Patient characteristics at diagnosis
| Category . | n (%)/median (IQR) . |
|---|---|
| All | 263 |
| Age, y | 44 (27-62) |
| Ethnicity | |
| White | 214 (84) |
| African American | 22 (8) |
| Asian | 9 (4) |
| Hispanic or Latino | 7 (3) |
| Other | 2 (2) |
| Unknown | 9 (3) |
| Sex | |
| Female | 132 (50.2) |
| Male | 131 (49.8) |
| Disease phenotype | |
| IAA +/– nonhemolytic PNH clone | 216 (82) |
| IAA + hemolytic PNH clone | 13 (5) |
| Primary hemolytic PNH | 34 (13) |
| PNH granulocytic clone size | 5.6 (1-37.2) |
| Severity IAA | |
| Severe | 166 (71) |
| Moderate | 66 (28) |
| Treatment characteristics | |
| First-line treatment (except allo-HCT)* | |
| ATG/CSA | 129 (53) |
| ATG/CSA/eltrombopag | 14 (6) |
| CSA | 22 (9) |
| CSA/eltrombopag | 8 (3) |
| CSA/other | 5 (2) |
| Androgens | 10 (4) |
| Anticomplement | 26 (11) |
| Eltrombopag | 3 (1) |
| None | 14 (6) |
| Other | 11 (5) |
| Allo-HCT (all lines) | 46 (17) |
| N° lines* | |
| 0 | 17 (8) |
| 1 | 17 (8) |
| 2 | 53 (25) |
| 3 | 26 (13) |
| 4 | 13 (6) |
| ≥5 | 3 (14) |
| Category . | n (%)/median (IQR) . |
|---|---|
| All | 263 |
| Age, y | 44 (27-62) |
| Ethnicity | |
| White | 214 (84) |
| African American | 22 (8) |
| Asian | 9 (4) |
| Hispanic or Latino | 7 (3) |
| Other | 2 (2) |
| Unknown | 9 (3) |
| Sex | |
| Female | 132 (50.2) |
| Male | 131 (49.8) |
| Disease phenotype | |
| IAA +/– nonhemolytic PNH clone | 216 (82) |
| IAA + hemolytic PNH clone | 13 (5) |
| Primary hemolytic PNH | 34 (13) |
| PNH granulocytic clone size | 5.6 (1-37.2) |
| Severity IAA | |
| Severe | 166 (71) |
| Moderate | 66 (28) |
| Treatment characteristics | |
| First-line treatment (except allo-HCT)* | |
| ATG/CSA | 129 (53) |
| ATG/CSA/eltrombopag | 14 (6) |
| CSA | 22 (9) |
| CSA/eltrombopag | 8 (3) |
| CSA/other | 5 (2) |
| Androgens | 10 (4) |
| Anticomplement | 26 (11) |
| Eltrombopag | 3 (1) |
| None | 14 (6) |
| Other | 11 (5) |
| Allo-HCT (all lines) | 46 (17) |
| N° lines* | |
| 0 | 17 (8) |
| 1 | 17 (8) |
| 2 | 53 (25) |
| 3 | 26 (13) |
| 4 | 13 (6) |
| ≥5 | 3 (14) |
Data reported for the Cleveland Clinic cohort only.
Allo-HTC, allogeneic hematopoietic stem cell transplant; ATG, antithymocyte globuline; IQR, interquartile range.
Missing data.