Univariate analysis for OS, EFS, GEFS, and acute and chronic GvHD
| . | OS (95% CI) . | EFS∗ (95% CI) . | GEFS† (95% CI) . | Grade II-IV aGvHD (95% CI) . | Grade III-IV aGvHD (95% CI) . | cGvHD (95% CI) . | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1-y . | 3-y . | P value‡ . | 1-y . | 3-y . | P value‡ . | 1-y . | 3-y . | P value‡ . | Day 100 . | P value§ . | Day 100 . | P value§ . | 1-year . | P value§ . | |
| Method | .013 | .804 | .080 | .009 | .007 | .173 | |||||||||
| TCRαβ-HSCT (n = 167) | 82% (76%-88%) | 78% (71%-84%) | 62% (54%-70%) | 58% (50%-66%) | 54% (46%-62%) | 53% (44%-61%) | 16% (10%-22%) | 6% (2%-9%) | 7% (3%-11%) | ||||||
| PTCY-HSCT (n = 139) | 71% (63%-78%) | 66% (57%-74%) | 60% (52%-69%) | 57% (48%-66%) | 46% (38%-55%) | 41% (32%-50%) | 30% (22%-38%) | 15% (9%-21%) | 11% (6%-17%) | ||||||
| Year of HSCT | .367 | .768 | .360 | .989 | .238 | .859 | |||||||||
| 2011 to 2015 (n = 114) | 81% (73%-88%) | 75% (67%-83%) | 65% (55%-74%) | 58% (49%-68%) | 54% (45%-64%) | 49% (39%-59%) | 22% (15%-30%) | 7% (2%-12%) | 10% (5%-16%) | ||||||
| 2016 to 2019 (n = 192) | 74% (68%-81%) | 70% (63%-77%) | 59% (52%-67%) | 57% (49%-65%) | 48% (41%-56%) | 46% (39%-54%) | 22% (16%-28%) | 11% (7%-16%) | 8% (4%-12%) | ||||||
| Age at HSCT | .665 | .691 | .547 | .921 | .590 | .382 | |||||||||
| <5 y (n = 235) | 77% (71%-82%) | 72% (66%-78%) | 61% (55%-68%) | 57% (50%-64%) | 52% (45%-58%) | 49% (42%-56%) | 22% (17%-28%) | 9% (5%-13%) | 9% (5%-12%) | ||||||
| >5 y (n = 71) | 77% (68%-87%) | 74% (63%-84%) | 62% (50%-75%) | 60% (47%-73%) | 46% (34%-59%) | 42% (30%-55%) | 23% (13%-32%) | 11% (4%-19%) | 11% (3%-18%) | ||||||
| Diagnosis | .084 | .197 | .258 | .589 | .831 | .692 | |||||||||
| Non-SCID (n = 183) | 81% (75%-87%) | 75% (68%-82%) | 64% (56%-71%) | 61% (54%-69%) | 53% (45%-60%) | 50% (42%-57%) | 24% (18%-30%) | 9% (5%-14%) | 9% (5%-13%) | ||||||
| SCID (n=123) | 70% (62%-78%) | 68% (59%-76%) | 58% (49%-67%) | 52% (42%-62%) | 48% (38%-57%) | 44% (35%-54%) | 20% (13%-27%) | 10% (5%-15%) | 9% (4%-14%) | ||||||
| Pre-HSCT infection | .002 | .108 | .020 | .394 | .121 | .337 | |||||||||
| No (n = 66) | 92% (86%-99%) | 87% (78%-96%) | 72% (60%-84%) | 67% (54%-80%) | 66% (54%-78%) | 58% (45%-72%) | 19% (9%-28%) | 5% (0%-10%) | 6% (0%-12%) | ||||||
| Yes (n = 240) | 73% (67%-78%) | 68% (62%-74%) | 59% (52%-65%) | 55% (48%-62%) | 47% (40%-53%) | 44% (38%-51%) | 23% (18%-29%) | 11% (7%-15%) | 10% (6%-14%) | ||||||
| Pre-HSCT organ damage | .002 | .090 | .018 | .283 | .031 | .615 | |||||||||
