Baseline characteristics of the patients by treatment group
| Characteristics . | ELTR + IST (n = 49), n (%) . | IST (n = 49), n (%) . | P value∗ . |
|---|---|---|---|
| Age (yr), median (range) | 10.5 (2-17.7) | 8.7 (2.1-16.8) | .285 |
| Sex | |||
| Male | 35 (71.4) | 30 (61.2) | .285 |
| Female | 14 (28.6) | 19 (38.8) | — |
| Ethnicity | |||
| White | 47 (96) | 46 (94) | .646 |
| Asian | 2 (4.0) | 3 (6.0) | — |
| Type of AA | |||
| Idiopathic | 45 (91.8) | 42 (85.7) | .337 |
| Hepatitis-associated | 4 (8.2) | 7 (14.3) | — |
| Severity of AA | |||
| Severe | 18 (36.7) | 21 (42.9) | .536 |
| Very severe | 31 (63.3) | 28 (57.1) | — |
| Absolute neutrophil count, per μL, median (range) | 150 (0-960) | 170 (0-1170) | .803 |
| Reticulocyte count, per μL, median (range) | 12 320 (0-70 140) | 14 750 (0-75 900) | .561 |
| Platelet count, per μL, median (range) | 15 (2-36) | 12 (1-37) | .094 |
| PNH | |||
| Negative | 35 (71.4) | 30 (61.2) | .285 |
| Positive | 14 (28.6) | 19 (38.8) | — |
| GPI-deficient neutrophils (PNH+) | |||
| <1% | 10 (20.4) | 12 (24.5) | — |
| ≥1% | 4 (8.2) | 7 (14.3) | — |
| Cytogenetics | |||
| Normal karyotype | 19 (38.8) | 21 (42.9) | .681 |
| Insufficient metaphases | 30 (61.2) | 28 (57.1) | — |
| Telomere length | |||
| Normal | 38 (86.4) | 34 (70.8) | .071 |
| Below normal | 6 (13.6) | 14 (29.2) | — |
| ND | 5 | 1 | — |
| Characteristics . | ELTR + IST (n = 49), n (%) . | IST (n = 49), n (%) . | P value∗ . |
|---|---|---|---|
| Age (yr), median (range) | 10.5 (2-17.7) | 8.7 (2.1-16.8) | .285 |
| Sex | |||
| Male | 35 (71.4) | 30 (61.2) | .285 |
| Female | 14 (28.6) | 19 (38.8) | — |
| Ethnicity | |||
| White | 47 (96) | 46 (94) | .646 |
| Asian | 2 (4.0) | 3 (6.0) | — |
| Type of AA | |||
| Idiopathic | 45 (91.8) | 42 (85.7) | .337 |
| Hepatitis-associated | 4 (8.2) | 7 (14.3) | — |
| Severity of AA | |||
| Severe | 18 (36.7) | 21 (42.9) | .536 |
| Very severe | 31 (63.3) | 28 (57.1) | — |
| Absolute neutrophil count, per μL, median (range) | 150 (0-960) | 170 (0-1170) | .803 |
| Reticulocyte count, per μL, median (range) | 12 320 (0-70 140) | 14 750 (0-75 900) | .561 |
| Platelet count, per μL, median (range) | 15 (2-36) | 12 (1-37) | .094 |
| PNH | |||
| Negative | 35 (71.4) | 30 (61.2) | .285 |
| Positive | 14 (28.6) | 19 (38.8) | — |
| GPI-deficient neutrophils (PNH+) | |||
| <1% | 10 (20.4) | 12 (24.5) | — |
| ≥1% | 4 (8.2) | 7 (14.3) | — |
| Cytogenetics | |||
| Normal karyotype | 19 (38.8) | 21 (42.9) | .681 |
| Insufficient metaphases | 30 (61.2) | 28 (57.1) | — |
| Telomere length | |||
| Normal | 38 (86.4) | 34 (70.8) | .071 |
| Below normal | 6 (13.6) | 14 (29.2) | — |
| ND | 5 | 1 | — |
ND, no data.
The demographics and baseline characteristics of the patients were similar across the 2 groups. The median values of continuous variables were compared between randomization groups by the Mann-Whitney test, and the frequencies of categorical variables were compared using Pearson’s χ-squared test. The P values of these tests are shown in the last column.