Demographic and clinical characteristics of patients at baseline
| Characteristic . | All patients, N = 340 (100%) . | MDS-CMML, N = 173 (51%) . | MPN-CMML, N = 167 (49%) . | P value∗ . |
|---|---|---|---|---|
| Age at diagnosis, median (range) | 71 (17-88) | 71 (17-88) | 71 (38-86) | .478 |
| Sex, n (%) | .712 | |||
| Male | 235 (69.1) | 118 (68.2) | 117 (70.1) | |
| Female | 105 (30.9) | 55 (31.8) | 50 (29.9) | |
| Race, n (%) | .735 | |||
| White | 303 (89.9) | 152 (88.9) | 151 (91.0) | |
| Black | 14 (4.2) | 9 (5.3) | 5 (3.0) | |
| Hispanic | 11 (3.3) | 6 (3.5) | 5 (3.0) | |
| Other | 9 (2.7) | 4 (2.3) | 5 (3.0) | |
| WHO category, n (%) | .036 | |||
| CMML-1 | 298 (87.7) | 158 (91.3) | 140 (83.8) | |
| CMML-2 | 42 (12.3) | 15 (8.7) | 27 (16.2) | |
| FAB category, n (%) | <.001 | |||
| MDS-CMML | 173 (51) | 173 (100) | 0 (0.0) | |
| MPN-CMML | 167 (49) | 0 (0.0) | 167 (100) | |
| Indolent CMML, n (%) | .140 | |||
| Indolent CMML | 55 (16.2) | 33 (9.1) | 22 (13.2) | |
| Nonindolent CMML | 285 (83.8) | 140 (80.9) | 145 (86.8) | |
| Laboratory values, median (range) | ||||
| WBC, ×109/L | 12.6 (2.4-288.6) | 7.0 (2.4-12.8) | 25.1 (13-288.6) | <.001 |
| ANC, ×109/L | 6.1 (0.1-155.6) | 3.2 (0.1-9.8) | 13.5 (1.6-155.6) | <.001 |
| ALC, ×109/L | 2.2 (0.4-39.9) | 1.7 (0.4-4.7) | 3.3 (0.5-22.0) | <.001 |
| AMC, ×109/L | 2.4 (0.3-39.9) | 1.4 (0.3-6.4) | 4.2 (1.0-39.9) | <.001 |
| IMC, % | 1.0% (0%-37%) | 0.0% (0%-21%) | 3.0% (0%-37%) | <.001 |
| Circulating blasts, % | 0.0% (0%-12%) | 0.0% (0%-12%) | 0.0% (0%-14%) | <.001 |
| Hgb, g/dL | 11.1 (3.4-18.8) | 11.3 (5.7-18.8) | 11.0 (3.4-16.2) | .116 |
| Platelets, ×109/L | 102 (2-730) | 92 (5-712) | 108 (2-1945) | .170 |
| Serum iron, μg/dL | 85 (11-304) | 71 (38-86) | 86 (19-304) | .207 |
| Elevated LDH, n (%) | <.001 | |||
| Yes | 176 (53.0) | 68 (40.7) | 108 (65.5) | |
| No | 156 (47.0) | 99 (59.3) | 57 (34.5) | |
| Ferritin >1000 μg/L, n (%) | .260 | |||
| Yes | 51 (17.7) | 23 (15.2) | 28 (20.3) | |
| No | 238 (82.3) | 128 (84.8) | 110 (79.7) | |
| Circulating blasts, n (%) | <.001 | |||
| Yes | 87 (25.7) | 24 (13.9) | 63 (38.0) | |
| No | 252 (74.3) | 149 (86.1) | 103 (62.0) | |
| Cytopenias, n (%) | .040 | |||
| None | 99 (29.1) | 42 (24.3) | 57 (34.1) | |
| 1 line | 174 (51.2) | 59 (51.4) | 85 (50.9) | |
| Bicytopenia | 64 (18.8) | 39 (22.5) | 25 (15.0) | |
| Pancytopenia | 3 (0.9) | 3 (1.8) | 0 (0.0) | |
