Characteristics of the 83 patients with DDX41mut MDS or AML
| Modalities . | All (N = 83) . | AML (n = 64) . | MDS (n = 19) . |
|---|---|---|---|
| Age at diagnosis | |||
| Median (IQR) | 60.6 (53.7-64.3) | 60 (49.6-64.2) | 61.9 (59.6-64.3) |
| Age at HSCT | |||
| Median (IQR) | 61.63 (55-65.4) | 60.6 (50.2-64.7) | 64.6 (61.8-65.8) |
| Gender, n (%) | |||
| Female | 13 (15.7) | 10 (15.6) | 3 (15.8) |
| Male | 70 (84.3) | 54 (84.4) | 16 (84.2) |
| Disease status at HSCT, n (%) | |||
| CR1 | 63 (78.8) | 58 (93.5) | 5 (27.8) |
| CR2 | 4 (4.9) | 4 (6.5) | 0 (0) |
| CR (number unknown) | 3 (3.8) | 0 (0) | 3 (16.7) |
| Primary induction failure | 2 (2.5) | 0 (0) | 2 (11.1) |
| Improvement (no CR) | 1 (1.2) | 0 (0) | 1 (5.6) |
| Progressive | 2 (2.5) | 0 (0) | 2 (11.1) |
| Upfront HSCT | 5 (6.2) | 0 (0) | 5 (27.8) |
| Not available | 3 | 2 | 1 |
| Donor type, n (%) | |||
| MSD | 24 (28.9) | 17 (26.6) | 7 (36.8) |
| Matched other relative | 1 (1.2) | 1 (1.6) | 0 (0) |
| MMRD | 11 (13.3) | 8 (12.5) | 3 (15.8) |
| MUD 10/10 | 38 (45.8) | 30 (46.9) | 8 (42.1) |
| MMUD 9/10 | 5 (6) | 5 (7.8) | 0 (0) |
| UD (unknown mismatch) | 2 (2.4) | 2 (3.1) | 0 (0) |
| CBU | 2 (2.4) | 1 (1.6) | 1 (5.3) |
| Source of stem cells, n (%) | |||
| BM | 10 (12) | 10 (15.6) | 0 (0) |
| BM+PB | 2 (2.4) | 2 (3.1) | 0 (0) |
| PB | 69 (83.1) | 51 (79.7) | 18 (94.7) |
| Double CBU | 2 (2.4) | 1 (1.6) | 1 (5.3) |
| Myeloablative regimen, n (%) | |||
| No | 62 (74.7) | 46 (71.9) | 16 (84.2) |
| Yes | 21 (25.3) | 18 (28.1) | 3 (15.8) |
| TBI, n (%) | |||
| No | 71 (85.5) | 53 (82.8) | 18 (94.7) |
| Yes | 12 (14.5) | 11 (17.2) | 1 (5.3) |
| ATG, n (%) | |||
| No | 25 (30.1) | 20 (31.2) | 5 (26.3) |
| Yes | 58 (69.9) | 44 (68.8) | 14 (73.7) |
| GVHD prophylaxis, n (%) | |||
| No | 1 (1.2) | 1 (1.6) | 0 (0) |
| ATG only | 2 (2.4) | 1 (1.6) | 1 (5.3) |
| CSA | 13 (15.7) | 10 (15.6) | 3 (15.8) |
| CSA+MMF | 37 (44.6) | 25 (39.1) | 12 (63.2) |
| CSA+MTX | 18 (21.7) | 18 (28.1) | 0 (0) |
| MMF+TACRO | 1 (1.2) | 1 (1.6) | 0 (0) |
| PTCY+CSA+MMF | 12 (14.5) | 9 (14.1) | 3 (15.8) |
| Modalities . | All (N = 83) . | AML (n = 64) . | MDS (n = 19) . |
|---|---|---|---|
| Age at diagnosis | |||
| Median (IQR) | 60.6 (53.7-64.3) | 60 (49.6-64.2) | 61.9 (59.6-64.3) |
