Patient and transplantation characteristics
| . | Entire cohort n = 306 . | TCRαβ-HSCT n = 167 . | PTCY-HSCT n = 139 . | P value . | |||
|---|---|---|---|---|---|---|---|
| Missing data . | Results . | Missing data . | Results . | Missing data . | Results . | ||
| Year of HSCT, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | <.001∗ | |||
| 2011 to 2015 | 114 (37.3) | 79 (47.3) | 35 (25.2) | ||||
| 2016 to 2019 | 192 (62.7) | 88 (52.7) | 104 (74.8) | ||||
| Median age at HSCT (range), y | 0 (0.0) | 1.2 (0.03 - 19.6) | 0 (0.0) | 1.1 (0.03-18.0) | 0 (0.0) | 1.4 (0.2-19.6) | .213‡ |
| Diagnosis, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | .208∗ | |||
| SCID | 123 (40.2) | 73 (43.7) | 50 (36.0) | ||||
| Non-SCID IEI | 183 (59.8) | 94 (56.3) | 89 (64.0) | ||||
| Performance score, n (%) | 71 (23.2) | 28 (16.8) | 43 (30.9) | .341∗ | |||
| ≥ 80 | 187 (79.6) | 114 (82.0) | 73 (76.0) | ||||
| < 80 | 48 (20.4) | 25 (18.0) | 23 (24.0) | ||||
| Pre-HSCT infection, n (%) | 0 (0.0) | 240 (78.4) | 0 (0.0) | 122 (73.1) | 0 (0.0) | 118 (84.9) | .018∗ |
| Pre-HSCT organ damage, n (%) | 5 (1.6) | 121 (40.2) | 3 (1.8) | 47 (28.7) | 2 (1.4) | 74 (54.0) | <.001∗ |
| Pre-HSCT autoimmunity, n (%) | 27 (8.8) | 82 (29.4) | 11 (6.6) | 44 (28.2) | 16 (11.5) | 38 (30.9) | .721∗ |
| Pre-HSCT malignancy, n (%) | 0 (0.0) | 21 (6.9) | 0 (0.0) | 11 (6.6) | 0 (0.0) | 10 (7.2) | >.999 |
| Pre-HSCT morbidity score, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | .002∗ | |||
| 0 to 1 | 159 (52.0) | 101 (60.5) | 58 (41.7) | ||||
| ≥ 2 | 147 (48.0) | 66 (39.5) | 81 (58.3) | ||||
| Donor, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | >.999∗ | |||
| MMUD | 15 (4.9) | 8 (4.8) | 7 (5.0) | ||||
| MMFD | 291 (95.1) | 159 (95.2) | 132 (95.0) | ||||
| HLA matching, n (%) | 52 (17.0) | 11 (6.6) | 41 (29.5) | .141† | |||
| 9/10 | 19 (7.5) | 13 (8.3) | 6 (6.1) | ||||
| 8/10 | 7 (2.8) | 7 (4.5) | 0 (0.0) | ||||
| ≤7/10 | 217 (85.4) | 130 (83.3) | 87 (88.8) | ||||
| Other/incomplete information§ | 11 (4.3) | 6 (3.8) | 5 (5.1) | ||||
| Recipient/donor CMV serostatus, n (%) | 108 (35.3) | 41 (24.6) | 67 (48.2) | .098∗ | |||
| −/− | 29 (14.6) | 14 (11.1) | 15 (20.8) | ||||
| −/+, +/−, +/+ | 169 (85.4) | 112 (88.9) | 57 (79.2) | ||||
| Conditioning, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | <.001∗ | |||
| Busulfan-based | 112 (36.6) | 22 (13.2) | 90 (64.7) | ||||
| Treosulfan-based | 115 (37.6) | 99 (59.3) | 16 (11.5) | ||||
| Other/none | 79 (25.8) | 46 (27.5) | 33 (23.7) | ||||
| Serotherapy, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | <.001† | |||
| None | 77 (25.2) | 46 (27.5) | 31 (22.3) | ||||
| ATG only | 175 (57.2) | 113 (67.7) | 62 (44.6) | ||||