| Marrow blast burden, median (range) | 3 (0-16) | 2 (0-16) | 3 (0-17) | .115 |
| Marrow cellularity, median (range) | 80 (30-100) | 75 (30-100) | 90 (30-100) | .79 |
| RBC-TD, n (%) | .323 | |||
| Yes | 64 (18.8) | 29 (16.8) | 35 (21.0) | |
| No | 276 (81.2) | 144 (83.2) | 132 (79.0) | |
| PLT-TD, n (%) | .950 | |||
| Yes | 12 (3.5) | 6 (3.5) | 6 (3.6) | |
| No | 328 (96.5) | 167 (96.5) | 161 (96.4) | |
| Splenomegaly, n (%) | .010 | |||
| Yes | 61 (18.0) | 22 (12.7) | 39 (23.5) | |
| No | 278 (82.0) | 151 (87.3) | 127 (76.5) | |
| Cytogenetics | ||||
| CPSS karyotype risk, n (%) | .063 | |||
| Good | 261 (77.4) | 139 (80.8) | 122 (73.9) | |
| Intermediate | 37 (11.0) | 20 (11.6) | 17 (10.3) | |
| Poor | 39 (11.6) | 13 (7.6) | 26 (15.8) | |
| IPSS karyotype category, n (%) | .299 | |||
| Good | 264 (78.3) | 139 (80.8) | 125 (75.8) | |
| Intermediate | 53 (15.7) | 26 (15.1) | 27 (16.4) | |
| Poor | 20 (6.0) | 7 (4.1) | 13 (7.8) | |
| Risk assessment models | ||||
| IPSS-R, n (%) | .628 | |||
| Very Low | 95 (28.1) | 54 (31.2) | 41 (24.7) | |
| Low | 137 (40.4) | 67 (38.7) | 70 (42.2) | |
| Intermediate | 57 (16.8) | 30 (17.3) | 27 (16.3) | |
| High | 39 (11.5) | 17 (9.9) | 22 (13.3) | |
| Very High | 11 (3.2) | 5 (2.9) | 6 (3.5) | |
| IPSS-M, n (%) | .003 | |||
| Very Low | 24 (7.1) | 18 (10.4) | 6 (3.6) | |
| Low | 99 (29.1) | 60 (34.7) | 39 (23.4) | |
| Moderate Low | 72 (21.2) | 37 (21.4) | 35 (20.8) | |
| Moderate High | 54 (15.9) | 25 (14.4) | 29 (17.4) | |
| High | 61 (17.9) | 24 (13.9) | 37 (22.2) | |
| Very High | 30 (8.8) | 9 (5.2) | 21 (12.6) | |
| CPSS, n (%) | <.001 | |||
| Low | 118 (34.7) | 115 (66.5) | 3 (1.8) | |
| Intermediate-1 | 125 (36.8) | 36 (20.8) | 89 (53.3) | |
| Intermediate-2 | 80 (23.5) | 21 (12.1) | 59 (35.3) | |
| High | 17 (5.0) | 1 (0.6) | 16 (9.6) | |
| CPSS-Mol, n (%) | <.001 | |||
| Low | 53 (15.6) | 53 (30.6) | 0 (0.0) | |
| Intermediate-1 | 69 (20.3) | 48 (27.8) | 21 (12.6) | |
| Intermediate-2 | 137 (40.3) | 60 (34.7) | 77 (46.1) | |
| High | 81 (23.8) | 12 (6.9) | 69 (23.3) | |
| 4-point IPSS-M, n (%) | .001 | |||
| Low | 123 (36.2) | 78 (45.1) | 45 (26.9) | |
| Intermediate-1 | 72 (21.1) | 37 (21.4) | 35 (21.0) | |
| Intermeidate-2 | 54 (15.9) | 25 (14.4) | 29 (17.4) | |
| High | 91 (26.8) | 33 (19.1) | 58 (34.7) | |
| Mayo Molecular Model, n (%) | <.001 | |||
