Table 1.
Patient cohort demographic characteristics
| FA | Other diagnoses | ||
|---|---|---|---|
| Total, n | 23 | 29 | |
| Sex | |||
| Male | 11 (48%) | 20 (69%) | |
| Female | 12 (52%) | 9 (31%) | |
| Age, y | |||
| Range | 5-27 | 1-18 | |
| Median | 7 | 6 | |
| Preparative regimen, n | Bu/Cy/ATG (2), Bu/Cy/Flu/ATG (21) | Bu/Cy (1), Bu/Cy/ATG (5), Bu/Flu (2), Bu/Flu/ATG (1), Bu/Flu/Cam (2), Bu/Thio/Flu (2), Cam/Flu/Mel (10), Cy/ATG (2), Cy + TBI (3), Flu/Mel (1) | |
| Stem cell source | Bone marrow, 1 (4%); PBSCs, 22 (96%) | Bone marrow, 24 (83%); cord blood, 2 (7%); PBSCs, 3 (10%) | |
| Stem cell donor type | Related, 2 (9%); unrelated, 21 (91%) | Related, 12 (34%); unrelated, 17 (49%) | |
| GVHD prophylaxis | Calcineurin inhibitor, 5 (22%); T cell–depleted graft alone, 18 (78%) | Calcineurin inhibitor, 29 (100%) | |
| Additional information, (n) | Complementation groups represented (n): A (6), B (1), C (6), E (2), G (1), I (1), J (1), O (1), normal FISH/unknown (4) | Diagnoses represented (n): AA (5), ALL (2), AML (4), β-thalassemia (3), CGD (1), DBA (1), sickle cell disease (5), HLH (2), Hurler’s disease (1), pancytopenia (1), SCID (1), TCLL (1), WAS (2) | |
AA, aplastic anemia; ALL, acute lymphocytic leukemia; AML, acute myelogenous leukemia; ATG, anti-thymocyte globulin; Bu, busulfan; Cam, Campath (alemtuzumab); CGD, chronic granulomatous disease; Cy, cyclophosphamide; DBA, Diamond-Blackfan anemia; FISH, fluorescence in situ hybridization; Flu, fludarabine; GVHD, graft-versus-host disease; HLH, hemophagocytic lymphoproliferative histiocytosis; Mel, melphalan; SCID, severe combined immunodeficiency; TBI, total body irradiation; TCLL, T-cell leukemia; Thio, thiotepa; WAS, Wiskott-Aldrich syndrome.