Patient demographics for both the phase 2 cohort and the CIBMTR comparator cohort
| Characteristic . | Phase 2 cohort (n = 51) . | CIBMTR cohort (n = 1865) . |
|---|---|---|
| Patients | ||
| Median age at HCT, y (range) | 11.73 (0.67-21.79) | 12.24 (0.34-21.96) |
| Sex, n (%) | ||
| Male | 31 (61) | 1098 (60) |
| Female | 20 (39) | 730 (40) |
| Donor, n (%) | ||
| Median age, y (range) | 35 (6-61) | 23.4 (0-60) |
| Donor sex | ||
| Male | 29 (57) | 1043 (57) |
| Female | 22 (43) | 769 (42) |
| Data missing | — | 16 (1) |
| Donor/patient relations, n (%) | ||
| Sex mismatch (F≥M) | 12 (23.5) | 446 (24.4) |
| Donor relationship, n (%) | ||
| Parent; maternal | 37 (72.5); 16 (43) | 0 |
| Sibling | 12 (23.5) | 527 (29) |
| Other related | 2 (4) | 0 |
| Unrelated | 0 | 886 (48) |
| CB | 0 | 415 (23) |
| Disease status, n (%) | ||
| ALL CR1 | 16 (31.4) | 475 (26) |
| ALL CR2 | 14 (27.5) | 487 (26.6) |
| AML CR1 | 11 (21.6) | 617 (33.8) |
| AML CR2 | 5 (9.8) | 249 (13.6) |
| MDS | 5 (9.8) | 0 |
| Cell source | ||
| Haplo (5/10, 6/10, 7/10, 8/10) | 51 | 0 |
| Matched sibling donor | 0 | 527 (29) |
| MURD (9/10, 10/10) | 0 | 670 (37) |
| MMURD (7/8) | 0 | 216 (12) |
| CB 8/8 | 0 | 47 (3) |
| CB 7/8 | — | 75 (4) |
| CB ≤6/8 | — | 199 (11) |
| CB multidonor | — | 82 (4) |
| CB missing | — | 5 (<1) |
| CMV | ||
| Patient | ||
| Positive | 27 (53) | 1056 (58) |
| Negative | 24 (47) | 564 (31) |
| Data missing | 0 | 208 (11) |
| Donor | ||
| Positive | 27 (53) | 734 (40) |
| Negative | 24 (47) | 886 (48) |
| Data missing | 0 | 208 (11) |
| Conditioning regimens, n (%) | ||
| MAC | 27 (53) | 1828 |
| TBI | 16 (31) | — |
| Busulfan | 11 (22) | — |
| RTC | 24 (47) | 0 |
| rATG/flu/mel/thio | 22 (43) | — |
| TLI | 2 (4) | — |
| NK alloreactivity | ||
| Ligand mismatch (yes/no) | 44/7 | — |
| B-content value 0-1 vs ≥2 | 20/31 | — |
| Race | ||
| Black | 5 (10) | 141 (7.7) |
| Asian | 9 (17.6) | 151 (8.3) |
| White | 24 (47) | 1344 (73.5) |
| Other | 13 (25.5) | 192 (10.5) |
| Ethnicity | ||
| Non-Hispanic | 24 (47) | 1196 (65.4) |
| Hispanic | 23 (45.1) | 554 (30.3) |
| Other | 4 (7.8) | 78 (4.3) |
| Follow-up | ||
| Median follow-up, d (range) | 609 (29-1772) | 740 (42-1961) |
| Median cell dose infused/recipient weight (range) ×106/kg | ||
| CD34+ cells (n = 51) | 12.19 (2.57-36.4) | — |
| TCR-αβ cells (n = 51) | 0.02845 (0-0.418) | — |
| TCR-γδ cells (n = 51) | 7.739 (0.1077-1810) | — |
| NK cells (n = 47) | 41.13 (3.41-193.3) | — |
| CD20+ cells (n = 51) | 0.06435 (0.007-2.756) | — |
