Table 3. Treatment of ‘young' CMML patients (age⩽65) with hypomethylating agents and their outcome stratified by their allogenic stem cell transplant status.
| Variables | All n=75 | CMML patients that underwent Allogenic transplant n=29 (39%) | CMML patients without transplant n=45 (61%) | P-value |
|---|---|---|---|---|
| 1. 5-Azacitidine n (%) | 45 (60%) | 20 (71%) | 25 (56%) | — |
| Age | 56 (30–65) | 52 (30–63) | 59 (44–65) | 0.0009 |
| Male (%) | 35 (78%) | 14 (70%) | 21 (84%) | 0.3 |
| WHO morphological subtype; n (%) | ||||
| CMML-1 | 35 (78%) | 16 (80%) | 19 (76%) | 0.7 |
| CMML-2 | 10 (22%) | 4 (20%) | 6 (24%) | — |
| ASXL1 mutated ‘N' evaluable=20 | 9 (45%) | 4 (50%) | 5 (42%) | 0.7 |
| SRSF2 mutated ‘N' evaluable=19 | 9 (47%) | 2 (22%) | 7 (70%) | 0.04 |
| Mayo Molecular Model risk categorization ‘N' evaluable=19 | ||||
| Low | 1 (5%) | 0 (0%) | 1 (9%) | — |
| Intermediate-1 | 8 (42%) | 4 (50%) | 4 (36%) | 0.3 |
| Intermediate-2 | 7 (37%) | 0 (0%) | 3 (27%) | — |
| High | 3 (16%) | 4 (50%) | 3 (27%) | — |
| Mayo-French cytogenetic stratification ‘N' evaluable=74 | ||||
| Low | 33 (73%) | 14 (70%) | 19 (76%) | 0.5 |
| Intermediate | 11 (24%) | 6 (30%) | 5 (20%) | — |
| High | 1 (2%) | 0 (0%) | 1 (4%) | — |
| Median cycles (range) | 5 (1–44) | 4 (2–44) | 6 (1–36) | 0.3 |
| Median duration of therapy in months (range) | 4.6 (0.1–32) | 3.8 (0.1–18) | 4.6 (0.1–32) | 0.6 |
| Best response ‘N' evaluable=40 | ||||
| HI | 5 (13%) | 2 (10%) | 3 (14%) | 0.4 |
| PR | 10 (25%) | 6 (32%) | 4 (19%) | — |
| CR | 3 (7%) | 0 (0%) | 3 (14%) | — |
| No response | 22 (55%) | 11 (58%) | 7 (33%) | — |
| Reason for discontinuation of therapy ‘N' evaluable=30 | ||||
| Patient choice | 1 (3%) | 0 (0%) | 1 (6%) | — |
| Intolerance | 3 (10%) | 0 (0%) | 3 (18%) | 0.01 |
| Progression | 14 (47%) | 7 (47%) | 7 (41%) | — |
| Bridge to transplant | 8 (27%) | 8 (53%) | 0 (0%) | — |
| Death | 4 (13%) | 0 (0%) | 4 (23%) | — |
| Second HMA n (%) | 5 (11%) | 3 (15%) | 2 (8%) | — |
| 2. Decitabine | 30 (40%) | 9 (31%) | 20 (68%) | — |
| Age | 58 (33–65) | 57 (43–65) | 59 (33–65) | 0.7 |
| Male (%) | 18 (60%) | 3 (37%) | 15 (75%) | 0.6 |
| WHO morphological subtype; n (%) | ||||
| CMML-1 | 21 (70%) | 5 (56%) | 16 (76%) | 0.3 |
| CMML-2 | 9 (30%) | 4 (44%) | 5 (24%) | — |
| ASXL1 mutated ‘N' evaluable=21 | 6 (28%) | 1 (20%) | 5 (31%) | 0.6 |
| SRSF2 mutated ‘N' evaluable=15 | 7 (47%) | 1 (33%) | 6 (54%) | 0.5 |
| Mayo Molecular Model risk categorization ‘N' evaluable=19 | ||||
| Low | 2 (10%) | 0 (0%) | 2 (14%) | 0.5 |
| Intermediate-1 | 6 (32%) | 1 (20%) | 5 (36%) | — |
| Intermediate-2 | 7 (37%) | 2 (40%) | 5 (36%) | — |
| High | 4 (21%) | 2 (40%) | 2 (14%) | — |
| Mayo-French cytogenetic risk stratification ‘N' evaluable=29 | ||||
| Low | 22 (76%) | 7 (78%) | 14 (74%) | 0.8 |
| Intermediate | 6 (21%) | 2 (22%) | 4 (21%) | — |
| High | 1 (3%) | 0 (0%) | 1 (5%) | — |
| Median number of cycles (range) | 6 (1–30) | 4 (1–12) | 7 (2–25) | 0.4 |
| Median duration of therapy in months (range) | 5.0 (0.1–49) | 4.2 (0.1–13) | 5.0 (1.0–49) | 0.1 |
| Best response ‘N' evaluable=20 | ||||
| HI | 2 (10%) | 1 (14%) | 1 (8%) | — |
| PR | 1 (5%) | 0 (0%) | 1 (8%) | 0.3 |
| CR | 6 (32%) | 1 (14%) | 5 (38%) | — |
| No response | 11 (55%) | 5 (71%) | 6 (46%) | — |
| Reason for discontinuation of therapy ‘n' evaluable=15 | ||||
| Progression | 9 (60%) | 2 (40%) | 7 (70%) | 0.03 |
| Bridge to transplant | 3 (20%) | 3 (60%) | 0 (0%) | — |
| Death | 3 (20%) | 0 (0%) | 3 (30%) | — |
| Second HMA n (%) | 2 (7%) | 0 (0%) | 2 (10%) | — |
Abbreviations: ASXL1, additional sex combs 1 gene; CMML, chronic myelomonocytic leukemia; CR, complete remission; HI, hematologic improvement; HMA, hypomethylating agent; PR, partial remission; SRSF2, serine/arginine-rich splicing factor 2; WHO, World Health Organization. Bold entries signify statistically significant P-values (i.e. P<0.05).