Characteristics of aplastic anemia patients included in EPAG-IST group and historic IST group
| EPAG-IST group . | Historic IST group (n = 102) . | P* . | ||||
|---|---|---|---|---|---|---|
| Cohort 1 (n = 30) . | Cohort 2 (n = 31) . | Cohort 3 + extension (n = 117) . | All patients (n = 178) . | |||
| Age distribution (y), n (%) | ||||||
| <18 | 5 (17) | 6 (19) | 33 (28) | 44 (25) | 23 (23) | .68 |
| 18-39 | 11 (37) | 13 (42) | 40 (34) | 64 (36) | 41 (40) | .49 |
| 40-60 | 8 (27) | 9 (29) | 22 (19) | 39 (22) | 22 (22) | .95 |
| >60 | 6 (20) | 3 (10) | 22 (19) | 31 (17) | 16 (16) | .71 |
| Disease severity, n (%) | ||||||
| VSAA | 13 (43) | 10 (32) | 53 (45) | 76 (43) | 35 (34) | .16 |
| SAA | 17 (57) | 21 (68) | 64 (55) | 102 (57) | 67 (66) | |
| Treatment, n (%) | ||||||
| 3 mo EPAG | — | 31 (100) | — | 31 (18) | — | |
| 6 mo EPAG | 30 (100) | — | 117 (100) | 147 (82) | — | |
| No CSA maintenance | 30 (100) | 14 (45) | — | 44 (25) | — | |
| CSA maintenance | — | 17 (55) | 117 (100) | 134 (75) | 102 (100) | <.001 |
| 6 mo response, n (%) | ||||||
| Overall response | 24 (80) | 27 (87) | 94 (80) | 145 (81) | 68 (67) | .008 |
| Complete response | 10 (33) | 8 (26) | 51 (44) | 69 (39) | 17 (17) | <.001 |
| Partial response | 14 (47) | 19 (61) | 43 (37) | 76 (43) | 51 (50) | .24 |
| No response | 3 (10) | 2 (6) | 11 (9) | 16 (9) | 30 (29) | <.001 |
| Off study | 3 (10) | 2 (6) | 12 (10) | 17 (10) | 4 (4) | .06 |
| Baseline GPI; deficient neutrophils, n (%) | ||||||
| <1% | 22 (73) | 19 (61) | 77 (66) | 118 (66) | 59 (58) | .17 |
| ≥1% | 8 (27) | 12 (39) | 40 (34) | 60 (34) | 43 (42) | |
| Baseline telomere length, n (%) | ||||||
| <10% | 8 (27) | 7 (23) | 13 (11) | 28 (16) | N/A | N/A |
| ≥10% | 22 (73) | 24 (77) | 100 (85) | 146 (82) | N/A | |
| Median time to follow up,† mo (range) | 73.5 (2.8-99.2) | 62.9 (5.6-74.1) | 36.5 (3.2-65.6) | 48.7 (2.8-99.2) | 87.8 (2.9–190.8) | <.001 |
| EPAG-IST group . | Historic IST group (n = 102) . | P* . | ||||
|---|---|---|---|---|---|---|
| Cohort 1 (n = 30) . | Cohort 2 (n = 31) . | Cohort 3 + extension (n = 117) . | All patients (n = 178) . | |||
| Age distribution (y), n (%) | ||||||
| <18 | 5 (17) | 6 (19) | 33 (28) | 44 (25) | 23 (23) | .68 |
| 18-39 | 11 (37) | 13 (42) | 40 (34) | 64 (36) | 41 (40) | .49 |
| 40-60 | 8 (27) | 9 (29) | 22 (19) | 39 (22) | 22 (22) | .95 |
| >60 | 6 (20) | 3 (10) | 22 (19) | 31 (17) | 16 (16) | .71 |
| Disease severity, n (%) | ||||||
| VSAA | 13 (43) | 10 (32) | 53 (45) | 76 (43) | 35 (34) | .16 |
| SAA | 17 (57) | 21 (68) | 64 (55) | 102 (57) | 67 (66) | |
| Treatment, n (%) | ||||||
| 3 mo EPAG | — | 31 (100) | — | 31 (18) | — | |
| 6 mo EPAG | 30 (100) | — | 117 (100) | 147 (82) | — | |
| No CSA maintenance | 30 (100) | 14 (45) | — | 44 (25) | — | |
| CSA maintenance | — | 17 (55) | 117 (100) | 134 (75) | 102 (100) | <.001 |
| 6 mo response, n (%) | ||||||
| Overall response | 24 (80) | 27 (87) | 94 (80) | 145 (81) | 68 (67) | .008 |
| Complete response | 10 (33) | 8 (26) | 51 (44) | 69 (39) | 17 (17) | <.001 |
| Partial response | 14 (47) | 19 (61) | 43 (37) | 76 (43) | 51 (50) | .24 |
| No response | 3 (10) | 2 (6) | 11 (9) | 16 (9) | 30 (29) | <.001 |
| Off study | 3 (10) | 2 (6) | 12 (10) | 17 (10) | 4 (4) | .06 |
| Baseline GPI; deficient neutrophils, n (%) | ||||||
| <1% | 22 (73) | 19 (61) | 77 (66) | 118 (66) | 59 (58) | .17 |
| ≥1% | 8 (27) | 12 (39) | 40 (34) | 60 (34) | 43 (42) | |
| Baseline telomere length, n (%) | ||||||
| <10% | 8 (27) | 7 (23) | 13 (11) | 28 (16) | N/A | N/A |
| ≥10% | 22 (73) | 24 (77) | 100 (85) | 146 (82) | N/A | |
| Median time to follow up,† mo (range) | 73.5 (2.8-99.2) | 62.9 (5.6-74.1) | 36.5 (3.2-65.6) | 48.7 (2.8-99.2) | 87.8 (2.9–190.8) | <.001 |