| Characteristic . | Phase 2 cohort (n = 51) . | CIBMTR cohort (n = 1865) . |
|---|---|---|
| Patients | ||
| Median age at HCT, y (range) | 11.73 (0.67-21.79) | 12.24 (0.34-21.96) |
| Sex, n (%) | ||
| Male | 31 (61) | 1098 (60) |
| Female | 20 (39) | 730 (40) |
| Donor, n (%) | ||
| Median age, y (range) | 35 (6-61) | 23.4 (0-60) |
| Donor sex | ||
| Male | 29 (57) | 1043 (57) |
| Female | 22 (43) | 769 (42) |
| Data missing | — | 16 (1) |
| Donor/patient relations, n (%) | ||
| Sex mismatch (F≥M) | 12 (23.5) | 446 (24.4) |
| Donor relationship, n (%) | ||
| Parent; maternal | 37 (72.5); 16 (43) | 0 |
| Sibling | 12 (23.5) | 527 (29) |
| Other related | 2 (4) | 0 |
| Unrelated | 0 | 886 (48) |
| CB | 0 | 415 (23) |
| Disease status, n (%) | ||
| ALL CR1 | 16 (31.4) | 475 (26) |
| ALL CR2 | 14 (27.5) | 487 (26.6) |
| AML CR1 | 11 (21.6) | 617 (33.8) |
| AML CR2 | 5 (9.8) | 249 (13.6) |
| MDS | 5 (9.8) | 0 |
| Cell source | ||
| Haplo (5/10, 6/10, 7/10, 8/10) | 51 | 0 |
| Matched sibling donor | 0 | 527 (29) |
| MURD (9/10, 10/10) | 0 | 670 (37) |
| MMURD (7/8) | 0 | 216 (12) |
| CB 8/8 | 0 | 47 (3) |
| CB 7/8 | — | 75 (4) |
| CB ≤6/8 | — | 199 (11) |
| CB multidonor | — | 82 (4) |
| CB missing | — | 5 (<1) |
| CMV | ||
| Patient | ||
| Positive | 27 (53) | 1056 (58) |
| Negative | 24 (47) | 564 (31) |
| Data missing | 0 | 208 (11) |
| Donor | ||
| Positive | 27 (53) | 734 (40) |
| Negative | 24 (47) | 886 (48) |
| Data missing | 0 | 208 (11) |
| Conditioning regimens, n (%) | ||
| MAC | 27 (53) | 1828 |
| TBI | 16 (31) | — |
| Busulfan | 11 (22) | — |
| RTC | 24 (47) | 0 |
| rATG/flu/mel/thio | 22 (43) | — |
| TLI | 2 (4) | — |
| NK alloreactivity | ||
| Ligand mismatch (yes/no) | 44/7 | — |
| B-content value 0-1 vs ≥2 | 20/31 | — |
| Race | ||
| Black | 5 (10) | 141 (7.7) |
| Asian | 9 (17.6) | 151 (8.3) |
| White | 24 (47) | 1344 (73.5) |
| Other | 13 (25.5) | 192 (10.5) |
| Ethnicity | ||
| Non-Hispanic | 24 (47) | 1196 (65.4) |
| Hispanic | 23 (45.1) | 554 (30.3) |
| Other | 4 (7.8) | 78 (4.3) |
| Follow-up | ||
| Median follow-up, d (range) | 609 (29-1772) | 740 (42-1961) |
| Median cell dose infused/recipient weight (range) ×106/kg | ||
| CD34+ cells (n = 51) | 12.19 (2.57-36.4) | — |
| TCR-αβ cells (n = 51) | 0.02845 (0-0.418) | — |
| TCR-γδ cells (n = 51) | 7.739 (0.1077-1810) | — |
| NK cells (n = 47) | 41.13 (3.41-193.3) | — |
| CD20+ cells (n = 51) | 0.06435 (0.007-2.756) | — |
CMV cytomegalovirus; TLI, total lymphoid irradiation.