Transplant-related characteristics and outcomes for 83 patients with SCID
| . | N . | Deaths . | 5-Year OS, % (95% CI) . | P . | Neurologic events . | 5-Year cumulative incidence of neurologic events, % (95% CI) . | P . | Neurologic event–free survivors . | 5-Year NEFS, % (95% CI) . | P . |
|---|---|---|---|---|---|---|---|---|---|---|
| Overall | 83 | 21 | 78 (69-87) | 8 | 11 (4-18) | 54 | 68 (58-78) | |||
| Year of HCT | .02 | |||||||||
| 1990-2004 | 40 | 16 | 65 (50-80) | 4 | 12 (1-22) | .81 | 20 | 57 (42-73) | .04 | |
| 2005-2016 | 43 | 5 | 88 (79-98) | 4 | 10 (1-20) | 34 | 79 (67-91) | |||
| Donor | .31 | |||||||||
| Matched related | 11 | 1 | 100 (70-100) | 0 | 0 (0-30) | .39 | 10 | 100 (70-100) | .11 | |
| Mismatched related | 58 | 17 | 74 (62-83) | 7 | 14 (4-23) | 34 | 62 (49-74) | |||
| Unrelated | 14 | 3 | 79 (57-100) | 1 | 10 (1-29) | 10 | 70 (47-95) | |||
| Conditioning | .27 | |||||||||
| None | 41 | 7 | 83 (71-94) | 7 | 19 (10-37) | .06 | 27 | 66 (51-80) | .98 | |
| Immunosuppression | 25 | 9 | 75 (58-93) | 0 | 0 (0-16) | 16 | 75 (58-93) | |||
| RIC/MAC | 17 | 5 | 68 (44-91) | 1 | 8 (2-51) | 11 | 62 (37-87) | |||
| GVHD prophylaxis | .24 | |||||||||
| T-cell depletion | 58 | 17 | 74 (62-85) | 7 | 14 (4-23) | .21 | 34 | 62 (49-74) | .07 | |
| CNI+/− others | 25 | 4 | 88 (75-100) | 1 | 5 (1-14) | 20 | 84 (69-98) | |||
| Acute GVHD grade II-IV* | .22 | |||||||||
| No | 51 | 9 | 85 (80-90) | 6 | 13 (8-18) | .55 | 36 | 74 (68-80) | .51 | |
| Yes | 28 | 8 | 73 (64-82) | 2 | 8 (3-13) | 18 | 67 (58-76) | |||
| >1 HCT | .09 | |||||||||
| No | 52 | 10 | 82 (71-93) | 5 | 10 (2-19) | .92 | 37 | 72 (60-85) | .14 | |
| Yes | 31 | 11 | 71 (55-87) | 3 | 12 (1-25) | 17 | 61 (44-78) |
| . | N . | Deaths . | 5-Year OS, % (95% CI) . | P . | Neurologic events . | 5-Year cumulative incidence of neurologic events, % (95% CI) . | P . | Neurologic event–free survivors . | 5-Year NEFS, % (95% CI) . | P . |
|---|---|---|---|---|---|---|---|---|---|---|
| Overall | 83 | 21 | 78 (69-87) | 8 | 11 (4-18) | 54 | 68 (58-78) | |||
| Year of HCT | .02 | |||||||||
| 1990-2004 | 40 | 16 | 65 (50-80) | 4 | 12 (1-22) | .81 | 20 | 57 (42-73) | .04 | |
| 2005-2016 | 43 | 5 | 88 (79-98) | 4 | 10 (1-20) | 34 | 79 (67-91) | |||
| Donor | .31 | |||||||||
| Matched related | 11 | 1 | 100 (70-100) | 0 | 0 (0-30) | .39 | 10 | 100 (70-100) | .11 | |
| Mismatched related | 58 | 17 | 74 (62-83) | 7 | 14 (4-23) | 34 | 62 (49-74) | |||
| Unrelated | 14 | 3 | 79 (57-100) | 1 | 10 (1-29) | 10 | 70 (47-95) | |||
| Conditioning | .27 | |||||||||
| None | 41 | 7 | 83 (71-94) | 7 | 19 (10-37) | .06 | 27 | 66 (51-80) | .98 | |
| Immunosuppression | 25 | 9 | 75 (58-93) | 0 | 0 (0-16) | 16 | 75 (58-93) | |||
| RIC/MAC | 17 | 5 | 68 (44-91) | 1 | 8 (2-51) | 11 | 62 (37-87) | |||
| GVHD prophylaxis | .24 | |||||||||
| T-cell depletion | 58 | 17 | 74 (62-85) | 7 | 14 (4-23) | .21 | 34 | 62 (49-74) | .07 | |
| CNI+/− others | 25 | 4 | 88 (75-100) | 1 | 5 (1-14) | 20 | 84 (69-98) | |||
| Acute GVHD grade II-IV* | .22 | |||||||||
| No | 51 | 9 | 85 (80-90) | 6 | 13 (8-18) | .55 | 36 | 74 (68-80) | .51 | |
| Yes | 28 | 8 | 73 (64-82) | 2 | 8 (3-13) | 18 | 67 (58-76) | |||
| >1 HCT | .09 | |||||||||
| No | 52 | 10 | 82 (71-93) | 5 | 10 (2-19) | .92 | 37 | 72 (60-85) | .14 | |
| Yes | 31 | 11 | 71 (55-87) | 3 | 12 (1-25) | 17 | 61 (44-78) |
CNI, calcineurin inhibitor; GVHD, graft-versus-host disease.
Of 79 engrafted patients.