Patient characteristics
| . | TMD07 . | ML-DS . | . | Historical control . | . | |||
|---|---|---|---|---|---|---|---|---|
| . | N (of) . | % . | N (of) . | % . | P . | N (of) . | % . | P . |
| Total | 102 | 100 | 17 | 100 | 146 | 100 | ||
| Elevated WBC | 17 (102) | 17 | 4 (17) | 24 | .476 | 21 (128) | 16 | .148 |
| Elevated aspartate transaminase | 17 (87) | 20 | 2 (17) | 12 | .506 | 27 (96) | 28 | .225 |
| Elevated alanine transaminase | 26 (90) | 29 | 4 (17) | 24 | .769 | 23 (96) | 24 | .507 |
| Pathologic coagulation | 20 (54) | 37 | 3 (9) | 33 | 1.000 | 23 (104) | 22 | .059 |
| Hydrops fetalis | 7 (96) | 7 | 1 (17) | 6 | 1.000 | 7 (146) | 5 | .416 |
| Pleural effusion | 6 (94) | 6 | 0 (17) | 0 | .588 | 24 (146) | 16 | .027 |
| Pericardial effusion | 19 (95) | 20 | 2 (17) | 12 | .510 | 17 (146) | 12 | .096 |
| Ascites | 17 (98) | 17 | 3 (17) | 18 | 1.000 | 12 (146) | 8 | .042 |
| Splenomegaly | 28 (97) | 29 | 5 (17) | 29 | 1.000 | 44 (104) | 42 | .056 |
| Hepatomegaly | 43 (97) | 44 | 9 (17) | 53 | .592 | 62 (104) | 60 | .035 |
| Cholestasis | 21 (91) | 23 | 2 (16) | 13 | .344 | 16 (104) | 15 | .202 |
| Liver dysfunction* | 30 (97) | 31 | 5 (17) | 29 | 1.000 | 21 (104) | 20 | .105 |
| Intensive care | 41 (75) | 55 | 7 (11) | 64 | .745 | 37 (146) | 25 | <.001 |
| Ventilation required | 30 (76) | 39 | 4 (11) | 36 | 1.000 | 30 (104) | 29 | .152 |
| Cardiac defects | 69 (100) | 69 | 13 (17) | 76 | .573 | 68 (146) | 47 | <.001 |
| . | TMD07 . | ML-DS . | . | Historical control . | . | |||
|---|---|---|---|---|---|---|---|---|
| . | N (of) . | % . | N (of) . | % . | P . | N (of) . | % . | P . |
| Total | 102 | 100 | 17 | 100 | 146 | 100 | ||
| Elevated WBC | 17 (102) | 17 | 4 (17) | 24 | .476 | 21 (128) | 16 | .148 |
| Elevated aspartate transaminase | 17 (87) | 20 | 2 (17) | 12 | .506 | 27 (96) | 28 | .225 |
| Elevated alanine transaminase | 26 (90) | 29 | 4 (17) | 24 | .769 | 23 (96) | 24 | .507 |
| Pathologic coagulation | 20 (54) | 37 | 3 (9) | 33 | 1.000 | 23 (104) | 22 | .059 |
| Hydrops fetalis | 7 (96) | 7 | 1 (17) | 6 | 1.000 | 7 (146) | 5 | .416 |
| Pleural effusion | 6 (94) | 6 | 0 (17) | 0 | .588 | 24 (146) | 16 | .027 |
| Pericardial effusion | 19 (95) | 20 | 2 (17) | 12 | .510 | 17 (146) | 12 | .096 |
| Ascites | 17 (98) | 17 | 3 (17) | 18 | 1.000 | 12 (146) | 8 | .042 |
| Splenomegaly | 28 (97) | 29 | 5 (17) | 29 | 1.000 | 44 (104) | 42 | .056 |
| Hepatomegaly | 43 (97) | 44 | 9 (17) | 53 | .592 | 62 (104) | 60 | .035 |
| Cholestasis | 21 (91) | 23 | 2 (16) | 13 | .344 | 16 (104) | 15 | .202 |
| Liver dysfunction* | 30 (97) | 31 | 5 (17) | 29 | 1.000 | 21 (104) | 20 | .105 |
| Intensive care | 41 (75) | 55 | 7 (11) | 64 | .745 | 37 (146) | 25 | <.001 |
| Ventilation required | 30 (76) | 39 | 4 (11) | 36 | 1.000 | 30 (104) | 29 | .152 |
| Cardiac defects | 69 (100) | 69 | 13 (17) | 76 | .573 | 68 (146) | 47 | <.001 |
| . | Median (range) . | Median (range) . | P . | Median (range) . | P . | |||
|---|---|---|---|---|---|---|---|---|
| Age at diagnosis, d | 4 (0-88) | 7 (0-88) | .278 | 3 (0-65) | .216 | |||
| Birth weight, kg | 2.9 (1.1-4.3) | 3.2 (1.6-3.7) | .098 | 2.9 (1.3-5.6) | .874 | |||
| Gestational age, wk | 37 (28-43) | 37 (30-39) | .965 | 37 (30-41) | .928 | |||
| WBC count, × 109/L | 24.0 (3.2-306) | 23.0 (3.2-149.1) | .447 | 40.3 (3.9-556) | .177 | |||
| Platelets, × 109/L | 102 (13-901) | 94 (15-505) | .803 | 119 (4-1047) | .609 | |||
| Hemoglobin, g/L | 15 (6.7-24.2) | 13.1 (7-20.2) | .408 | 14.5 (4.8-25.7) | .259 | |||
| Blasts peripheral blood, % | 27.5 (0-91) | 41 (0-89) | .368 | 39 (2-95) | .042 | |||
| Blasts bone marrow, % | 15.0 (6-60) | 8.0 | 32 (5-93) | .465 | ||||
| . | Median (range) . | Median (range) . | P . | Median (range) . | P . | |||
|---|---|---|---|---|---|---|---|---|
| Age at diagnosis, d | 4 (0-88) | 7 (0-88) | .278 | 3 (0-65) | .216 | |||
| Birth weight, kg | 2.9 (1.1-4.3) | 3.2 (1.6-3.7) | .098 | 2.9 (1.3-5.6) | .874 | |||
| Gestational age, wk | 37 (28-43) | 37 (30-39) | .965 | 37 (30-41) | .928 | |||
| WBC count, × 109/L | 24.0 (3.2-306) | 23.0 (3.2-149.1) | .447 | 40.3 (3.9-556) | .177 | |||
| Platelets, × 109/L | 102 (13-901) | 94 (15-505) | .803 | 119 (4-1047) | .609 | |||
| Hemoglobin, g/L | 15 (6.7-24.2) | 13.1 (7-20.2) | .408 | 14.5 (4.8-25.7) | .259 | |||
| Blasts peripheral blood, % | 27.5 (0-91) | 41 (0-89) | .368 | 39 (2-95) | .042 | |||
| Blasts bone marrow, % | 15.0 (6-60) | 8.0 | 32 (5-93) | .465 | ||||
“N (of)” refers to the number of patients for whom the data for the respective category/parameter were available. As not all parameters were available for all patients, this number is lower than the total number of patients included in the study. Bolded numbers indicate P < .05.
Hepatomegaly and elevated liver enzymes and/or cholestasis.