Table 1 Patient characteristics.
Characteristic | Treosulfan (n = 114) | Busulfan (n = 92) |
|---|---|---|
Age at SCT (years, median (range)) | 5.4 (0.2–18.2) | 8.5 (0.4–17.8) |
Sex (M/F) (%) | 62/38 | 58/42 |
Diagnosis for HSCT | ||
Beta-thalassemia (%) | 35 (30.7) | 6 (6.5) |
Sickle cell disease (%) | 20 (17.5) | 0 (0) |
Inborn errors of immunity (%) | 32 (28.1) | 10 (10.9) |
Hematological malignancy (%) | 18 (15.8) | 63 (68.5) |
Bone marrow failure (%) | 9 (7.9) | 13 (14.1) |
Donor | ||
MSD (%) | 36 (31.6) | 18 (19.6) |
MUD (≥ 9/10) (%) | 62 (54.4) | 69 (75.0) |
MMFD (haplo) (%) | 16 (14.0) | 5 (5.4) |
Stem cell source | ||
BM (%) | 85 (74.6) | 63 (68.5) |
PBSC (%) | 14 (12.3) | 14 (15.2) |
CB (%) | 15 (13.2) | 15 (16.3) |
Conditioning | ||
Treo-Flu-Thiotepa (%) | 76 (66.7) | – |
Treo-Flu (%) | 36 (31.6) | – |
Treo-Other (%) | 2 (1.7) | – |
Bu-Flu-Clo (%) | – | 54 (58.7) |
Bu-Flu (%) | – | 28 (30.4) |
Bu-Flu-Thiotepa (%) | 7 (7.6) | |
Bu-Cy-Mel (%) | – | 3 (3.3) |
Serotherapy | ||
ATG (%) | 77 (67.5) | 71 (77.2) |
Alemtuzumab (%) | 27 (23.7) | 7 (7.6) |
No (%) | 10 (8.8) | 14 (15.2) |
GvHD prophylaxis | ||
CsA + MTX(%) | 60 (52.6) | 57 (62.0) |
PTCy + MMF + CsA (%) | 16 (14.0) | 0 (0) |
CsA + Pred (%) | 9 (7.9) | 11 (12.0) |
CsA (%) | 9 (7.9) | 3 (3.3) |
Other (%) | 13 (11.4) | 16 (17.4) |
None (%) | 7 (6.1) | 5 (5.4) |