Demographic and clinical parameters of the pediatric SCD cohort
| Demographics at study entry . | Total cohort (N = 180) . | HC (n = 96) . | BT (n = 84) . |
|---|---|---|---|
| Age (y), mean (SD, range) | 12 (4, 4-19) | 12 (7, 4-19) | 12 (6, 4-19) |
| Gender, male/female | 82/98 | 45/51 | 37/47 |
| ABO blood group, O/non-O (%) | 60.5/39.5 | 60/40 | 61/39 |
| Age (y) at starting therapy, mean (SD) | 7.1 (4.7) | 6.2 (3.9) | 8.7 (5.3) |
| Duration of therapy (mo), mean (SD) | 66.0 (42.9) | 65.1 (37.7) | 67.7 (50.3) |
| HC dose (mg/kg), mean (range) | NA | 19 (10-33) | NA |
| Indication for therapy (%) | |||
| Routinely offered at age >1 y | 100 | NA | |
| Abnormal TCD | NA | 47 | |
| Ischemia on MRI (silent stroke) | NA | 27 | |
| Stroke | NA | 8 | |
| Recurrent crises despite HC | NA | 18 | |
| Demographics at study entry . | Total cohort (N = 180) . | HC (n = 96) . | BT (n = 84) . |
|---|---|---|---|
| Age (y), mean (SD, range) | 12 (4, 4-19) | 12 (7, 4-19) | 12 (6, 4-19) |
| Gender, male/female | 82/98 | 45/51 | 37/47 |
| ABO blood group, O/non-O (%) | 60.5/39.5 | 60/40 | 61/39 |
| Age (y) at starting therapy, mean (SD) | 7.1 (4.7) | 6.2 (3.9) | 8.7 (5.3) |
| Duration of therapy (mo), mean (SD) | 66.0 (42.9) | 65.1 (37.7) | 67.7 (50.3) |
| HC dose (mg/kg), mean (range) | NA | 19 (10-33) | NA |
| Indication for therapy (%) | |||
| Routinely offered at age >1 y | 100 | NA | |
| Abnormal TCD | NA | 47 | |
| Ischemia on MRI (silent stroke) | NA | 27 | |
| Stroke | NA | 8 | |
| Recurrent crises despite HC | NA | 18 | |
MRI, magnetic resonance imaging; NA, not applicable; SD, standard deviation; TCD, transcranial Doppler.