TABLE II.
Summary of unconditioned HCT characteristics
| Matched Sibling Donor | Unrelated Donor: Adult Umbilical Cord Blood | |||||||
|---|---|---|---|---|---|---|---|---|
| IL2RG/JAK3 | ADA | Other° | Overall | IL2RG/JAK3 | ADA | Other° | Overall | |
| Genotype (number) | 20/3 | 23 | 20 | 66 | 21/0 | 7 | 9 | 37 |
| Days from Diagnosis to HCT, median (range)° | 23 (1–153) | 27 (12–86) | 32 (12–1389) | 25 (1–1389) | 56 (25–281) | 31 (16–40) | 55 (16–111) | 49 (16–281)* |
| Donor Match^ | ||||||||
| “Complete” | 21 (91%) | 23 (100%) | 19 (95%) | 63 (95%) | 17 (81%) | 7 (100%) | 8 (89%) | 32 (86%) |
| ≥1 Known Mismatches | 2 (9%) | 0 | 1 (5%) | 3 (5%) | 4 (19%) | 0 | 1 (11%) | 5 (14%) |
| Stem Cell Source | ||||||||
| Bone Marrow | 21 (91%) | 23 (100%) | 20 (100%) | 64 (97%) | 9 (43%) | 1 (14%) | 6 (67%) | 16 (43%)* |
| Umbilical Cord Blood | 2 (9%) | 0 | 0 | 2 (3%) | 9 (43%) | 6 (86%) | 3 (33%) | 18 (49%)* |
| PBSC (TCD) | 0 | 0 | 0 | 0 | 3 (14%) | 0 | 0 | 3 (8%)* |
| Serotherapy Given | 0 | 0 | 2 (10%) | 2 (3%) | 10 (48%)* | 1 (14%) | 3 (33%) | 14 (38%)* |
| GVHD Prophylaxis Agents# | ||||||||
| Ex Vivo TCD | 4 (17%) | 0 | 1 (5%) | 5 (7%) | 3 (14%) | 0 | 0 | 3 (8%) |
| None | 16 (70%) | 17 (74%) | 6 (30%) | 39 (59%) | 1 (5%)* | 0 | 0 | 1 (3%)* |
| One | 2 (9%) | 6 (26%) | 5 (25%) | 13 (20%) | 3 (14%) | 2 (29%) | 4 (45%) | 9 (24%) |
| Two | 1 (4%) | 0 | 8 (40%) | 9 (14%) | 6 (29%)* | 4 (57%)* | 3 (33%) | 13 (35%)* |
| Three | 0 | 0 | 0 | 0 | 8 (38%)* | 1 (14%) | 2 (22%) | 11 (30%)* |
ADA, adenosine deaminase; PBSC, peripheral blood stem cells; TCD, ex vivo T cell depletion; GVHD, graft versus host disease
*
P <0.05 compared to sibling donor recipients
°See methods for details
°Excluding patients treated with ADA enzyme replacement therapy prior to HCT
^
As defined by era of HCT
#
Including Pre-HCT Serotherapy