Demographics and patient characteristics at the start of deferasirox treatment
| Characteristic . | Deferasirox cohort (n = 296) . | Crossover cohort (n = 259) . | All patients (n = 555) . |
|---|---|---|---|
| Mean age, y (range) | 17.1 (2-49) | 18.3 (3-54) | 17.7 (2-54) |
| Age group, n (%) | |||
| 2 to < 16 y | 153 (51.7) | 120 (46.3) | 273 (49.2) |
| ≥ 16 y | 143 (48.3) | 139 (53.7) | 282 (50.8) |
| Male:female | 140:156 | 134:125 | 274:281 |
| Race (white:Oriental:other)n | 263:9:24 | 225:10:24 | 488:19:48 |
| Mean LIC ± SD, mg Fe/g dw | |||
| Biopsy | 15.5 ± 9.9 | 11.5 ± 7.8 | 13.3 ± 9.2 |
| SQUID | 6.1 ± 2.8 | 5.2 ± 2.9 | 5.7 ± 2.8 |
| LIC category, n (%)* | |||
| < 7 mg Fe/g dw (biopsy) | 60 (20.3) | 64 (24.7) | 124 (22.3) |
| < 7 mg Fe/g dw (SQUID) | 33 (11.1) | 35 (13.5) | 68 (12.3) |
| 7-14 mg Fe/g dw (biopsy) | 68 (23.0) | 93 (35.9) | 161 (29.0) |
| 7-14 mg Fe/g dw (SQUID) | 15 (5.1) | 6 (2.3) | 21 (3.8) |
| ≥ 14 mg Fe/g dw (biopsy) | 120 (40.5) | 58 (22.4) | 178 (32.1) |
| ≥ 14 mg Fe/g dw (SQUID) | 0 | 2 (0.8) | 2 (0.4) |
| Median serum ferritin (range), ng/mL | 2211 (321-12 646) | 1758 (273-8529) | 2007 (273-12 646) |
| Serum ferritin category, n (%) | |||
| ≤ 1000 ng/mL | 21 (7.1) | 48 (18.5) | 69 (12.4) |
| > 1000-2500 ng/mL | 155 (52.4) | 140 (54.1) | 295 (53.2) |
| > 2500-4000 ng/mL | 64 (21.6) | 50 (19.3) | 114 (20.5) |
| > 4000 ng/mL | 56 (18.9) | 21 (8.1) | 77 (13.9) |
| Characteristic . | Deferasirox cohort (n = 296) . | Crossover cohort (n = 259) . | All patients (n = 555) . |
|---|---|---|---|
| Mean age, y (range) | 17.1 (2-49) | 18.3 (3-54) | 17.7 (2-54) |
| Age group, n (%) | |||
| 2 to < 16 y | 153 (51.7) | 120 (46.3) | 273 (49.2) |
| ≥ 16 y | 143 (48.3) | 139 (53.7) | 282 (50.8) |
| Male:female | 140:156 | 134:125 | 274:281 |
| Race (white:Oriental:other)n | 263:9:24 | 225:10:24 | 488:19:48 |
| Mean LIC ± SD, mg Fe/g dw | |||
| Biopsy | 15.5 ± 9.9 | 11.5 ± 7.8 | 13.3 ± 9.2 |
| SQUID | 6.1 ± 2.8 | 5.2 ± 2.9 | 5.7 ± 2.8 |
| LIC category, n (%)* | |||
| < 7 mg Fe/g dw (biopsy) | 60 (20.3) | 64 (24.7) | 124 (22.3) |
| < 7 mg Fe/g dw (SQUID) | 33 (11.1) | 35 (13.5) | 68 (12.3) |
| 7-14 mg Fe/g dw (biopsy) | 68 (23.0) | 93 (35.9) | 161 (29.0) |
| 7-14 mg Fe/g dw (SQUID) | 15 (5.1) | 6 (2.3) | 21 (3.8) |
| ≥ 14 mg Fe/g dw (biopsy) | 120 (40.5) | 58 (22.4) | 178 (32.1) |
| ≥ 14 mg Fe/g dw (SQUID) | 0 | 2 (0.8) | 2 (0.4) |
| Median serum ferritin (range), ng/mL | 2211 (321-12 646) | 1758 (273-8529) | 2007 (273-12 646) |
| Serum ferritin category, n (%) | |||
| ≤ 1000 ng/mL | 21 (7.1) | 48 (18.5) | 69 (12.4) |
| > 1000-2500 ng/mL | 155 (52.4) | 140 (54.1) | 295 (53.2) |
| > 2500-4000 ng/mL | 64 (21.6) | 50 (19.3) | 114 (20.5) |
| > 4000 ng/mL | 56 (18.9) | 21 (8.1) | 77 (13.9) |
LIC at start of deferasirox was not available for 1 patient in the crossover cohort.