| Low | 35 (10.3) | 23 (13.4) | 12 (7.2) | |
| Intermediate-1 | 98 (28.9) | 66 (38.4) | 32 (19.2) | |
| Intermediate-2 | 109 (32.2) | 55 (31.9) | 54 (32.3) | |
| High | 97 (28.6) | 28 (16.3) | 69 (41.3) | |
| GFM model, n (%) | <.001 | |||
| Low | 146 (43.1) | 126 (73.3) | 20 (11.9) | |
| Intermediate | 124 (36.6) | 38 (22.1) | 86 (51.5) | |
| High | 69 (20.3) | 8 (4.6) | 61 (36.5) | |
| Clinical end points | ||||
| AML transformation, n (%) | .017 | |||
| Yes | 75 (22.1) | 29 (16.8) | 46 (27.5) | |
| No | 265 (77.9) | 144 (83.2) | 121 (72.5) | |
| AML at 1 year, n (%) | .065 | |||
| Yes | 29 (8.5) | 10 (5.8) | 19 (11.4) | |
| No | 311 (91.5) | 163 (94.2) | 148 (88.6) | |
| AML at 2 years, n (%) | .012 | |||
| Yes | 54 (15.9) | 19 (11.0) | 35 (21.0) | |
| No | 286 (84.1) | 154 (89.0) | 132 (79.0) | |
| AML at 4 years, n (%) | .014 | |||
| Yes | 69 (20.3) | 26 (15.0) | 43 (25.7) | |
| No | 271 (79.7) | 147 (85.0) | 124 (74.3) | |
| Allogeneic SCT, n (%) | 39 (11.5) | 14 (8.1) | 25 (15.0) | .047 |
| OS, median (range), y | 3.2 (2.7-3.7) | 4.1 (3.2-5.1) | 2.3 (1.9-2.7) | <.001 |
| Characteristic . | All patients, N = 340 (100%) . | MDS-CMML, N = 173 (51%) . | MPN-CMML, N = 167 (49%) . | P value∗ . |
|---|---|---|---|---|
| Age at diagnosis, median (range) | 71 (17-88) | 71 (17-88) | 71 (38-86) | .478 |
| Sex, n (%) | .712 | |||
| Male | 235 (69.1) | 118 (68.2) | 117 (70.1) | |
| Female | 105 (30.9) | 55 (31.8) | 50 (29.9) | |
| Race, n (%) | .735 | |||
| White | 303 (89.9) | 152 (88.9) | 151 (91.0) | |
| Black | 14 (4.2) | 9 (5.3) | 5 (3.0) | |
| Hispanic | 11 (3.3) | 6 (3.5) | 5 (3.0) | |
| Other | 9 (2.7) | 4 (2.3) | 5 (3.0) | |
| WHO category, n (%) | .036 | |||
| CMML-1 | 298 (87.7) | 158 (91.3) | 140 (83.8) | |
| CMML-2 | 42 (12.3) | 15 (8.7) | 27 (16.2) | |
| FAB category, n (%) | <.001 | |||
| MDS-CMML | 173 (51) | 173 (100) | 0 (0.0) | |
| MPN-CMML | 167 (49) | 0 (0.0) | 167 (100) | |
| Indolent CMML, n (%) | .140 | |||
| Indolent CMML | 55 (16.2) | 33 (9.1) | 22 (13.2) | |
| Nonindolent CMML | 285 (83.8) | 140 (80.9) | 145 (86.8) | |
| Laboratory values, median (range) | ||||
| WBC, ×109/L | 12.6 (2.4-288.6) | 7.0 (2.4-12.8) | 25.1 (13-288.6) | <.001 |
| ANC, ×109/L | 6.1 (0.1-155.6) | 3.2 (0.1-9.8) | 13.5 (1.6-155.6) | <.001 |
| ALC, ×109/L | 2.2 (0.4-39.9) | 1.7 (0.4-4.7) | 3.3 (0.5-22.0) | <.001 |
| AMC, ×109/L | 2.4 (0.3-39.9) | 1.4 (0.3-6.4) | 4.2 (1.0-39.9) | <.001 |
