Baseline ADA-SCID and transplant/GT characteristics of contemporary cohort receiving either HCT or GT as FDCT after 2000 and without an active infection at the time of cellular therapy
| . | HCT (n = 33) . | GT (n = 33) . | P . |
|---|---|---|---|
| Sex | .80† | ||
| Female | 22 (66.7%) | 21 (63.6%) | |
| Male | 11 (33.3%) | 12 (36.4%) | |
| Age at diagnosis (d) | .47‡ | ||
| Median (min-max) | 42 (0-4635) | 27 (0-1157) | |
| Decade of ADA diagnosis | .42*,† | ||
| 1990-1999 | 1 (3%) | 2 (6.1%) | |
| 2000-2009 | 10 (30.3%) | 5 (15.2%) | |
| 2010-2017 | 22 (66.7%) | 26 (78.8%) | |
| PIDTC protocol | .14* | ||
| 6901 | 19 (57.6%) | 13 (39.4%) | |
| 6902 | 14 (42.4%) | 20 (60.6%) | |
| SCID subtype | .52* | ||
| Typical SCID | 28 (84.8%) | 26 (78.8%) | |
| Leaky SCID | 5 (15.2%) | 7 (21.2%) | |
| Trigger for diagnosis | .08* | ||
| Family history | 8 (25%) | 3 (9.1%) | |
| Infection | 16 (50%) | 14 (42.4%) | |
| Newborn screening | 8 (25%) | 16 (48.5%) | |
| Missing | 1 | 0 | |
| Baseline PHA response | .58* | ||
| <10% lower limit of normal | 18 (81.8%) | 12 (80%) | |
| 10-30% lower limit of normal | 1 (4.5%) | 2 (13.3%) | |
| >30% lower limit of normal | 3 (13.6%) | 1 (6.7%) | |
| Missing | 11 | 18 | |
| Infectious status at time of ADA diagnosis | .40* | ||
| Active infection | 9 (29%) | 5 (15.2%) | |
| No previous infection | 16 (51.6%) | 20 (60.6%) | |
| Resolved infection | 6 (19.2%) | 8 (24.2%) | |
| Missing | 2 | 0 | |
| Need for supplemental oxygen§ | .61* | ||
| No | 22 (75.9%) | 22 (81.5%) | |
| Yes | 7 (24.1%) | 5 (18.5%) | |
| Missing | 4 | 6 | |
| Need for CPAP§ | .61*,† | ||
| No | 27 (96.4%) | 25 (92.6%) | |
| Yes | 1 (3.6%) | 2 (7.4%) | |
| Missing | 5 | 6 | |
| Need for mechanical ventilation§ | .68*,† | ||
| No | 28 (87.5%) | 24 (92.3%) | |
| Yes | 4 (12.5%) | 2 (7.7%) | |
| Missing | 1 | 7 | |
| Failure to thrive (reported by center)§ | .007* | ||
| No | 12 (42.9%) | 24 (77.4%) | |
| Yes | 16 (57.1%) | 7 (22.6%) | |
| Missing | 5 | 2 | |
| Decade of FDCT | .27* | ||
| 2000-2009 | 11 (33.3%) | 7 (21.2%) | |
| 2010-2017 | 22 (66.7%) | 26 (78.8%) | |
| Duration of ERT before FDCT (days) | .015‡ | ||
| Median (range) | 90 (20-2723) n = 15 patients | 202 (67-5268) | |
| Age at FDCT (days) | .002‡ | ||
| Median (range) | 135 (8-4783) | 317 (98-5601) | |
| HCT donor | N/A | ||
| HLA-matched sibling donor | 9 (27.3%) | N/A | |
| HLA-matched family donor | 3 (9.1%) | N/A | |
| Unrelated donor | 13 (39.4%) | N/A | |
| HLA-mismatched related donor (haploidentical) | 8 (24.2%) | N/A | |
| Graft type | N/A | ||
| Bone marrow | 21 (63.6%) | N/A | |
| Cord blood | 8 (24.2%) | N/A | |
| Peripheral blood stem cells | 4 (12.1%) | N/A | |
| Conditioning regimen intensity | <.001*,† | ||
| None | 15 (45.5%) | 0 (0%) | |
| Immune suppression only | 1 (3%) | 0 (0%) | |
| Reduced intensity | 6 (18.2%) | 33 (100%) | |
| Myeloablative | 11 (33.3%) | 0 (0%) | |
| Serotherapy in conditioning | N/A | ||
| ATG | 8 (24.2%) | N/A | |
| Alemtuzumab | 3 (9.1%) | N/A | |
| None | 22 (66.7%) | 33 (100%) |
| . | HCT (n = 33) . | GT (n = 33) . | P . |
|---|---|---|---|
| Sex | .80† | ||
| Female | 22 (66.7%) | 21 (63.6%) | |
