Table 1.
Schedule of study evaluations.
| Evaluation | Enrollment | Year 1a | Year 2b |
| Medical historyc | Yes | Yes | Yes |
| Prior/concomitant medicationsc | Yes | Yes | Yes |
| Physical examinationc | Yes | Yes | Yes |
| Labs (complete blood count with differential, reticulocyte count)c | Yes | Yes | Yes |
| Hemoglobin electrophoresis | Yes | No | No |
| Hemoglobin F leveld | Yes | Yes | Yes |
| TCDe examinationf | Yes | Yes | Yes |
| Special studiesg | Yes | No | No |
| Brain MRI/MRAh for participants on hydroxyurea only | Yes | No | Yes |
aYear 1 is 12 months plus or minus 3 months.
bYear 2 is 24 months plus or minus 6 months.
cParticipants on hydroxyurea will undergo these evaluations monthly until reaching maximum tolerated dose (MTD) and then quarterly after MTD is reached.
dQuarterly for participants on hydroxyurea.
eTCD: transcranial Doppler.
fEvery 6 months for participants on hydroxyurea.
gSpecial studies includes specimens for genomic DNA analysis and serum biomarkers. Will be additionally collected at study exit for children on hydroxyurea.
hMRI/MRA: magnetic resonance imaging/magnetic resonance angiogram.