Demographic and baseline characteristics
| . | DC . | SC . | ||
|---|---|---|---|---|
| Characteristic . | n . | % . | n . | % . |
| Total | 13 | 100.0 | 3 | 100 |
| Sex, male | 4 | 30.8 | 2 | 66 |
| Age at enrollment, y | ||||
| 2-11 | 6 | 46.2 | 0 | 0 |
| 12-17 | 7 | 53.8 | 3 | 100 |
| Primary race, Black | 13 | 100.0 | 3 | 100 |
| Sickle cell or thalassemia disease type | — | — | ||
| HbSS | 13 | 100.0 | 2 | 66 |
| HbSS/β0 thalassemia | — | — | 1 | 33 |
| CMV seropositive | 7 | 53.8 | 2 | 66 |
| Performance status | ||||
| 100 | 6 | 46.2 | 2 | 66 |
| 90 | 3 | 23.1 | 0 | 0 |
| 80 | 4 | 30.8 | 0 | 0 |
| 70 | 0 | — | 1 | 33 |
| Three or more painful events in the past 2 y | 6 | 46.2 | 2 | 66 |
| ACS (≥2 episodes in the past 2 y) | 2 | 15.4 | 1 | 33 |
| Neurologic event | 4 | 30.8 | 1 | 33 |
| Abnormal MRI/MRA results | 5 | 38.5 | 1 | 33 |
| Long-term PRBC transfusions | 8 | 61.5 | 1 | 33 |
| Hydroxyurea use before enrollment | 10 | 76.9 | 2 | 66 |
| Conditioning regimen A | 3 | 23.0 | — | — |
| (−35 to −10 hydroxyurea, 30 mg/kg per d orally; −9 to −6 busulfan 1 mg/kg per dose, IV every 6 h × 16 doses; −5 to −2 cyclophosphamide 50 mg/kg per d × 4 doses; and −3 to −1 eATG 30 mg/kg per d × 3 doses) | ||||
| Conditioning regimen B * | 10 | 77.0 | 2 | 66 |
| (−35 to −15 hydroxyurea 30 mg/kg/day orally; −14 to −10 fludarabine 35 mg/m2 IV daily × 5 days; −9 to −6 busulfan 1 mg/kg/dose IV every 6 h × 16 doses; and −5 to −2 cyclophosphamide 50 mg/kg per day × 4 doses) | ||||
| Conditioning regimen B (modified)* | — | — | 1 | — |
| (−12 to −10 eATG 30 mg/kg per day) | ||||
| . | DC . | SC . | ||
|---|---|---|---|---|
| Characteristic . | n . | % . | n . | % . |
| Total | 13 | 100.0 | 3 | 100 |
| Sex, male | 4 | 30.8 | 2 | 66 |
| Age at enrollment, y | ||||
| 2-11 | 6 | 46.2 | 0 | 0 |
| 12-17 | 7 | 53.8 | 3 | 100 |
| Primary race, Black | 13 | 100.0 | 3 | 100 |
| Sickle cell or thalassemia disease type | — | — | ||
| HbSS | 13 | 100.0 | 2 | 66 |
| HbSS/β0 thalassemia | — | — | 1 | 33 |
| CMV seropositive | 7 | 53.8 | 2 | 66 |
| Performance status | ||||
| 100 | 6 | 46.2 | 2 | 66 |
| 90 | 3 | 23.1 | 0 | 0 |
| 80 | 4 | 30.8 | 0 | 0 |
| 70 | 0 | — | 1 | 33 |
| Three or more painful events in the past 2 y | 6 | 46.2 | 2 | 66 |
| ACS (≥2 episodes in the past 2 y) | 2 | 15.4 | 1 | 33 |
| Neurologic event | 4 | 30.8 | 1 | 33 |
| Abnormal MRI/MRA results | 5 | 38.5 | 1 | 33 |
| Long-term PRBC transfusions | 8 | 61.5 | 1 | 33 |
| Hydroxyurea use before enrollment | 10 | 76.9 | 2 | 66 |
| Conditioning regimen A | 3 | 23.0 | — | — |
| (−35 to −10 hydroxyurea, 30 mg/kg per d orally; −9 to −6 busulfan 1 mg/kg per dose, IV every 6 h × 16 doses; −5 to −2 cyclophosphamide 50 mg/kg per d × 4 doses; and −3 to −1 eATG 30 mg/kg per d × 3 doses) | ||||
| Conditioning regimen B * | 10 | 77.0 | 2 | 66 |
| (−35 to −15 hydroxyurea 30 mg/kg/day orally; −14 to −10 fludarabine 35 mg/m2 IV daily × 5 days; −9 to −6 busulfan 1 mg/kg/dose IV every 6 h × 16 doses; and −5 to −2 cyclophosphamide 50 mg/kg per day × 4 doses) | ||||
| Conditioning regimen B (modified)* | — | — | 1 | — |
| (−12 to −10 eATG 30 mg/kg per day) | ||||
ACS, acute chest syndrome; MRA, magnetic resonance angiography; MRI, magnetic resonance imaging; PRBC, packed red blood cell; TCD, transcranial Doppler ultrasound.
The last patient in the SC cohort received additional eATG 30 mg/kg per day on days −12 to −10.