Demographic, disease, and baseline patient characteristics
| Variable . | Deferasirox 5 mg/kg/d (n = 55) . | Deferasirox 10 mg/kg/d (n = 55) . | Placebo 5 and 10 mg/kg/d (n = 56) . |
|---|---|---|---|
| Disease, n (%) | |||
| β-thalassemia intermedia | 32 (58.2) | 30 (54.5) | 33 (58.9) |
| α-thalassemia* | 5 (9.1) | 9 (16.4) | 8 (14.3) |
| HbE/β-thalassemia | 18 (32.7) | 16 (29.1) | 15 (26.8) |
| Mean age, y ± SD | 33.1 ± 12.3 | 31.7 ± 11.7 | 31.4 ± 12.2 |
| Median age, y (range) | 33 (10-60) | 31 (12-69) | 32 (10-59) |
| Pediatric patients, n (%)† | 6 (10.9) | 7 (12.7) | 8 (14.3) |
| Male: female ratio, n | 29:26 | 29:26 | 31:25 |
| Splenectomy, n (%) | 29 (52.7) | 31 (56.4) | 28 (50.0) |
| Prior transfusions received, n (%)‡ | 49 (89.1) | 50 (90.9) | 46 (82.1) |
| Previous chelation, n (%) | 8 (14.5) | 16 (29.1) | 20 (35.7) |
| Deferoxamine | 7 (12.7) | 15 (27.3) | 17 (30.4) |
| Deferiprone | 0 (0) | 1 (1.8) | 3 (5.4) |
| Deferoxamine + deferiprone | 1 (1.8) | 0 (0) | 0 (0) |
| Mean LIC, mg Fe/g dw ± SD§ | 13.11 ± 7.29 | 14.56 ± 7.92 | 15.94 ± 10.85 |
| Median LIC, mg Fe/g dw (range) | 11.7 (2.6-38.6) | 11.7 (5.0-32.8) | 13.0 (5.0-49.1) |
| LIC category, n (%)¶ | |||
| < 7 mg Fe/g dw | 10 (18.2) | 8 (14.5) | 13 (23.2) |
| 7-15 mg Fe/g dw | 31 (56.4) | 26 (47.3) | 20 (35.7) |
| > 15 mg Fe/g dw | 14 (25.5) | 21 (38.2) | 22 (39.3) |
| Mean serum ferritin, ng/mL ± SD | 1141 ± 805 | 1174 ± 684 | 1305 ± 1017 |
| Median serum ferritin, ng/mL (range) | 988 (370-5609) | 1015 (342-4224) | 994 (304-6419) |
| Serum ferritin category, n (%) | |||
| < 500 ng/mL | 5 (9.1) | 4 (7.3) | 8 (14.3) |
| 500-1000 ng/mL | 24 (43.6) | 23 (41.8) | 20 (35.7) |
| 1000-2500 ng/mL | 23 (41.8) | 26 (47.3) | 23 (41.1) |
| > 2500 ng/mL | 3 (5.5) | 2 (3.6) | 5 (8.9) |
| Variable . | Deferasirox 5 mg/kg/d (n = 55) . | Deferasirox 10 mg/kg/d (n = 55) . | Placebo 5 and 10 mg/kg/d (n = 56) . |
|---|---|---|---|
| Disease, n (%) | |||
| β-thalassemia intermedia | 32 (58.2) | 30 (54.5) | 33 (58.9) |
| α-thalassemia* | 5 (9.1) | 9 (16.4) | 8 (14.3) |
| HbE/β-thalassemia | 18 (32.7) | 16 (29.1) | 15 (26.8) |
| Mean age, y ± SD | 33.1 ± 12.3 | 31.7 ± 11.7 | 31.4 ± 12.2 |
| Median age, y (range) | 33 (10-60) | 31 (12-69) | 32 (10-59) |
| Pediatric patients, n (%)† | 6 (10.9) | 7 (12.7) | 8 (14.3) |
| Male: female ratio, n | 29:26 | 29:26 | 31:25 |
| Splenectomy, n (%) | 29 (52.7) | 31 (56.4) | 28 (50.0) |
| Prior transfusions received, n (%)‡ | 49 (89.1) | 50 (90.9) | 46 (82.1) |
| Previous chelation, n (%) | 8 (14.5) | 16 (29.1) | 20 (35.7) |
| Deferoxamine | 7 (12.7) | 15 (27.3) | 17 (30.4) |
| Deferiprone | 0 (0) | 1 (1.8) | 3 (5.4) |
| Deferoxamine + deferiprone | 1 (1.8) | 0 (0) | 0 (0) |
| Mean LIC, mg Fe/g dw ± SD§ | 13.11 ± 7.29 | 14.56 ± 7.92 | 15.94 ± 10.85 |
| Median LIC, mg Fe/g dw (range) | 11.7 (2.6-38.6) | 11.7 (5.0-32.8) | 13.0 (5.0-49.1) |
| LIC category, n (%)¶ | |||
| < 7 mg Fe/g dw | 10 (18.2) | 8 (14.5) | 13 (23.2) |
| 7-15 mg Fe/g dw | 31 (56.4) | 26 (47.3) | 20 (35.7) |
| > 15 mg Fe/g dw | 14 (25.5) | 21 (38.2) | 22 (39.3) |
| Mean serum ferritin, ng/mL ± SD | 1141 ± 805 | 1174 ± 684 | 1305 ± 1017 |
| Median serum ferritin, ng/mL (range) | 988 (370-5609) | 1015 (342-4224) | 994 (304-6419) |
| Serum ferritin category, n (%) | |||
| < 500 ng/mL | 5 (9.1) | 4 (7.3) | 8 (14.3) |
| 500-1000 ng/mL | 24 (43.6) | 23 (41.8) | 20 (35.7) |
| 1000-2500 ng/mL | 23 (41.8) | 26 (47.3) | 23 (41.1) |
| > 2500 ng/mL | 3 (5.5) | 2 (3.6) | 5 (8.9) |
HbH disease (n = 8), HbH Constant Spring (n = 6), genotype not determined (n = 6), CSEA Bart's (n = 1), and Hb Agrino (n = 1).
Pediatric subjects were < 18 years of age.
Patients did not receive any transfusion in the 6 months prior to study entry.
95% of the ULN for LIC is < 1.8 mg Fe/g dw.21
One patient was missing from the placebo group.