EFS and OS for ADA-SCID
| . | Result (95% confidence interval) . | P . | Pairwise comparison P . |
|---|---|---|---|
| Five-year EFS for entire cohort by FDCT | |||
| ERT-GT | 75.3% (34.4%-92.7%) | .005 | ERT-GT vs ERT-HCT: P = .26 |
| ERT-HCT | 73% (53.1%-85.5%) | ERT-GT vs HCT: P < .01 | |
| HCT | 49.5% (34.9%-62.5%) | ERT-HCT vs HCT: P = .06 | |
| Five-year OS for entire cohort by FDCT | |||
| ERT-GT | 100% (NA) | .01 | ERT-GT vs ERT-HCT: P = .01 |
| ERT-HCT | 79.6% (60%-90.3%) | ERT-GT vs HCT: P < .01 | |
| HCT | 72.5% (57.7%-82.8%) | ERT-HCT vs HCT: P = .56 | |
| Five-year EFS for all transplant patients by donor type* | |||
| MSD/MFD | 90.5% (67%-97.5%) | .001 | MSD/MFD vs. URD/UCB: P = .1 |
| URD/UCB | 69.4% (46%-84.2%) | MSD/MFD vs. MMRD: P < .01 | |
| MMRD | 34.6% (19.7%-50.1%) | URD/UCB vs MMRD: P = .02 | |
| Five-year OS for all transplant patients by donor type* | |||
| MSD/MFD | 100% (NA) | .004 | MSD/MFD vs. URD/UCB: P = .03 |
| URD/UCB | 78.5% (55.7%-90.5%) | MSD/MFD vs. MMRD: P < .01 | |
| MMRD | 60.2% (42.6%-73.9%) | URD/UCB vs MMRD: P = .19 | |
| Five-year EFS for all transplant patients by age at transplant* | |||
| <3.5 mo | 71.6% (49.4%-85.3%) | .18 | NA |
| ≥3.5 mo | 51.9% (37.5%-64.4%) | ||
| Five-year OS for all transplant patients by age at transplant* | |||
| <3.5 mo | 91.6% (70.5%-97.9%) | .02 | NA |
| ≥3.5 mo | 67.9% (53.7%-78.6%) | ||
| Five-year EFS for all transplant patients by absence or presence of active infection at transplant* | |||
| Without infection | 68.2% (52.6%-79.6%) | .003 | NA |
| With infection | 33.1% (14.3%-53.4%) | ||
| Five-year OS for all transplant patients by absence or presence of active infection at transplant* | |||
| Without infection | 82.3% (67.3%-90.9%) | .02 | NA |
| With infection | 64.7% (43.1%-79.9%) |
| . | Result (95% confidence interval) . | P . | Pairwise comparison P . |
|---|---|---|---|
| Five-year EFS for entire cohort by FDCT | |||
| ERT-GT | 75.3% (34.4%-92.7%) | .005 | ERT-GT vs ERT-HCT: P = .26 |
| ERT-HCT | 73% (53.1%-85.5%) | ERT-GT vs HCT: P < .01 | |
| HCT | 49.5% (34.9%-62.5%) | ERT-HCT vs HCT: P = .06 | |
| Five-year OS for entire cohort by FDCT | |||
| ERT-GT | 100% (NA) | .01 | ERT-GT vs ERT-HCT: P = .01 |
| ERT-HCT | 79.6% (60%-90.3%) | ERT-GT vs HCT: P < .01 | |
| HCT | 72.5% (57.7%-82.8%) | ERT-HCT vs HCT: P = .56 | |
| Five-year EFS for all transplant patients by donor type* | |||
| MSD/MFD | 90.5% (67%-97.5%) | .001 | MSD/MFD vs. URD/UCB: P = .1 |
| URD/UCB | 69.4% (46%-84.2%) | MSD/MFD vs. MMRD: P < .01 | |
| MMRD | 34.6% (19.7%-50.1%) | URD/UCB vs MMRD: P = .02 | |
| Five-year OS for all transplant patients by donor type* | |||
| MSD/MFD | 100% (NA) | .004 | MSD/MFD vs. URD/UCB: P = .03 |
| URD/UCB | 78.5% (55.7%-90.5%) | MSD/MFD vs. MMRD: P < .01 | |
| MMRD | 60.2% (42.6%-73.9%) | URD/UCB vs MMRD: P = .19 | |
| Five-year EFS for all transplant patients by age at transplant* | |||
| <3.5 mo | 71.6% (49.4%-85.3%) | .18 | NA |
| ≥3.5 mo | 51.9% (37.5%-64.4%) | ||
| Five-year OS for all transplant patients by age at transplant* | |||
| <3.5 mo | 91.6% (70.5%-97.9%) | .02 | NA |
| ≥3.5 mo | 67.9% (53.7%-78.6%) | ||
| Five-year EFS for all transplant patients by absence or presence of active infection at transplant* | |||
| Without infection | 68.2% (52.6%-79.6%) | .003 | NA |
| With infection | 33.1% (14.3%-53.4%) | ||
| Five-year OS for all transplant patients by absence or presence of active infection at transplant* | |||
| Without infection | 82.3% (67.3%-90.9%) | .02 | NA |
| With infection | 64.7% (43.1%-79.9%) |
P value is from a log-rank test.
MMFD, mismatched family donor (haploidentical); NA, not applicable.
Includes all allogeneic hematopoietic cell transplant patients, including those who did not (HCT) and those who did (ERT-HCT) receive pretransplant ERT.