Demographic and baseline patient characteristics
| Variable . | All patients, n = 60* . |
|---|---|
| Disease, n (%) | |
| β-thalassemia major | 59 (98.3) |
| DBA | 1 (1.7) |
| Age, y | 22.8 ± 7.3 |
| Median age (range), y | 22.0 (10.0-41.0) |
| Male:female, n | 28:32 |
| Race, n (%) | |
| White | 50 (83.3) |
| Black | 1 (1.7) |
| Asian | 9 (15.0) |
| Hepatitis status, n (%) | |
| Hepatitis B | 1 (1.7) |
| Hepatitis C | 16 (26.7) |
| Hepatitis B and C | 1 (1.7) |
| No hepatitis | 42 (70.0) |
| Time since start of blood transfusions, y | 22.3 ± 7.3 |
| Total no. of blood transfusions received, n | 371.5 ± 161.6 |
| Previous chelation therapy, n (%) | 60 (100) |
| DFO | 12 (20.0) |
| Deferiprone | 8 (13.3) |
| DFX | 20 (33.3) |
| DFO + deferiprone | 18 (30.0) |
| Other† | 2 (3.3) |
| Time since start of first chelation therapy, y | 18.6 ± 8.1 |
| mT2*, ms | |
| 5 to <6 | 10 (16.7) |
| 6 to <10 | 50 (83.3) |
| LVEF, % | 66.5 ± 5.3 |
| LIC, mg Fe/g dw | 33.4 ± 14.5 |
| LIC categories, mg Fe/g dw, n (%) | |
| 7 to <15 | 6 (10.0) |
| 15 to <30 | 13 (21.7) |
| ≥30 | 41 (68.3) |
| Median serum ferritin (range), ng/mL | 5551 (1163-11 317) |
| Variable . | All patients, n = 60* . |
|---|---|
| Disease, n (%) | |
| β-thalassemia major | 59 (98.3) |
| DBA | 1 (1.7) |
| Age, y | 22.8 ± 7.3 |
| Median age (range), y | 22.0 (10.0-41.0) |
| Male:female, n | 28:32 |
| Race, n (%) | |
| White | 50 (83.3) |
| Black | 1 (1.7) |
| Asian | 9 (15.0) |
| Hepatitis status, n (%) | |
| Hepatitis B | 1 (1.7) |
| Hepatitis C | 16 (26.7) |
| Hepatitis B and C | 1 (1.7) |
| No hepatitis | 42 (70.0) |
| Time since start of blood transfusions, y | 22.3 ± 7.3 |
| Total no. of blood transfusions received, n | 371.5 ± 161.6 |
| Previous chelation therapy, n (%) | 60 (100) |
| DFO | 12 (20.0) |
| Deferiprone | 8 (13.3) |
| DFX | 20 (33.3) |
| DFO + deferiprone | 18 (30.0) |
| Other† | 2 (3.3) |
| Time since start of first chelation therapy, y | 18.6 ± 8.1 |
| mT2*, ms | |
| 5 to <6 | 10 (16.7) |
| 6 to <10 | 50 (83.3) |
| LVEF, % | 66.5 ± 5.3 |
| LIC, mg Fe/g dw | 33.4 ± 14.5 |
| LIC categories, mg Fe/g dw, n (%) | |
| 7 to <15 | 6 (10.0) |
| 15 to <30 | 13 (21.7) |
| ≥30 | 41 (68.3) |
| Median serum ferritin (range), ng/mL | 5551 (1163-11 317) |
Values are mean ± SD unless otherwise stated.
DBA, Diamond–Blackfan anemia; DFO, deferoxamine; DFX, deferasirox; LIC, liver iron concentration, LVEF, left ventricular ejection fraction; mT2*, myocardial T2*.
Recruited across 7 countries (Turkey, n = 18; Egypt, n = 15; Italy, n = 14; Thailand, n = 7; United Kingdom, n = 3; Greece, n = 2; Taiwan, n = 1).
One patient received DFO for 180 months (22.6 mg/kg) plus DFX for 50 months (42 mg/kg) and 1 patient received 1 week on DFO-deferiprone combination therapy, followed by 1 week on DFX-DFO combination therapy for a total of 4 months; DFO (50 mg/kg), deferiprone (96 mg/kg), DFX (40 mg/kg).