Table 1.
Summary of patient demographic, treatment and disease characteristics. Values are represented as n (%)
| n = 126 | Busulfan (72) | Treosulfan (54) | |
|---|---|---|---|
| Sex | Male | 42 (58.33) | 33 (61.11) |
| Female | 30 (41.67) | 21 (38.89) | |
| Age (mo) a | 51.25 (7.39–227.14) | 33.28 (4.5–209.97) | |
| Weight (kg)a | 14 (5.17–47) | 10.1 (3.76–35.8) | |
| Conditioning | Alone | 5 (6.94) | |
| + Cyclo | 5 (6.94) | ||
| + Cyclo/Melph | 10 (13.89) | ||
| + Cyclo/Melph/Alem | 5 (6.94) | ||
| + Cyclo/Alem or ATG | 5 (6.94) | ||
| + Flu | 9 (12.50) | 9 (16.67) | |
| + Flu/Thio | 7 (12.96) | ||
| + Flu/Thio/ATG | 8 (14.81) | ||
| + Flu/Alem or ATG | 27 (37.50) | 19 (35.19) | |
| + Melph | 6 (8.33) | ||
| + Flu/Alem/Thio | 11 (20.37) | ||
| Diagnosis | Immunodeficiency | 30 (41.67) | 26 (48.15) |
| Cancer | 30 (41.67) | 18 (33.33) | |
| Others | 12 (16.67) | 10 (18.52) | |
| Transplant type | Bone marrow | 62 (86.11) | 48 (88.89) |
| Gene therapy | 7 (9.72) | 4 (7.41) | |
| Peripheral blood | 3 (4.16) | ‐ | |
| Umbilical cord | ‐ | 2 (3.70) | |
| Donor type | Autologous | 12 (16.67) | 1 (1.85) |
| Haploidentical | ‐ | 4 (7.41) | |
| Mismatched family donor | 2 (2.78) | 4 (7.41) | |
| Mismatched unrelated donor | 19 (26.39) | 20 (37.04) | |
| Matched sibling donor | 21 (29.17) | 3 (5.56) | |
| Matched unrelated donor | 18 (25.00) | 17 (31.48) | |
| Unknown | ‐ | 5 (9.26) | |
| Cytomegalovirus | No | 38 (52.78) | 29 (53.70) |
| Yes | 27 (37.50) | 19 (35.19) | |
| Unknown | 7 (9.72) | 6 (11.11) | |
| Epstein–Barr virus | No | 33 (45.83) | 27 (50.00) |
| Yes | 32 (44.44) | 22 (40.74) | |
| Unknown | 7 (9.72) | 5 (9.26) | |
| Granulocyte‐Colony stimulating factors | No | 25 (34.72) | 22 (40.74) |
| Yes | 46 (63.89) | 31 (57.41) | |
| Unknown | 1 (1.39) | 1 (1.85) | |
| Deceased | 10 (13.89) | 9 (16.67) | |
a
Values are represented as mean (range). Cyclo, Cyclophosphamide; Flu, Fludarabine; Melph, Melphalan; Alem, Alemtuzumab; Thio, Thiotepa; ATG, Anti‐thymocyte globulin.