Response rates, infections, hospitalization days, relapses, late complications, and deaths
| . | All patients . | No G-CSF . | With G-CSF . | P . |
|---|---|---|---|---|
| Best response ever reached | ||||
| Complete response, n (%) | 21 (11) | 9 (9) | 12 (12) | .535 |
| Partial response, n (%) | 113 (59) | 54 (57) | 59 (61) | |
| No response, n (%) | 58 (30) | 32 (34) | 26 (27) | |
| Best response ever reached for very SAA | ||||
| Complete response, n (%) | 6 (8) | 2 (5) | 4 (13) | |
| Partial response, n (%) | 33 (47) | 19 (49) | 14 (45) | .513 |
| No response, n (%) | 31 (44) | 18 (46) | 13 (42) | |
| Best response ever reached for SAA | ||||
| Complete response, n (%) | 15 (12) | 7 (12) | 8 (12) | |
| Partial response, n (%) | 80 (66) | 35 (63) | 45 (68) | .764 |
| No response, n (%) | 27 (22) | 14 (25) | 13 (20) | |
| No. of infectious episodes per month at risk during the first 90 days | ||||
| All patients, n/N (%) | 137/462 (30) | 81/228 (36) | 56/234 (24) | .006 |
| Patients with SAA, n/N (%) | 67/302 (22) | 33/136 (24) | 34/16 (20) | .431 |
| Patients with very SAA, n/N (%) | 70/160 (44) | 48/92 (52) | 22/68 (32) | .014 |
| Days spent in the hospital during the first 30 days, n/N (%) | 3008/3551 (84) | 1612/1858 (87) | 1396/1693 (82) | .0003 |
| No. of patients with first relapse (%) | 38 (20) | 19 (20) | 19 (20) | .679 |
| No. of HSCT as second- or third-line therapy (%) | 19 (10) | 11 (11) | 9 (9) | .570 |
| Secondary malignant neoplasm, n (%) | 7 (3.6) | 4 (4) | 3 (3) | .488 |
| PNH | ||||
| At diagnosis, n (%) | 23 (12) | 14 15) | 9 (9) | .350 |
| Since first IS treatment, n (%) | 14 (7) | 8 (8) | 6 (6) | .590 |
| Number of deaths (%) | 44 (23) | 23 (24) | 21 (22) | .673 |
| Cumulative incidence of a complications at 6 years (95% CI) | ||||
| Relapse | 33 (24-46) | 33 (21-53) | 32 (21-51) | .792 |
| Malignant neoplasm | 4 (2-10) | 6 (2-16) | 3 (1-11) | .537 |
| PNH clone | 19 (14-27) | 22 (15-33) | 16 (9-27) | .067 |
| . | All patients . | No G-CSF . | With G-CSF . | P . |
|---|---|---|---|---|
| Best response ever reached | ||||
| Complete response, n (%) | 21 (11) | 9 (9) | 12 (12) | .535 |
| Partial response, n (%) | 113 (59) | 54 (57) | 59 (61) | |
| No response, n (%) | 58 (30) | 32 (34) | 26 (27) | |
| Best response ever reached for very SAA | ||||
| Complete response, n (%) | 6 (8) | 2 (5) | 4 (13) | |
| Partial response, n (%) | 33 (47) | 19 (49) | 14 (45) | .513 |
| No response, n (%) | 31 (44) | 18 (46) | 13 (42) | |
| Best response ever reached for SAA | ||||
| Complete response, n (%) | 15 (12) | 7 (12) | 8 (12) | |
| Partial response, n (%) | 80 (66) | 35 (63) | 45 (68) | .764 |
| No response, n (%) | 27 (22) | 14 (25) | 13 (20) | |
| No. of infectious episodes per month at risk during the first 90 days | ||||
| All patients, n/N (%) | 137/462 (30) | 81/228 (36) | 56/234 (24) | .006 |
| Patients with SAA, n/N (%) | 67/302 (22) | 33/136 (24) | 34/16 (20) | .431 |
| Patients with very SAA, n/N (%) | 70/160 (44) | 48/92 (52) | 22/68 (32) | .014 |
| Days spent in the hospital during the first 30 days, n/N (%) | 3008/3551 (84) | 1612/1858 (87) | 1396/1693 (82) | .0003 |
| No. of patients with first relapse (%) | 38 (20) | 19 (20) | 19 (20) | .679 |
| No. of HSCT as second- or third-line therapy (%) | 19 (10) | 11 (11) | 9 (9) | .570 |
| Secondary malignant neoplasm, n (%) | 7 (3.6) | 4 (4) | 3 (3) | .488 |
| PNH | ||||
| At diagnosis, n (%) | 23 (12) | 14 15) | 9 (9) | .350 |
| Since first IS treatment, n (%) | 14 (7) | 8 (8) | 6 (6) | .590 |
| Number of deaths (%) | 44 (23) | 23 (24) | 21 (22) | .673 |
| Cumulative incidence of a complications at 6 years (95% CI) | ||||
| Relapse | 33 (24-46) | 33 (21-53) | 32 (21-51) | .792 |
| Malignant neoplasm | 4 (2-10) | 6 (2-16) | 3 (1-11) | .537 |
| PNH clone | 19 (14-27) | 22 (15-33) | 16 (9-27) | .067 |
IS indicates immunosuppression.