| No (n = 180) | 84% (78%-89%) | 80% (74%-86%) | 68% (61%-76%) | 63% (55%-71%) | 57% (49%-65%) | 53% (45%-61%) | 20% (14%-26%) | 7% (3%-11%) | 9% (4%-13%) | ||||||
| Yes (n = 121) | 67% (59%-76%) | 63% (54%-72%) | 54% (45%-63%) | 53% (44%-62%) | 43% (34%-52%) | 41% (32%-50%) | 26% (18%-34%) | 14% (8%-21%) | 10% (4%-16%) | ||||||
| Pre-HSCT autoimmunity | .517 | .230 | .088 | .127 | .221 | .333 | |||||||||
| No (n = 197) | 79% (74%-85%) | 75% (69%-81%) | 65% (58%-72%) | 61% (53%-68%) | 54% (47%-62%) | 51% (43%-58%) | 20% (14%-25%) | 8% (4%-12%) | 10% (6%-14%) | ||||||
| Yes (n = 82) | 77% (67%-86%) | 72% (61%-82%) | 59% (47%-70%) | 55% (44%-67%) | 47% (36%-59%) | 45% (34%-57%) | 27% (17%-37%) | 12% (5%-20%) | 6% (0%-11%) | ||||||
| Pre-HSCT malignancy | .657 | .342 | .740 | .702 | .453 | .483 | |||||||||
| No (n = 285) | 76% (72%-81%) | 72% (66%-77%) | 61% (55%-67%) | 57% (50%-63%) | 50% (44%-57%) | 47% (41%-53%) | 23% (18%-27%) | 9% (6%-13%) | 9% (5%-12%) | ||||||
| Yes (n = 21) | 81% (64%-98%) | 76% (57%-94%) | 68% (48%-89%) | 68% (48%-89%) | 53% (30%-75%) | 53% (30%-75%) | 19% (2%-36%) | 14% (0%-29%) | 15% (0%-31%) | ||||||
| Pre-HSCT morbidity score|| | <.001 | .031 | .010 | .755 | .042 | .853 | |||||||||
| 0 to 1 (n = 159) | 87% (82%-92%) | 83% (76%-89%) | 69% (61%-77%) | 63% (54%-72%) | 57% (49%-65%) | 52% (44%-61%) | 21% (15%-28%) | 6% (3%-10%) | 9% (4%-14%) | ||||||
| ≥2 (n = 147) | 66% (58%-73%) | 61% (53%-69%) | 54% (46%-62%) | 52% (44%-61%) | 44% (36%-52%) | 42% (34%-51%) | 24% (17%-31%) | 13% (8%-19%) | 9% (4%-14%) | ||||||
| Conditioning | <.001 | <.001 | <.001 | .123 | .001 | .646 | |||||||||
| Busulfan-based (n = 112) | 79% (71%-86%) | 78% (70%-85%) | 68% (58%-77%) | 68% (58%-77%) | 54% (44%-64%) | 51% (41%-61%) | 25% (17%-33%) | 14% (7%-20%) | 7% (2%-11%) | ||||||
| Treosulfan-based (n = 115) | 87% (81%-93%) | 83% (76%-90%) | 68% (58%-77%) | 63% (53%-73%) | 57% (48%-67%) | 55% (45%-65%) | 16% (9%-23%) | 2% (0%-4%) | 11% (5%-17%) | ||||||
| Other/none (n = 79) | 60% (49%-71%) | 49% (37%-61%) | 45% (34%-56%) | 38% (27%-50%) | 37% (26%-48%) | 32% (21%-43%) | 28% (18%-38%) | 16% (8%-24%) | 10% (3%-17%) | ||||||
| . | OS (95% CI) . | EFS∗ (95% CI) . | GEFS† (95% CI) . | Grade II-IV aGvHD (95% CI) . | Grade III-IV aGvHD (95% CI) . | cGvHD (95% CI) . | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1-y . | 3-y . | P value‡ . | 1-y . | 3-y . | P value‡ . | 1-y . | 3-y . | P value‡ . | Day 100 . | P value§ . | Day 100 . | P value§ . | 1-year . | P value§ . | |
