Baseline characteristics
Patient . | Cohort . | Age (y) . | Sex . | Disease type . | Disease-related complications/comorbidities . | HCT-CI . | Donor . | HLA match . | CD34 dose (× 106/kg) . | CD3 dose (× 108/kg) . | |||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Age (y) . | Sex . | Relation . | HbS (%) . | ||||||||||
1 | 1 | 44 | F | HbSS | ESRD on PD, pHTN, diastolic dysfunction, iron overload, history of ACS/VOC | 6 | 38 | F | Sister | 39 | 5/10 | 15.9 | 5.50 |
2 | 1 | 20 | M | HbSS | pHTN, iron overload, stroke, moyamoya | 10 | 47 | F | Mother | 40 | 6/10 | 15.0 | 2.65 |
3 | 1 | 36 | F | HbSS | pHTN, history of VOC, RA | 6 | 46 | F | Sister | 0 | 7/10 | 9.76 | 2.83 |
4 | 2 | 37 | F | HbSS | Iron overload, history of VOC, MS | 7 | 66 | F | Mother | 39 | 8/10 | 10.2 | 3.78 |
5 | 2 | 47 | M | HbSS | Systolic dysfunction, cirrhosis/NRH, history of ACS/VOC, stroke | 7 | 60 | F | Sister | 38 | 6/10 | 11.9 | 7.93 |
6 | 2 | 36 | M | HbSS | Systolic dysfunction, iron overload, history of ACS/VOC, stroke | 8 | 28 | M | Brother | 38 | 7/10 | 28.0 | 5.01 |
7 | 2 | 31 | F | HbSS | Iron overload, moyamoya | 3 | 60 | F | Mother | 38 | 7/10 | 13.0 | 8.07 |
8 | 2 | 35 | M | β-thal | Iron overload | 3 | 67 | F | Mother | 0 | 6/10 | 8.79 | 3.75 |
9 | 2 | 21 | M | HbSS | Iron overload, history of ACS/VOC, stroke, moyamoya | 7 | 51 | F | Mother | 39 | 5/10 | 12.2 | 3.51 |
10 | 2 | 25 | M | β-thal | Iron overload | 7 | 55 | F | Mother | 0 | 7/10 | 7.04 | 1.86 |
11 | 2 | 24 | M | HbSS | ESRD on HD, systolic dysfunction, iron overload, history of ACS/VOC | 6 | 20 | M | Brother | 39 | 7/10 | 13.4 | 2.59 |
12 | 3 | 37 | M | HbSS | ESRD on PD, iron overload, history of VOC | 6 | 61 | F | Mother | 34 | 8/10 | 25.6 | 4.73 |
13 | 3 | 41 | M | HbSS | ESRD on PD, systolic and diastolic dysfunction, iron overload, history of VOC | 10 | 45 | F | Sister | 0 | 8/10 | 15.9 | 5.08 |
14 | 3 | 37 | M | HbSS | CRI, iron overload, history of ACS/VOC, stroke | 3 | 56 | F | Mother | 40 | 7/10 | 10.2 | 2.98 |
15 | 3 | 31 | F | HbSS | pHTN, iron overload, history of ACS/VOC, TIA | 7 | 30 | F | Sister | 42 | 7/10 | 15.1 | 4.00 |
16 | 3 | 56 | F | HbSC | Iron overload, history of ACS/VOC | 3 | 31 | M | Son | 40 | 8/10 | 29.7 | 3.78 |
17 | 3 | 26 | M | HbSS | History of ACS/VOC | 3 | 51 | F | Mother | 39 | 6/10 | 16.8 | 3.948 |
18 | 3 | 20 | F | HbSS | Iron overload, history of ACS/VOC | 2 | 51 | F | Mother | 40 | 6/10 | 10.2 | 6.14 |
19 | 3 | 47 | F | HbSS | CRI, diastolic dysfunction, iron overload, history of ACS/VOC | 9 | 23 | F | Sister | 0 | 5/10 | 16.6 | 2.95 |
20 | 3 | 34 | F | HbS β0-thal | Cirrhosis, history of VOC | 8 | 30 | M | Brother | 0 | 8/10 | 9.70 | 5.28 |
21 | 3 | 27 | M | HbSS | Iron overload, history of ACS/VOC, stroke, moyamoya | 6 | 52 | F | Mother | 39 | 5/10 | 11.5 | 3.65 |
22 | 3 | 36 | M | HbSS | Iron overload, history of ACS/VOC | 5 | 64 | M | Father | 35 | 5/10 | 12.2 | 2.42 |
23 | 3 | 42 | M | HbSS | pHTN, systolic dysfunction, history of ACS | 6 | 23 | F | Sister | 0 | 7/10 | 10.1 | 6.12 |
Patient . | Cohort . | Age (y) . | Sex . | Disease type . | Disease-related complications/comorbidities . | HCT-CI . | Donor . | HLA match . | CD34 dose (× 106/kg) . | CD3 dose (× 108/kg) . | |||
---|---|---|---|---|---|---|---|---|---|---|---|---|---|
Age (y) . | Sex . | Relation . | HbS (%) . | ||||||||||