| Alemtuzumab only | 53 (17.3) | 7 (4.2) | 46 (33.1) | ||||
| ATG and alemtuzumab | 1 (0.3) | 1 (0.6) | 0 (0.0) | ||||
| Rituximab, n (%) | 0 (0.0) | 108 (35.3) | 0 (0.0) | 69 (41.3) | 0 (0.0) | 39 (28.1) | .022∗ |
| Post-HSCT GvHD prophylaxis, n (%) | 34 (11.1) | 32 (19.2) | 2 (1.4) | <.001∗ | |||
| None | 41 (15.1) | 41 (30.4) | 0 (0.0) | ||||
| CNI | 43 (15.8) | 34 (25.2) | 9 (6.6) | ||||
| CNI+MMF | 141 (51.8) | 19 (14.1) | 122 (89.1) | ||||
| MMF | 25 (9.2) | 23 (17.0) | 2 (1.5) | ||||
| Others | 22 (8.1) | 18 (13.3) | 4 (2.9) | ||||
| Stem cell source, n (%) | 2 (0.7) | 2 (1.2) | 0 (0.0) | <.001† | |||
| BM | 116 (38.2) | 3 (1.8) | 113 (81.3) | ||||
| PBSC | 182 (59.9) | 162 (98.2) | 20 (14.4) | ||||
| BM+PBSC | 6 (2.0) | 0 (0.0) | 6 (4.3) | ||||
| Median TNC (range), × 108/kg | 32 (10.5) | 10.6 (2.0-100.0) | 14 (8.4) | 12.3 (2.0-100.0) | 18 (12.9) | 8.4 (2.1-58.0) | <.001‡ |
| Median CD34+ cell dose (range), × 106/kg | 17 (5.6) | 13.0 (1.6-56.6) | 9 (5.4) | 18.4 (2.0-56.6) | 8 (5.8) | 8.7 (1.6-47.7) | <.001‡ |
| Median CD3+ cell dose, range, × 106/kg | 85 (27.8) | 25.0 (0.0-993.0) | 37 (22.2) | 19.9 (0.0-557.0) | 48 (34.5) | 60.7 (0.4-993.0) | <.001‡ |
| . | Entire cohort n = 306 . | TCRαβ-HSCT n = 167 . | PTCY-HSCT n = 139 . | P value . | |||
|---|---|---|---|---|---|---|---|
| Missing data . | Results . | Missing data . | Results . | Missing data . | Results . | ||
| Year of HSCT, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | <.001∗ | |||
| 2011 to 2015 | 114 (37.3) | 79 (47.3) | 35 (25.2) | ||||
| 2016 to 2019 | 192 (62.7) | 88 (52.7) | 104 (74.8) | ||||
| Median age at HSCT (range), y | 0 (0.0) | 1.2 (0.03 - 19.6) | 0 (0.0) | 1.1 (0.03-18.0) | 0 (0.0) | 1.4 (0.2-19.6) | .213‡ |
| Diagnosis, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | .208∗ | |||
| SCID | 123 (40.2) | 73 (43.7) | 50 (36.0) | ||||
| Non-SCID IEI | 183 (59.8) | 94 (56.3) | 89 (64.0) | ||||
| Performance score, n (%) | 71 (23.2) | 28 (16.8) | 43 (30.9) | .341∗ | |||
| ≥ 80 | 187 (79.6) | 114 (82.0) | 73 (76.0) | ||||
| < 80 | 48 (20.4) | 25 (18.0) | 23 (24.0) | ||||
| Pre-HSCT infection, n (%) | 0 (0.0) | 240 (78.4) | 0 (0.0) | 122 (73.1) | 0 (0.0) | 118 (84.9) | .018∗ |
| Pre-HSCT organ damage, n (%) | 5 (1.6) | 121 (40.2) | 3 (1.8) | 47 (28.7) | 2 (1.4) | 74 (54.0) | <.001∗ |
| Pre-HSCT autoimmunity, n (%) | 27 (8.8) | 82 (29.4) | 11 (6.6) | 44 (28.2) | 16 (11.5) | 38 (30.9) | .721∗ |
| Pre-HSCT malignancy, n (%) | 0 (0.0) | 21 (6.9) | 0 (0.0) | 11 (6.6) | 0 (0.0) | 10 (7.2) | >.999 |
| Pre-HSCT morbidity score, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | .002∗ | |||
| 0 to 1 | 159 (52.0) | 101 (60.5) | 58 (41.7) | ||||
| ≥ 2 | 147 (48.0) | 66 (39.5) | 81 (58.3) | ||||