| Male | 11 (33.3%) | 12 (36.4%) | |
| Age at diagnosis (d) | .47‡ | ||
| Median (min-max) | 42 (0-4635) | 27 (0-1157) | |
| Decade of ADA diagnosis | .42*,† | ||
| 1990-1999 | 1 (3%) | 2 (6.1%) | |
| 2000-2009 | 10 (30.3%) | 5 (15.2%) | |
| 2010-2017 | 22 (66.7%) | 26 (78.8%) | |
| PIDTC protocol | .14* | ||
| 6901 | 19 (57.6%) | 13 (39.4%) | |
| 6902 | 14 (42.4%) | 20 (60.6%) | |
| SCID subtype | .52* | ||
| Typical SCID | 28 (84.8%) | 26 (78.8%) | |
| Leaky SCID | 5 (15.2%) | 7 (21.2%) | |
| Trigger for diagnosis | .08* | ||
| Family history | 8 (25%) | 3 (9.1%) | |
| Infection | 16 (50%) | 14 (42.4%) | |
| Newborn screening | 8 (25%) | 16 (48.5%) | |
| Missing | 1 | 0 | |
| Baseline PHA response | .58* | ||
| <10% lower limit of normal | 18 (81.8%) | 12 (80%) | |
| 10-30% lower limit of normal | 1 (4.5%) | 2 (13.3%) | |
| >30% lower limit of normal | 3 (13.6%) | 1 (6.7%) | |
| Missing | 11 | 18 | |
| Infectious status at time of ADA diagnosis | .40* | ||
| Active infection | 9 (29%) | 5 (15.2%) | |
| No previous infection | 16 (51.6%) | 20 (60.6%) | |
| Resolved infection | 6 (19.2%) | 8 (24.2%) | |
| Missing | 2 | 0 | |
| Need for supplemental oxygen§ | .61* | ||
| No | 22 (75.9%) | 22 (81.5%) | |
| Yes | 7 (24.1%) | 5 (18.5%) | |
| Missing | 4 | 6 | |
| Need for CPAP§ | .61*,† | ||
| No | 27 (96.4%) | 25 (92.6%) | |
| Yes | 1 (3.6%) | 2 (7.4%) | |
| Missing | 5 | 6 | |
| Need for mechanical ventilation§ | .68*,† | ||
| No | 28 (87.5%) | 24 (92.3%) | |
| Yes | 4 (12.5%) | 2 (7.7%) | |
| Missing | 1 | 7 | |
| Failure to thrive (reported by center)§ | .007* | ||
| No | 12 (42.9%) | 24 (77.4%) | |
| Yes | 16 (57.1%) | 7 (22.6%) | |
| Missing | 5 | 2 | |
| Decade of FDCT | .27* | ||
| 2000-2009 | 11 (33.3%) | 7 (21.2%) | |
| 2010-2017 | 22 (66.7%) | 26 (78.8%) | |
| Duration of ERT before FDCT (days) | .015‡ | ||
| Median (range) | 90 (20-2723) n = 15 patients | 202 (67-5268) | |
| Age at FDCT (days) | .002‡ | ||
| Median (range) | 135 (8-4783) | 317 (98-5601) | |
| HCT donor | N/A | ||
| HLA-matched sibling donor | 9 (27.3%) | N/A | |
| HLA-matched family donor | 3 (9.1%) | N/A | |
| Unrelated donor | 13 (39.4%) | N/A | |
| HLA-mismatched related donor (haploidentical) | 8 (24.2%) | N/A | |
| Graft type | N/A | ||
| Bone marrow | 21 (63.6%) | N/A | |
| Cord blood | 8 (24.2%) | N/A | |
| Peripheral blood stem cells | 4 (12.1%) | N/A | |
| Conditioning regimen intensity | <.001*,† | ||
| None | 15 (45.5%) | 0 (0%) | |
| Immune suppression only | 1 (3%) | 0 (0%) | |
| Reduced intensity | 6 (18.2%) | 33 (100%) | |
| Myeloablative | 11 (33.3%) | 0 (0%) | |
| Serotherapy in conditioning | N/A | ||
| ATG | 8 (24.2%) | N/A | |
| Alemtuzumab | 3 (9.1%) | N/A | |
| None | 22 (66.7%) | 33 (100%) |
N/A, not applicable.
χ2 test.
Exact test.
Wilcoxon rank-sum test.
Need for supplemental oxygen, CPAP, mechanical ventilation, and failure to thrive refer to the presence or absence of these features between date of ADA diagnosis and the start of first definitive cellular therapy. In this analysis, however, all patients had no active infection at the time of starting first definitive cellular therapy.