Baseline characteristics of patients enrolled
| . | Adequate responders, n = 5 . | Inadequate responders, n = 10 . |
|---|---|---|
| Age, y | 9-38 y | 3-36 y |
| Younger than 20 | 3 | 7 |
| 21-40 | 2 | 3 |
| Sex | ||
| Male | 3 | 7 |
| Female | 2 | 3 |
| Ethnicity | ||
| Asian | 1 | 4 |
| Black | 1 | 1 |
| White | 3 | 5 |
| Baseline disease | ||
| Sickle cell | 1 | 1 |
| Thalassemia | 4 | 9†‡ |
| Estimated hepatic iron concentration* | 1.8-4.6 | 2.9-19.5 |
| Less than 4 mg/g dry wt liver | 3 | 3 |
| 4-7 mg/g | 2 | 1 |
| More than 7 mg/g | 0 | 6 |
| . | Adequate responders, n = 5 . | Inadequate responders, n = 10 . |
|---|---|---|
| Age, y | 9-38 y | 3-36 y |
| Younger than 20 | 3 | 7 |
| 21-40 | 2 | 3 |
| Sex | ||
| Male | 3 | 7 |
| Female | 2 | 3 |
| Ethnicity | ||
| Asian | 1 | 4 |
| Black | 1 | 1 |
| White | 3 | 5 |
| Baseline disease | ||
| Sickle cell | 1 | 1 |
| Thalassemia | 4 | 9†‡ |
| Estimated hepatic iron concentration* | 1.8-4.6 | 2.9-19.5 |
| Less than 4 mg/g dry wt liver | 3 | 3 |
| 4-7 mg/g | 2 | 1 |
| More than 7 mg/g | 0 | 6 |
Assessed by T2* MRI or liver biopsy.
Includes one patient with alpha-thalassemia major: this patient was not on deferasirox at the time of enrollment in the study but had been on the drug for many months until developing acute liver toxicity.
Includes twin siblings with Hgb Dartmouth/alpha-thalassemia compound heterozygosity. Hemoglobin Dartmouth is a hyperunstable alpha globin, α2[T197>C], which encodes α2Leu66Pro. The other allele is (−SEA)α0 thalassemia deletion.14