Transplant characteristics
| . | Stratum A, typical SCID (N = 68) . | Stratum B, atypical SCID (n = 30)* . | P . |
|---|---|---|---|
| Active infection at time of HCT, n (%) | 23 (34) | 14 (47) | .444 |
| Age at HCT, d, n (range) | 103 (16-1630) | 142 (30-5137) | .037 |
| Donor, n (%) | .132 | ||
| MRD | 8 (12) | 3 (10) | |
| Bone marrow | 7 (10) | 3 (10) | |
| Peripheral stem cells | 1 (2) | 0 | |
| MMRD | 20 (29) | 3 (10) | |
| Bone marrow | 9 (13) | 2 (7) | |
| Peripheral stem cells | 11 (16) | 1 (3) | |
| Adult URD | 19 (28) | 14 (47) | |
| Bone marrow | 17 (25) | 12 (40) | |
| Peripheral stem cells | 2 (3) | 2 (7) | |
| UCB | 21 (31) | 10 (33) | |
| Median stem cell dose in TNC × 107/kg (range; n) | |||
| MRD | 79.8 (27-130; 6) | 50.3 (14.4-111.7; 3) | |
| MMRD | 3.7 (1.3-233.3; 17) | 2.7 (1.9-3.6; 3) | |
| MMRD: CD34+ selection | 3.1 (2.0-4.8; 9) | 1.92 (1.92-1.92; 1) | |
| MMRD: T-cell depletion | 14.6 (1.3-233.3; 8) | 3.1 (2.7-3.6; 2) | |
| MUD | 48.2 (0.5-226; 16) | 66 (1.7-146.7; 11) | |
| UCB | 13.6 (7.8-42.1; 18) | 20 (15.9-24.8; 10) | |
| Conditioning, n (%) | .027 | ||
| None | 22 (32) | 2 (7) | |
| Immunosuppression (serotherapy) only | 10 (15) | 3 (10) | |
| RIC | 19 (28) | 13 (43) | |
| MAC (busulfan > 12 mg/kg) | 17 (25) | 12 (40) | |
| GVHD prophylaxis, n (%) | .020 | ||
| None | 5 (7) | 0 | |
| Ex vivo T-cell depletion | 23 (34) | 4 (13) | |
| Calcineurin inhibitor with or without other agents | 40 (59) | 26 (87) |
| . | Stratum A, typical SCID (N = 68) . | Stratum B, atypical SCID (n = 30)* . | P . |
|---|---|---|---|
| Active infection at time of HCT, n (%) | 23 (34) | 14 (47) | .444 |
| Age at HCT, d, n (range) | 103 (16-1630) | 142 (30-5137) | .037 |
| Donor, n (%) | .132 | ||
| MRD | 8 (12) | 3 (10) | |
| Bone marrow | 7 (10) | 3 (10) | |
| Peripheral stem cells | 1 (2) | 0 | |
| MMRD | 20 (29) | 3 (10) | |
| Bone marrow | 9 (13) | 2 (7) | |
| Peripheral stem cells | 11 (16) | 1 (3) | |
| Adult URD | 19 (28) | 14 (47) | |
| Bone marrow | 17 (25) | 12 (40) | |
| Peripheral stem cells | 2 (3) | 2 (7) | |
| UCB | 21 (31) | 10 (33) | |
| Median stem cell dose in TNC × 107/kg (range; n) | |||
| MRD | 79.8 (27-130; 6) | 50.3 (14.4-111.7; 3) | |
| MMRD | 3.7 (1.3-233.3; 17) | 2.7 (1.9-3.6; 3) | |
| MMRD: CD34+ selection | 3.1 (2.0-4.8; 9) | 1.92 (1.92-1.92; 1) | |
| MMRD: T-cell depletion | 14.6 (1.3-233.3; 8) | 3.1 (2.7-3.6; 2) | |
| MUD | 48.2 (0.5-226; 16) | 66 (1.7-146.7; 11) | |
| UCB | 13.6 (7.8-42.1; 18) | 20 (15.9-24.8; 10) | |
| Conditioning, n (%) | .027 | ||
| None | 22 (32) | 2 (7) | |
| Immunosuppression (serotherapy) only | 10 (15) | 3 (10) | |
| RIC | 19 (28) | 13 (43) | |
| MAC (busulfan > 12 mg/kg) | 17 (25) | 12 (40) | |
| GVHD prophylaxis, n (%) | .020 | ||
| None | 5 (7) | 0 | |
| Ex vivo T-cell depletion | 23 (34) | 4 (13) | |
| Calcineurin inhibitor with or without other agents | 40 (59) | 26 (87) |
Bold indicates statistically significant.
Patients with RD excluded.