Clinical characteristics of the cohort
| Variable . | Modalities . | Total (N = 275) . | No sMN (N = 231) . | sMN (N = 44) . |
|---|---|---|---|---|
| Age at diagnosis | Median ± SD (range), y | 54.0 ± 18.4 (18.0-89.4) | 51.2 ± 19.0 (18.0-89.4) | 61.0 ± 13.2 (25.0-78.3) |
| Sex, n (%) | Male | 136 (49.5) | 112 (48.5) | 24 (54.5) |
| Female | 139 (50.5) | 119 (51.5) | 20 (45.5) | |
| Disease severity at diagnosis, n (%) | SAA or VSAA | 222 (80.7) | 182 (78.8) | 40 (90.9) |
| Nonsevere AA | 53 (19.3) | 49 (21.2) | 4 (9.1) | |
| Treatment response, n (%) | No response | 88 (32.0) | 71 (30.7) | 17 (38.6) |
| Partial response | 96 (34.9) | 78 (33.8) | 18 (40.9) | |
| Complete response | 88 (32.0) | 79 (34.2) | 9 (20.5) | |
| Unknown | 3 (1.1) | 3 (1.3) | 0 (0.0) | |
| PNH clone at diagnosis, n (%) | Clone present | 111 (40.4) | 98 (42.4) | 13 (29.5) |
| Clone absent | 109 (39.6) | 87 (37.7) | 22 (50.0) | |
| Unknown | 55 (20.0) | 46 (19.9) | 9 (20.5) | |
| Event-free follow-up time | Median ± SD, y | 4.2 ± 6.6 | 4.5 ± 7.0 | 3.9 ± 3.2 |
| Somatic mutation testing at AA diagnosis, n (%) | Preleukemic mutations at baseline | 36 (13.1) | 29 (12.5) | 7 (15.9) |
| No detected preleukemic mutations at baseline | 159 (90.8) | 140 (60.6) | 19 (43.2) | |
| Baseline mutation data missing | 80 (29.1) | 62 (26.8) | 18 (40.9) | |
| Cohort, n (%) | Cleveland Clinic | 215 (78.2) | 181 (78.4) | 34 (77.2) |
| University of Texas Southwestern | 41 (14.9) | 36 (15.6) | 5 (11.4) | |
| University of Pennsylvania | 19 (6.9) | 14 (6.0) | 5 (11.4) |
| Variable . | Modalities . | Total (N = 275) . | No sMN (N = 231) . | sMN (N = 44) . |
|---|---|---|---|---|
| Age at diagnosis | Median ± SD (range), y | 54.0 ± 18.4 (18.0-89.4) | 51.2 ± 19.0 (18.0-89.4) | 61.0 ± 13.2 (25.0-78.3) |
| Sex, n (%) | Male | 136 (49.5) | 112 (48.5) | 24 (54.5) |
| Female | 139 (50.5) | 119 (51.5) | 20 (45.5) | |
| Disease severity at diagnosis, n (%) | SAA or VSAA | 222 (80.7) | 182 (78.8) | 40 (90.9) |
| Nonsevere AA | 53 (19.3) | 49 (21.2) | 4 (9.1) | |
| Treatment response, n (%) | No response | 88 (32.0) | 71 (30.7) | 17 (38.6) |
| Partial response | 96 (34.9) | 78 (33.8) | 18 (40.9) | |
| Complete response | 88 (32.0) | 79 (34.2) | 9 (20.5) | |
| Unknown | 3 (1.1) | 3 (1.3) | 0 (0.0) | |
| PNH clone at diagnosis, n (%) | Clone present | 111 (40.4) | 98 (42.4) | 13 (29.5) |
| Clone absent | 109 (39.6) | 87 (37.7) | 22 (50.0) | |
| Unknown | 55 (20.0) | 46 (19.9) | 9 (20.5) | |
| Event-free follow-up time | Median ± SD, y | 4.2 ± 6.6 | 4.5 ± 7.0 | 3.9 ± 3.2 |
| Somatic mutation testing at AA diagnosis, n (%) | Preleukemic mutations at baseline | 36 (13.1) | 29 (12.5) | 7 (15.9) |
| No detected preleukemic mutations at baseline | 159 (90.8) | 140 (60.6) | 19 (43.2) | |
| Baseline mutation data missing | 80 (29.1) | 62 (26.8) | 18 (40.9) | |
| Cohort, n (%) | Cleveland Clinic | 215 (78.2) | 181 (78.4) | 34 (77.2) |
| University of Texas Southwestern | 41 (14.9) | 36 (15.6) | 5 (11.4) | |
| University of Pennsylvania | 19 (6.9) | 14 (6.0) | 5 (11.4) |
For patients who developed sMNs, event-free follow-up time is defined as the duration (in years) from the diagnosis of AA to the onset of sMN. For patients who did not develop sMNs, event-free follow-up time is measured from the diagnosis of AA to the occurrence of death, transplantation, or the last recorded follow-up, whichever came first.