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. 2021 Feb 8;5(3):843–852. doi: 10.1182/bloodadvances.2020003248

Table 1.

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)

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.