00) | 2 (4.76) | 2 (1.69) |
| KRAS mutated | 0 (0.00) | 2 (4.76) | 2 (1.69) |
| PTPN11 mutated | 2 (2.63) | 0 (0.00) | 2 (1.69) |
| RB1 mutated | 1 (1.32) | 1 (2.38) | 2 (1.69) |
| WT1 mutated | 2 (2.63) | 0 (0.00) | 2 (1.69) |
| ZRSR2 mutated | 2 (2.63) | 0 (0.00) | 2 (1.69) |
| BRAF mutated | 1 (1.32) | 0 (0.00) | 1 (0.85) |
| GATA2 mutated | 1 (1.32) | 0 (0.00) | 1 (0.85) |
| JAK2 mutated | 1 (1.32) | 0 (0.00) | 1 (0.85) |
| SMC1A mutated | 0 (0.00) | 1 (2.38) | 1 (0.85) |
| ASXL1 wildtype/TET2 mutated | 14 (18.42) | 5 (11.90) | 19 (16.10) |
| ASXL1 wildtype/TET2 wildtype | 41 (53.95) | 23 (54.76) | 64 (54.24) |
| TP53 multi-hit | 6 (7.89) | 3 (7.14) | 9 (7.63) |
| TP53 single-hit∗ | 12 (15.79) | 5 (11.90) | 17 (14.41) |
| Number of mutated genes at baseline | |||
| 0 | 8 (10.53) | 7 (16.67) | 15 (12.71) |
| 1 | 16 (21.05) | 6 (14.29) | 22 (18.64) |
| 2 | 25 (32.89) | 9 (21.43) | 34 (28.81) |
| ≥3 | 27 (35.53) | 20 (47.62) | 47 (39.83) |
| . | Tamibarotene + azacitidine (N = 126) . | Placebo + azacitidine (N = 64) . | Total (N = 190) . |
|---|---|---|---|
| Cytogenetic risk status by IPSS-R, n (%) | |||
| Very good | 5 (3.97) | 1 (1.56) | 6 (3.16) |
| Good | 57 (45.24) | 27 (42.19) | 84 (44.21) |
| Intermediate | 17 (13.49) | 10 (15.63) | 27 (14.21) |
| Poor | 13 (10.32) | 9 (14.06) | 22 (11.58) |
| Very poor | 33 (26.19) | 16 (25.00) | 49 (25.79) |
| Missing | 1 (0.79) | 1 (1.56) | 2 (1.05) |
| Low hemoglobin (<11 g/dL) | |||
| Yes | 102 (80.95) | 53 (82.81) | 155 (81.58) |
| No | 24 (19.05) | 11 (17.19) | 35 (18.42) |
| Low platelets (<100 × 109/L) | |||
| Yes | 90 (71.43) | 46 (71.88) | 136 (71.58) |
| No | 36 (28.57) | 18 (28.13) | 54 (28.42) |
| Low absolute neutrophil count (<1.0 × 109/L) | |||
| Yes | 58 (46.03) | 26 (40.63) | 84 (44.21) |
| No | 67 (53.17) | 37 (57.81) | 104 (54.74) |
| Missing | 1 (0.79) | 1 (1.56) | 2 (1.05) |
| Molecular abnormalities for genes commonly associated with MDS∗ , n (%) | |||
| Participants with central NGS data | 76 (60.32) | 42 (65.63) | 118 (62.11) |
| ASXL1 mutated | 21 (27.63) | 14 (33.33) | 35 (29.66) |
| TET2 mutated | 21 (27.63) | 10 (23.81) | 31 (26.27) |
| TP53 mutated | 18 (23.68) | 8 (19.05) | 26 (22.03) |
| SRSF2 mutated | 11 (14.47) | 10 (23.81) | 21 (17.80) |
| RUNX1 mutated | 9 (11.84) | 8 (19.05) | 17 (14.41) |
| SF3B1 mutated | 11 (14.47) | 6 (14.29) | 17 (14.41) |
| STAG2 mutated | 7 (9.21) | 4 (9.52) | 11 (9.32) |
| DNMT3A mutated | 7 (9.21) | 3 (7.14) | 10 (8.47) |
| U2AF1 mutated | 6 (7.89) | 3 (7.14) | 9 (7.63) |
| U2AF1L5 mutated | 5 (6.58) | 3 (7.14) | 8 (6.78) |
| BCOR mutated | 6 (7.89) | 0 (0.00) | 6 (5.08) |
| CBL mutated | 3 (3.95) | 2 (4.76) | 5 (4.24) |
| IDH2 mutated | 4 (5.26) | 1 (2.38) | 5 (4.24) |
| NRAS mutated | 4 (5.26) | 1 (2.38) | 5 (4.24) |
| EZH2 mutated | 2 (2.63) | 2 (4.76) | 4 (3.39) |
| PPM1D mutated | 3 (3.95) | 1 (2.38) | 4 (3.39) |
| DDX41 mutated | 2 (2.63) | 1 (2.38) | 3 (2.54) |
| IDH1 mutated | 2 (2.63) | 1 (2.38) | 3 (2.54) |
| NF1 mutated | 2 (2.63) | 1 (2.38) | 3 (2.54) |
| SETBP1 mutated | 2 (2.63) | 1 (2.38) | 3 (2.54) |
| CEBPA mutated | 1 (1.32) | 1 (2.38) | 2 (1.69) |
| ETV6 mutated | 1 (1.32) | 1 (2.38) | 2 (1.69) |
| FLT3 mutated | 0 (0.00) | 2 (4.76) | 2 (1.69) |
| KRAS mutated | 0 (0.00) | 2 (4.76) | 2 (1.69) |
| PTPN11 mutated | 2 (2.63) | 0 (0.00) | 2 (1.69) |
| RB1 mutated | 1 (1.32) | 1 (2.38) | 2 (1.69) |
| WT1 mutated | 2 (2.63) | 0 (0.00) | 2 (1.69) |
| ZRSR2 mutated | 2 (2.63) | 0 (0.00) | 2 (1.69) |
| BRAF mutated | 1 (1.32) | 0 (0.00) | 1 (0.85) |
| GATA2 mutated | 1 (1.32) | 0 (0.00) | 1 (0.85) |
| JAK2 mutated | 1 (1.32) | 0 (0.00) | 1 (0.85) |
| SMC1A mutated | 0 (0.00) | 1 (2.38) | 1 (0.85) |
| ASXL1 wildtype/TET2 mutated | 14 (18.42) | 5 (11.90) | 19 (16.10) |
| ASXL1 wildtype/TET2 wildtype | 41 (53.95) | 23 (54.76) | 64 (54.24) |
| TP53 multi-hit | 6 (7.89) | 3 (7.14) | 9 (7.63) |
| TP53 single-hit∗ | 12 (15.79) | 5 (11.90) | 17 (14.41) |
| Number of mutated genes at baseline | |||
| 0 | 8 (10.53) | 7 (16.67) | 15 (12.71) |
| 1 | 16 (21.05) | 6 (14.29) | 22 (18.64) |
| 2 | 25 (32.89) | 9 (21.43) | 34 (28.81) |
| ≥3 | 27 (35.53) | 20 (47.62) | 47 (39.83) |
NGS, next-generation sequencing; WHO, World Health Organization.
Time since initial/high-risk diagnosis = (date of randomization - date of initial/high-risk diagnosis of MDS + 1) / 30.4375.
Participants may be counted in multiple categories. Mutation counts based on central NGS data.