| IMC, % | 1.0% (0%-37%) | 0.0% (0%-21%) | 3.0% (0%-37%) | <.001 |
| Circulating blasts, % | 0.0% (0%-12%) | 0.0% (0%-12%) | 0.0% (0%-14%) | <.001 |
| Hgb, g/dL | 11.1 (3.4-18.8) | 11.3 (5.7-18.8) | 11.0 (3.4-16.2) | .116 |
| Platelets, ×109/L | 102 (2-730) | 92 (5-712) | 108 (2-1945) | .170 |
| Serum iron, μg/dL | 85 (11-304) | 71 (38-86) | 86 (19-304) | .207 |
| Elevated LDH, n (%) | <.001 | |||
| Yes | 176 (53.0) | 68 (40.7) | 108 (65.5) | |
| No | 156 (47.0) | 99 (59.3) | 57 (34.5) | |
| Ferritin >1000 μg/L, n (%) | .260 | |||
| Yes | 51 (17.7) | 23 (15.2) | 28 (20.3) | |
| No | 238 (82.3) | 128 (84.8) | 110 (79.7) | |
| Circulating blasts, n (%) | <.001 | |||
| Yes | 87 (25.7) | 24 (13.9) | 63 (38.0) | |
| No | 252 (74.3) | 149 (86.1) | 103 (62.0) | |
| Cytopenias, n (%) | .040 | |||
| None | 99 (29.1) | 42 (24.3) | 57 (34.1) | |
| 1 line | 174 (51.2) | 59 (51.4) | 85 (50.9) | |
| Bicytopenia | 64 (18.8) | 39 (22.5) | 25 (15.0) | |
| Pancytopenia | 3 (0.9) | 3 (1.8) | 0 (0.0) | |
| Marrow blast burden, median (range) | 3 (0-16) | 2 (0-16) | 3 (0-17) | .115 |
| Marrow cellularity, median (range) | 80 (30-100) | 75 (30-100) | 90 (30-100) | .79 |
| RBC-TD, n (%) | .323 | |||
| Yes | 64 (18.8) | 29 (16.8) | 35 (21.0) | |
| No | 276 (81.2) | 144 (83.2) | 132 (79.0) | |
| PLT-TD, n (%) | .950 | |||
| Yes | 12 (3.5) | 6 (3.5) | 6 (3.6) | |
| No | 328 (96.5) | 167 (96.5) | 161 (96.4) | |
| Splenomegaly, n (%) | .010 | |||
| Yes | 61 (18.0) | 22 (12.7) | 39 (23.5) | |
| No | 278 (82.0) | 151 (87.3) | 127 (76.5) | |
| Cytogenetics | ||||
| CPSS karyotype risk, n (%) | .063 | |||
| Good | 261 (77.4) | 139 (80.8) | 122 (73.9) | |
| Intermediate | 37 (11.0) | 20 (11.6) | 17 (10.3) | |
| Poor | 39 (11.6) | 13 (7.6) | 26 (15.8) | |
| IPSS karyotype category, n (%) | .299 | |||
| Good | 264 (78.3) | 139 (80.8) | 125 (75.8) | |
| Intermediate | 53 (15.7) | 26 (15.1) | 27 (16.4) | |
| Poor | 20 (6.0) | 7 (4.1) | 13 (7.8) | |
| Risk assessment models | ||||
| IPSS-R, n (%) | .628 | |||
| Very Low | 95 (28.1) | 54 (31.2) | 41 (24.7) | |
| Low | 137 (40.4) | 67 (38.7) | 70 (42.2) | |
| Intermediate | 57 (16.8) | 30 (17.3) | 27 (16.3) | |
| High | 39 (11.5) | 17 (9.9) | 22 (13.3) | |
| Very High | 11 (3.2) | 5 (2.9) | 6 (3.5) | |
| IPSS-M, n (%) | .003 | |||
| Very Low | 24 (7.1) | 18 (10.4) | 6 (3.6) | |
