Patients enrolled in this study
| Subgroup . | No. of patients (families) . | Total (families) . | Age, y (range) . | Female/male ratio . | |
|---|---|---|---|---|---|
| WUMC . | Referred* . | ||||
| DC | 1 (1) | 21 (11) | 22 (12) | 28.3 (1.5-52.1) | 0.47 |
| DBA | 13 (9) | 28 (24) | 41 (33) | 11.8 (0.2-61.5) | 0.67 |
| SDS | 5 (5) | 0 (0) | 5 (5) | 20.1 (2.3-41.4) | 0.50 |
| MDS | 25 (24) | 6 (5) | 31 (29) | 46.8 (0.5-86.6) | 0.88 |
| PNH | 29 (29) | 16 (16) | 45 (45) | 36.6 (8.0-70.8) | 3.09 |
| AA | 67 (66) | 28 (25) | 95 (91) | 17.9 (0.05-75.8) | 0.69 |
| Other IBMF | 7 (6) | 1 (1) | 8 (7) | 9.3 (0.08-28.9) | 1.67 |
| All | 147 (140) | 100 (82) | 247 (221) | 23.5 (0.05-86.6) | 0.89 |
| Subgroup . | No. of patients (families) . | Total (families) . | Age, y (range) . | Female/male ratio . | |
|---|---|---|---|---|---|
| WUMC . | Referred* . | ||||
| DC | 1 (1) | 21 (11) | 22 (12) | 28.3 (1.5-52.1) | 0.47 |
| DBA | 13 (9) | 28 (24) | 41 (33) | 11.8 (0.2-61.5) | 0.67 |
| SDS | 5 (5) | 0 (0) | 5 (5) | 20.1 (2.3-41.4) | 0.50 |
| MDS | 25 (24) | 6 (5) | 31 (29) | 46.8 (0.5-86.6) | 0.88 |
| PNH | 29 (29) | 16 (16) | 45 (45) | 36.6 (8.0-70.8) | 3.09 |
| AA | 67 (66) | 28 (25) | 95 (91) | 17.9 (0.05-75.8) | 0.69 |
| Other IBMF | 7 (6) | 1 (1) | 8 (7) | 9.3 (0.08-28.9) | 1.67 |
| All | 147 (140) | 100 (82) | 247 (221) | 23.5 (0.05-86.6) | 0.89 |
WUMC indicates Washington University Medical Center; DC, dyskeratosis congenita; DBA, Diamond Blackfan syndrome; SDS, Shwachman-Diamond syndrome; MDS, myelodysplastic syndrome; PNH, paroxysmal nocturnal hemoglobinuria; AA, aplastic anemia; and IBMF, inherited bone marrow failure.
Patients enrolled through institutions outside Washington University.