| Age at HCT, yr, median (range) |
3.9 (0.25-25.5) |
4 (0.41-24.3) |
6.1 (0.43-10.8) |
.79 |
| Underlying genetic diagnosis |
|
|
|
|
| PRF1
|
3 (9) |
2 (9) |
2 (13) |
|
| UNC13D
|
2 (6) |
4 (17) |
2 (13) |
|
| STXBP2
|
6 (18) |
2 (9) |
1 (7) |
|
| STX11
|
0 |
0 |
0 |
|
| XIAP/BIRC4
|
4 (12) |
1 (4) |
1 (7) |
|
| SH2D1A
|
7 (21) |
5 (22) |
0 |
|
| RAB27A
|
0 |
0 |
1 (7) |
|
| Unknown |
9 (27) |
7 (30) |
7 (47) |
|
| Heterozygous for UNC13D and/or STXBP2 |
2 (6) |
2 (9) |
1 (7) |
|
| Donor match |
|
|
|
.12 |
| 8/8 HLA-A, -B, -C, -DR |
21 (64) |
21 (88) |
12 (80) |
|
| 1 or 2 allele mismatch |
12 (36) |
3 (13) |
3 (20) |
|
| Donor relation |
|
|
|
1.00 |
| Sibling |
8 (24) |
6 (25) |
3 (20) |
|
| Unrelated |
25 (76) |
18 (75) |
12 (80) |
|
| Stem cell source |
|
|
|
.44 |
| Bone marrow |
31 (94) |
24 (100) |
14 (93) |
|
| Peripheral blood stem cells |
2 (6) |
0 |
1 (7) |
|
| Total nucleated cell dose, × 108/kg, median (range) |
7 (1.4-23.3) |
7.4 (2.3-15) |
7 (3.2-10) |
.73 |
| CD3+ cell dose, × 107/kg, median (range) |
6.8 (1.8-58.8) |
5.9 (2.6-16.2) |
6.2 (2.1-15.7) |
.69 |
