Characteristics of the study cohort
| Parameters . | Values . |
|---|---|
| Number of patients | 449 |
| Age at enrollment (median [range])--y | 72 (16-91) |
| Sex (%) | |
| Male | 304 (67.7) |
| Female | 145 (32.3) |
| WHO−classification at enrollment (%) | |
| MDS | 384 (85.5) |
| Isolated del(5q) | 5 (1.1) |
| MDS-SLD | 2 (0.4) |
| MDS-MLD | 37 (8.2) |
| MDS-RS | 4 (0.9) |
| MDS−U | 4 (0.9) |
| MDS-EB1 | 149 (33.2) |
| MDS-EB2 | 183 (40.8) |
| AML | 36 (8.0) |
| MDS/MPN | 29 (6.5) |
| Atypical CML, BCR-ABL1-negative | 1 (0.2) |
| CMML | 23 (5.1) |
| MDS/MPN−U | 5 (1.1) |
| IPSS-R number (%)∗ | |
| Very low | 4 (0.9) |
| Low | 19 (4.4) |
| Intermediate | 95 (22.1) |
| High | 126 (29.4) |
| Very high | 185 (43.1) |
| IPSS-M number (%)∗ | |
| Very low | 2 (0.5) |
| Low | 20 (4.7) |
| Moderate low | 26 (6.1) |
| Moderate high | 40 (9.4) |
| High | 109 (25.5) |
| Very high | 230 (53.9) |
| Karyotype risks (IPSS-R−based) number (%) | |
| Very good | 8 (1.8) |
| Good | 184 (41.1) |
| Intermediate | 78 (17.4) |
| Poor | 54 (12.1) |
| Very poor | 124 (27.7) |
| Peripheral blood | |
| WBC (median [range]) × 109/L | 2.7 (0.3−107.9) |
| ANC (median [range]) × 109/L | 1.0 (0−38.2) |
| HB (median [range]) g/dL | 9.1 (4.1−15) |
| PLT (median [range]) × 109/L | 71.0 (5−1237) |
| Bone marrow | |
| Blast (median [range])--% | 9.8 (0-30) |
| Post azacitidine samples available (%) | 289 (64.4) |
| RBC transfusion dependent (%) | 212 (47.2) |
| PLT transfusion dependent (%) | 48 (10.7) |
| Response (best response) | |
| CR (%) | 72 (18.2) |
| mCR (%) | 75 (18.9) |
| PR (%) | 21 (5.3) |
| SD (%) | 142 (35.9) |
| PD (%) | 86 (21.7) |
| Cases underwent transplantation (%) | 74 (16.5) |
| Parameters . | Values . |
|---|---|
| Number of patients | 449 |
| Age at enrollment (median [range])--y | 72 (16-91) |
| Sex (%) | |
| Male | 304 (67.7) |
| Female | 145 (32.3) |
| WHO−classification at enrollment (%) | |
| MDS | 384 (85.5) |
| Isolated del(5q) | 5 (1.1) |
| MDS-SLD | 2 (0.4) |
| MDS-MLD | 37 (8.2) |
| MDS-RS | 4 (0.9) |
| MDS−U | 4 (0.9) |
| MDS-EB1 | 149 (33.2) |
| MDS-EB2 | 183 (40.8) |
| AML | 36 (8.0) |
| MDS/MPN | 29 (6.5) |
| Atypical CML, BCR-ABL1-negative | 1 (0.2) |
| CMML | 23 (5.1) |
| MDS/MPN−U | 5 (1.1) |
| IPSS-R number (%)∗ | |
| Very low | 4 (0.9) |
| Low | 19 (4.4) |
| Intermediate | 95 (22.1) |
| High | 126 (29.4) |
| Very high | 185 (43.1) |
| IPSS-M number (%)∗ | |
| Very low | 2 (0.5) |
| Low | 20 (4.7) |
| Moderate low | 26 (6.1) |
| Moderate high | 40 (9.4) |
| High | 109 (25.5) |
| Very high | 230 (53.9) |
| Karyotype risks (IPSS-R−based) number (%) | |
| Very good | 8 (1.8) |
| Good | 184 (41.1) |
| Intermediate | 78 (17.4) |
| Poor | 54 (12.1) |
| Very poor | 124 (27.7) |
| Peripheral blood | |
| WBC (median [range]) × 109/L | 2.7 (0.3−107.9) |
| ANC (median [range]) × 109/L | 1.0 (0−38.2) |
| HB (median [range]) g/dL | 9.1 (4.1−15) |
| PLT (median [range]) × 109/L | 71.0 (5−1237) |
| Bone marrow | |
| Blast (median [range])--% | 9.8 (0-30) |
| Post azacitidine samples available (%) | 289 (64.4) |
| RBC transfusion dependent (%) | 212 (47.2) |
| PLT transfusion dependent (%) | 48 (10.7) |
| Response (best response) | |
| CR (%) | 72 (18.2) |
| mCR (%) | 75 (18.9) |
| PR (%) | 21 (5.3) |
| SD (%) | 142 (35.9) |
| PD (%) | 86 (21.7) |
| Cases underwent transplantation (%) | 74 (16.5) |
ANC, absolute neutrophil count; CML, chronic myeloid leukemia; CMML, chronic myelomonocytic leukemia; HB, hemoglobine; MDS/MPN-U, Myelodysplastic/myeloproliferative neoplasms unclassifiable; MDS-EB1/2, MDS with excess blasts 1/2; MDS-MLD, MDS with multilineage dysplasia; MDS-RS, refractory anemia with ring sideroblasts; MDS-SLD, MDS with single lineage dysplasia; MDS-U, MDS unclassifiable; PD, progressive disease; PLT, platelet; PR, partial remission; SD, stable disease; WBC, white blood cell.
IPSS-R and IPSS-M were not calculated for CMML cases with WBC ≥ 12 × 109/L.