| Method | .013 | .804 | .080 | .009 | .007 | .173 | |||||||||
| TCRαβ-HSCT (n = 167) | 82% (76%-88%) | 78% (71%-84%) | 62% (54%-70%) | 58% (50%-66%) | 54% (46%-62%) | 53% (44%-61%) | 16% (10%-22%) | 6% (2%-9%) | 7% (3%-11%) | ||||||
| PTCY-HSCT (n = 139) | 71% (63%-78%) | 66% (57%-74%) | 60% (52%-69%) | 57% (48%-66%) | 46% (38%-55%) | 41% (32%-50%) | 30% (22%-38%) | 15% (9%-21%) | 11% (6%-17%) | ||||||
| Year of HSCT | .367 | .768 | .360 | .989 | .238 | .859 | |||||||||
| 2011 to 2015 (n = 114) | 81% (73%-88%) | 75% (67%-83%) | 65% (55%-74%) | 58% (49%-68%) | 54% (45%-64%) | 49% (39%-59%) | 22% (15%-30%) | 7% (2%-12%) | 10% (5%-16%) | ||||||
| 2016 to 2019 (n = 192) | 74% (68%-81%) | 70% (63%-77%) | 59% (52%-67%) | 57% (49%-65%) | 48% (41%-56%) | 46% (39%-54%) | 22% (16%-28%) | 11% (7%-16%) | 8% (4%-12%) | ||||||
| Age at HSCT | .665 | .691 | .547 | .921 | .590 | .382 | |||||||||
| <5 y (n = 235) | 77% (71%-82%) | 72% (66%-78%) | 61% (55%-68%) | 57% (50%-64%) | 52% (45%-58%) | 49% (42%-56%) | 22% (17%-28%) | 9% (5%-13%) | 9% (5%-12%) | ||||||
| >5 y (n = 71) | 77% (68%-87%) | 74% (63%-84%) | 62% (50%-75%) | 60% (47%-73%) | 46% (34%-59%) | 42% (30%-55%) | 23% (13%-32%) | 11% (4%-19%) | 11% (3%-18%) | ||||||
| Diagnosis | .084 | .197 | .258 | .589 | .831 | .692 | |||||||||
| Non-SCID (n = 183) | 81% (75%-87%) | 75% (68%-82%) | 64% (56%-71%) | 61% (54%-69%) | 53% (45%-60%) | 50% (42%-57%) | 24% (18%-30%) | 9% (5%-14%) | 9% (5%-13%) | ||||||
| SCID (n=123) | 70% (62%-78%) | 68% (59%-76%) | 58% (49%-67%) | 52% (42%-62%) | 48% (38%-57%) | 44% (35%-54%) | 20% (13%-27%) | 10% (5%-15%) | 9% (4%-14%) | ||||||
| Pre-HSCT infection | .002 | .108 | .020 | .394 | .121 | .337 | |||||||||
| No (n = 66) | 92% (86%-99%) | 87% (78%-96%) | 72% (60%-84%) | 67% (54%-80%) | 66% (54%-78%) | 58% (45%-72%) | 19% (9%-28%) | 5% (0%-10%) | 6% (0%-12%) | ||||||
| Yes (n = 240) | 73% (67%-78%) | 68% (62%-74%) | 59% (52%-65%) | 55% (48%-62%) | 47% (40%-53%) | 44% (38%-51%) | 23% (18%-29%) | 11% (7%-15%) | 10% (6%-14%) | ||||||
| Pre-HSCT organ damage | .002 | .090 | .018 | .283 | .031 | .615 | |||||||||
| No (n = 180) | 84% (78%-89%) | 80% (74%-86%) | 68% (61%-76%) | 63% (55%-71%) | 57% (49%-65%) | 53% (45%-61%) | 20% (14%-26%) | 7% (3%-11%) | 9% (4%-13%) | ||||||
| Yes (n = 121) | 67% (59%-76%) | 63% (54%-72%) | 54% (45%-63%) | 53% (44%-62%) | 43% (34%-52%) | 41% (32%-50%) | 26% (18%-34%) | 14% (8%-21%) | 10% (4%-16%) | ||||||
| Pre-HSCT autoimmunity | .517 | .230 | .088 | .127 | .221 | .333 | |||||||||