| Age at HSCT | |||
| Median (IQR) | 61.63 (55-65.4) | 60.6 (50.2-64.7) | 64.6 (61.8-65.8) |
| Gender, n (%) | |||
| Female | 13 (15.7) | 10 (15.6) | 3 (15.8) |
| Male | 70 (84.3) | 54 (84.4) | 16 (84.2) |
| Disease status at HSCT, n (%) | |||
| CR1 | 63 (78.8) | 58 (93.5) | 5 (27.8) |
| CR2 | 4 (4.9) | 4 (6.5) | 0 (0) |
| CR (number unknown) | 3 (3.8) | 0 (0) | 3 (16.7) |
| Primary induction failure | 2 (2.5) | 0 (0) | 2 (11.1) |
| Improvement (no CR) | 1 (1.2) | 0 (0) | 1 (5.6) |
| Progressive | 2 (2.5) | 0 (0) | 2 (11.1) |
| Upfront HSCT | 5 (6.2) | 0 (0) | 5 (27.8) |
| Not available | 3 | 2 | 1 |
| Donor type, n (%) | |||
| MSD | 24 (28.9) | 17 (26.6) | 7 (36.8) |
| Matched other relative | 1 (1.2) | 1 (1.6) | 0 (0) |
| MMRD | 11 (13.3) | 8 (12.5) | 3 (15.8) |
| MUD 10/10 | 38 (45.8) | 30 (46.9) | 8 (42.1) |
| MMUD 9/10 | 5 (6) | 5 (7.8) | 0 (0) |
| UD (unknown mismatch) | 2 (2.4) | 2 (3.1) | 0 (0) |
| CBU | 2 (2.4) | 1 (1.6) | 1 (5.3) |
| Source of stem cells, n (%) | |||
| BM | 10 (12) | 10 (15.6) | 0 (0) |
| BM+PB | 2 (2.4) | 2 (3.1) | 0 (0) |
| PB | 69 (83.1) | 51 (79.7) | 18 (94.7) |
| Double CBU | 2 (2.4) | 1 (1.6) | 1 (5.3) |
| Myeloablative regimen, n (%) | |||
| No | 62 (74.7) | 46 (71.9) | 16 (84.2) |
| Yes | 21 (25.3) | 18 (28.1) | 3 (15.8) |
| TBI, n (%) | |||
| No | 71 (85.5) | 53 (82.8) | 18 (94.7) |
| Yes | 12 (14.5) | 11 (17.2) | 1 (5.3) |
| ATG, n (%) | |||
| No | 25 (30.1) | 20 (31.2) | 5 (26.3) |
| Yes | 58 (69.9) | 44 (68.8) | 14 (73.7) |
| GVHD prophylaxis, n (%) | |||
| No | 1 (1.2) | 1 (1.6) | 0 (0) |
| ATG only | 2 (2.4) | 1 (1.6) | 1 (5.3) |
| CSA | 13 (15.7) | 10 (15.6) | 3 (15.8) |
| CSA+MMF | 37 (44.6) | 25 (39.1) | 12 (63.2) |
| CSA+MTX | 18 (21.7) | 18 (28.1) | 0 (0) |
| MMF+TACRO | 1 (1.2) | 1 (1.6) | 0 (0) |
| PTCY+CSA+MMF | 12 (14.5) | 9 (14.1) | 3 (15.8) |
BM, bone marrow; CBU, cord blood unit; CSA, ciclosporin A; IQR, interquartile range; MMF, mycophenolate mofetil; MMRD, mismatched related donor; MMUD, mismatched unrelated donor; MSD, matched sibling donor; MTX, methotrexate; PB, peripheral blood; RIC, reduced intensity conditioning; TACRO, tacrolimus; TBI, total body irradiation; TCD, T-cells depletion; UD, unrelated donor.