1 | 1 | 44 | F | HbSS | ESRD on PD, pHTN, diastolic dysfunction, iron overload, history of ACS/VOC | 6 | 38 | F | Sister | 39 | 5/10 | 15.9 | 5.50 |
2 | 1 | 20 | M | HbSS | pHTN, iron overload, stroke, moyamoya | 10 | 47 | F | Mother | 40 | 6/10 | 15.0 | 2.65 |
3 | 1 | 36 | F | HbSS | pHTN, history of VOC, RA | 6 | 46 | F | Sister | 0 | 7/10 | 9.76 | 2.83 |
4 | 2 | 37 | F | HbSS | Iron overload, history of VOC, MS | 7 | 66 | F | Mother | 39 | 8/10 | 10.2 | 3.78 |
5 | 2 | 47 | M | HbSS | Systolic dysfunction, cirrhosis/NRH, history of ACS/VOC, stroke | 7 | 60 | F | Sister | 38 | 6/10 | 11.9 | 7.93 |
6 | 2 | 36 | M | HbSS | Systolic dysfunction, iron overload, history of ACS/VOC, stroke | 8 | 28 | M | Brother | 38 | 7/10 | 28.0 | 5.01 |
7 | 2 | 31 | F | HbSS | Iron overload, moyamoya | 3 | 60 | F | Mother | 38 | 7/10 | 13.0 | 8.07 |
8 | 2 | 35 | M | β-thal | Iron overload | 3 | 67 | F | Mother | 0 | 6/10 | 8.79 | 3.75 |
9 | 2 | 21 | M | HbSS | Iron overload, history of ACS/VOC, stroke, moyamoya | 7 | 51 | F | Mother | 39 | 5/10 | 12.2 | 3.51 |
10 | 2 | 25 | M | β-thal | Iron overload | 7 | 55 | F | Mother | 0 | 7/10 | 7.04 | 1.86 |
11 | 2 | 24 | M | HbSS | ESRD on HD, systolic dysfunction, iron overload, history of ACS/VOC | 6 | 20 | M | Brother | 39 | 7/10 | 13.4 | 2.59 |
12 | 3 | 37 | M | HbSS | ESRD on PD, iron overload, history of VOC | 6 | 61 | F | Mother | 34 | 8/10 | 25.6 | 4.73 |
13 | 3 | 41 | M | HbSS | ESRD on PD, systolic and diastolic dysfunction, iron overload, history of VOC | 10 | 45 | F | Sister | 0 | 8/10 | 15.9 | 5.08 |
14 | 3 | 37 | M | HbSS | CRI, iron overload, history of ACS/VOC, stroke | 3 | 56 | F | Mother | 40 | 7/10 | 10.2 | 2.98 |
15 | 3 | 31 | F | HbSS | pHTN, iron overload, history of ACS/VOC, TIA | 7 | 30 | F | Sister | 42 | 7/10 | 15.1 | 4.00 |
16 | 3 | 56 | F | HbSC | Iron overload, history of ACS/VOC | 3 | 31 | M | Son | 40 | 8/10 | 29.7 | 3.78 |
17 | 3 | 26 | M | HbSS | History of ACS/VOC | 3 | 51 | F | Mother | 39 | 6/10 | 16.8 | 3.948 |
18 | 3 | 20 | F | HbSS | Iron overload, history of ACS/VOC | 2 | 51 | F | Mother | 40 | 6/10 | 10.2 | 6.14 |
19 | 3 | 47 | F | HbSS | CRI, diastolic dysfunction, iron overload, history of ACS/VOC | 9 | 23 | F | Sister | 0 | 5/10 | 16.6 | 2.95 |
20 | 3 | 34 | F | HbS β0-thal | Cirrhosis, history of VOC | 8 | 30 | M | Brother | 0 | 8/10 | 9.70 | 5.28 |
21 | 3 | 27 | M | HbSS | Iron overload, history of ACS/VOC, stroke, moyamoya | 6 | 52 | F | Mother | 39 | 5/10 | 11.5 | 3.65 |
22 | 3 | 36 | M | HbSS | Iron overload, history of ACS/VOC | 5 | 64 | M | Father | 35 | 5/10 | 12.2 | 2.42 |
23 | 3 | 42 | M | HbSS | pHTN, systolic dysfunction, history of ACS | 6 | 23 | F | Sister | 0 | 7/10 | 10.1 | 6.12 |
ACS, acute chest syndrome; β-thal, β-thalassemia; CRI, chronic renal insufficiency; ESRD, end-stage renal disease; F, female; HbS, hemoglobin S; HbSC, compound heterozygous HbS and HbC disease; HbSS, homozygous sickle cell disease; HbS β0-thal, compound heterozygous HbS and β0-thalassemia disease; HCT-CI, hematopoietic cell transplantation–specific comorbidity index; HD, hemodialysis; M, male; MS, multiple sclerosis; NRH, nodular regenerative hyperplasia; PD, peritoneal dialysis; pHTN, pulmonary hypertension; RA, rheumatoid arthritis; TIA, transient ischemic attack; VOC, vaso-occlusive crisis.