| No (n = 197) | 79% (74%-85%) | 75% (69%-81%) | 65% (58%-72%) | 61% (53%-68%) | 54% (47%-62%) | 51% (43%-58%) | 20% (14%-25%) | 8% (4%-12%) | 10% (6%-14%) | ||||||
| Yes (n = 82) | 77% (67%-86%) | 72% (61%-82%) | 59% (47%-70%) | 55% (44%-67%) | 47% (36%-59%) | 45% (34%-57%) | 27% (17%-37%) | 12% (5%-20%) | 6% (0%-11%) | ||||||
| Pre-HSCT malignancy | .657 | .342 | .740 | .702 | .453 | .483 | |||||||||
| No (n = 285) | 76% (72%-81%) | 72% (66%-77%) | 61% (55%-67%) | 57% (50%-63%) | 50% (44%-57%) | 47% (41%-53%) | 23% (18%-27%) | 9% (6%-13%) | 9% (5%-12%) | ||||||
| Yes (n = 21) | 81% (64%-98%) | 76% (57%-94%) | 68% (48%-89%) | 68% (48%-89%) | 53% (30%-75%) | 53% (30%-75%) | 19% (2%-36%) | 14% (0%-29%) | 15% (0%-31%) | ||||||
| Pre-HSCT morbidity score|| | <.001 | .031 | .010 | .755 | .042 | .853 | |||||||||
| 0 to 1 (n = 159) | 87% (82%-92%) | 83% (76%-89%) | 69% (61%-77%) | 63% (54%-72%) | 57% (49%-65%) | 52% (44%-61%) | 21% (15%-28%) | 6% (3%-10%) | 9% (4%-14%) | ||||||
| ≥2 (n = 147) | 66% (58%-73%) | 61% (53%-69%) | 54% (46%-62%) | 52% (44%-61%) | 44% (36%-52%) | 42% (34%-51%) | 24% (17%-31%) | 13% (8%-19%) | 9% (4%-14%) | ||||||
| Conditioning | <.001 | <.001 | <.001 | .123 | .001 | .646 | |||||||||
| Busulfan-based (n = 112) | 79% (71%-86%) | 78% (70%-85%) | 68% (58%-77%) | 68% (58%-77%) | 54% (44%-64%) | 51% (41%-61%) | 25% (17%-33%) | 14% (7%-20%) | 7% (2%-11%) | ||||||
| Treosulfan-based (n = 115) | 87% (81%-93%) | 83% (76%-90%) | 68% (58%-77%) | 63% (53%-73%) | 57% (48%-67%) | 55% (45%-65%) | 16% (9%-23%) | 2% (0%-4%) | 11% (5%-17%) | ||||||
| Other/none (n = 79) | 60% (49%-71%) | 49% (37%-61%) | 45% (34%-56%) | 38% (27%-50%) | 37% (26%-48%) | 32% (21%-43%) | 28% (18%-38%) | 16% (8%-24%) | 10% (3%-17%) | ||||||
Boldface indicates significant P values.
EFS is defined as survival without graft failure, secondary procedures (DLI or CD34 boost), and a second transplant.
GEFS is defined as survival without grade III-IV aGvHD, cGvHD, graft failure, secondary procedures (DLI or CD34 boost), and a second transplant.
Calculated via the log-rank test.
Calculated via Gray test.
The pre-HSCT morbidity score is calculated using the following variables: pretransplant infections, pretransplant organ damage, pretransplant malignancies, pretransplant lung disease, pretransplant gut disease, pretransplant renal problems, pretransplant neurological complications. A positive indication for any these variables contributes 1 point to a patient’s pre-HCT morbidity score.