| Low | 99 (29.1) | 60 (34.7) | 39 (23.4) | |
| Moderate Low | 72 (21.2) | 37 (21.4) | 35 (20.8) | |
| Moderate High | 54 (15.9) | 25 (14.4) | 29 (17.4) | |
| High | 61 (17.9) | 24 (13.9) | 37 (22.2) | |
| Very High | 30 (8.8) | 9 (5.2) | 21 (12.6) | |
| CPSS, n (%) | <.001 | |||
| Low | 118 (34.7) | 115 (66.5) | 3 (1.8) | |
| Intermediate-1 | 125 (36.8) | 36 (20.8) | 89 (53.3) | |
| Intermediate-2 | 80 (23.5) | 21 (12.1) | 59 (35.3) | |
| High | 17 (5.0) | 1 (0.6) | 16 (9.6) | |
| CPSS-Mol, n (%) | <.001 | |||
| Low | 53 (15.6) | 53 (30.6) | 0 (0.0) | |
| Intermediate-1 | 69 (20.3) | 48 (27.8) | 21 (12.6) | |
| Intermediate-2 | 137 (40.3) | 60 (34.7) | 77 (46.1) | |
| High | 81 (23.8) | 12 (6.9) | 69 (23.3) | |
| 4-point IPSS-M, n (%) | .001 | |||
| Low | 123 (36.2) | 78 (45.1) | 45 (26.9) | |
| Intermediate-1 | 72 (21.1) | 37 (21.4) | 35 (21.0) | |
| Intermeidate-2 | 54 (15.9) | 25 (14.4) | 29 (17.4) | |
| High | 91 (26.8) | 33 (19.1) | 58 (34.7) | |
| Mayo Molecular Model, n (%) | <.001 | |||
| Low | 35 (10.3) | 23 (13.4) | 12 (7.2) | |
| Intermediate-1 | 98 (28.9) | 66 (38.4) | 32 (19.2) | |
| Intermediate-2 | 109 (32.2) | 55 (31.9) | 54 (32.3) | |
| High | 97 (28.6) | 28 (16.3) | 69 (41.3) | |
| GFM model, n (%) | <.001 | |||
| Low | 146 (43.1) | 126 (73.3) | 20 (11.9) | |
| Intermediate | 124 (36.6) | 38 (22.1) | 86 (51.5) | |
| High | 69 (20.3) | 8 (4.6) | 61 (36.5) | |
| Clinical end points | ||||
| AML transformation, n (%) | .017 | |||
| Yes | 75 (22.1) | 29 (16.8) | 46 (27.5) | |
| No | 265 (77.9) | 144 (83.2) | 121 (72.5) | |
| AML at 1 year, n (%) | .065 | |||
| Yes | 29 (8.5) | 10 (5.8) | 19 (11.4) | |
| No | 311 (91.5) | 163 (94.2) | 148 (88.6) | |
| AML at 2 years, n (%) | .012 | |||
| Yes | 54 (15.9) | 19 (11.0) | 35 (21.0) | |
| No | 286 (84.1) | 154 (89.0) | 132 (79.0) | |
| AML at 4 years, n (%) | .014 | |||
| Yes | 69 (20.3) | 26 (15.0) | 43 (25.7) | |
| No | 271 (79.7) | 147 (85.0) | 124 (74.3) | |
| Allogeneic SCT, n (%) | 39 (11.5) | 14 (8.1) | 25 (15.0) | .047 |
| OS, median (range), y | 3.2 (2.7-3.7) | 4.1 (3.2-5.1) | 2.3 (1.9-2.7) | <.001 |
ALC, absolute lymphocyte count; ANC, absolute neutrophil count; Hgb, hemoglobin; IMC, immature myeloid cell; FAB, French-American-British; SCT, stem cell transplantation.
Statistically significant results (P < .05) are shown in bold.
Fisher exact test; Wilcoxon rank-sum test.