| Donor, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | >.999∗ | |||
| MMUD | 15 (4.9) | 8 (4.8) | 7 (5.0) | ||||
| MMFD | 291 (95.1) | 159 (95.2) | 132 (95.0) | ||||
| HLA matching, n (%) | 52 (17.0) | 11 (6.6) | 41 (29.5) | .141† | |||
| 9/10 | 19 (7.5) | 13 (8.3) | 6 (6.1) | ||||
| 8/10 | 7 (2.8) | 7 (4.5) | 0 (0.0) | ||||
| ≤7/10 | 217 (85.4) | 130 (83.3) | 87 (88.8) | ||||
| Other/incomplete information§ | 11 (4.3) | 6 (3.8) | 5 (5.1) | ||||
| Recipient/donor CMV serostatus, n (%) | 108 (35.3) | 41 (24.6) | 67 (48.2) | .098∗ | |||
| −/− | 29 (14.6) | 14 (11.1) | 15 (20.8) | ||||
| −/+, +/−, +/+ | 169 (85.4) | 112 (88.9) | 57 (79.2) | ||||
| Conditioning, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | <.001∗ | |||
| Busulfan-based | 112 (36.6) | 22 (13.2) | 90 (64.7) | ||||
| Treosulfan-based | 115 (37.6) | 99 (59.3) | 16 (11.5) | ||||
| Other/none | 79 (25.8) | 46 (27.5) | 33 (23.7) | ||||
| Serotherapy, n (%) | 0 (0.0) | 0 (0.0) | 0 (0.0) | <.001† | |||
| None | 77 (25.2) | 46 (27.5) | 31 (22.3) | ||||
| ATG only | 175 (57.2) | 113 (67.7) | 62 (44.6) | ||||
| Alemtuzumab only | 53 (17.3) | 7 (4.2) | 46 (33.1) | ||||
| ATG and alemtuzumab | 1 (0.3) | 1 (0.6) | 0 (0.0) | ||||
| Rituximab, n (%) | 0 (0.0) | 108 (35.3) | 0 (0.0) | 69 (41.3) | 0 (0.0) | 39 (28.1) | .022∗ |
| Post-HSCT GvHD prophylaxis, n (%) | 34 (11.1) | 32 (19.2) | 2 (1.4) | <.001∗ | |||
| None | 41 (15.1) | 41 (30.4) | 0 (0.0) | ||||
| CNI | 43 (15.8) | 34 (25.2) | 9 (6.6) | ||||
| CNI+MMF | 141 (51.8) | 19 (14.1) | 122 (89.1) | ||||
| MMF | 25 (9.2) | 23 (17.0) | 2 (1.5) | ||||
| Others | 22 (8.1) | 18 (13.3) | 4 (2.9) | ||||
| Stem cell source, n (%) | 2 (0.7) | 2 (1.2) | 0 (0.0) | <.001† | |||
| BM | 116 (38.2) | 3 (1.8) | 113 (81.3) | ||||
| PBSC | 182 (59.9) | 162 (98.2) | 20 (14.4) | ||||
| BM+PBSC | 6 (2.0) | 0 (0.0) | 6 (4.3) | ||||
| Median TNC (range), × 108/kg | 32 (10.5) | 10.6 (2.0-100.0) | 14 (8.4) | 12.3 (2.0-100.0) | 18 (12.9) | 8.4 (2.1-58.0) | <.001‡ |
| Median CD34+ cell dose (range), × 106/kg | 17 (5.6) | 13.0 (1.6-56.6) | 9 (5.4) | 18.4 (2.0-56.6) | 8 (5.8) | 8.7 (1.6-47.7) | <.001‡ |
| Median CD3+ cell dose, range, × 106/kg | 85 (27.8) | 25.0 (0.0-993.0) | 37 (22.2) | 19.9 (0.0-557.0) | 48 (34.5) | 60.7 (0.4-993.0) | <.001‡ |
Boldface indicates significant P values.
ATG, antithymocyte globulin; BM, bone marrow; CNI, calcineurin inhibitors; MMF, mycophenolate mofetil; MMFD, mismatched donor; MMUD, mismatched unrelated donor; TNC, total nucleated cell dose.
Calculated via the χ2 test.
Calculated via Fisher exact test.
Calculated via the Wilcoxon rank sum test.
The other HLA matching scores (and counts in the entire cohort) are: 4/6 (n = 5); 6/8 (n = 3); 7/8 (n = 2); 9/12 (n = 1).