Patient and transplant characteristics
| . | Acquired . | Fanconi . |
|---|---|---|
| Number | 137 | 15 |
| Gender F:M, n | 63:74 | 9:6 |
| Etiology, n | ||
| Idiopathic | 122 | – |
| Hepatitis associated | 10 | – |
| PNH | 2 | – |
| Insecticide | 3 | – |
| Age at diagnosis, median (range) | 10.7 (0.1-17.8) | 6.3 (3.0-13.3) |
| Time from diagnosis to transplantation, median (range) | 0.1 (0.1-9.1) | 2.0 (0.1-6.6) |
| Age at transplantation, median (range) | 11.1 (0.8-17.9) | 9.0 (5.1-15.9) |
| Length of follow-up, median (range) | 21.8 (1-38.1) | 20 (2-30) |
| First transplantation preparative regimen | ||
| Single-fraction TBI, n | 2 | – |
| Fractionated TBI | ||
| 12.0 Gy | 10 | – |
| 200-600 cGy | 12 | 4 |
| Cyclophosphamide ± ATG | 106 | 11 |
| Procarbazine ATG cyclophosphamide | 5 | – |
| None | 2 | – |
| Second/third preparative regimens, n | 15/4 | – |
| Donor, | ||
| Related | 118 | 12 |
| Unrelated | 16 | 3 |
| Syngenic | 3 | – |
| HLA-matched donor, yes:no | 128:19 | 15:0 |
| Source of stem cells | ||
| BM | 135 | 12 |
| Peripheral blood | – | 3 |
| Cord blood | 2 | – |
| Acute GVHD, grades 2-3, n (%) | 41 (30%) | 9 (60%) |
| Chronic GVHD, Clinical extensive, n | 36 | 5 |
| . | Acquired . | Fanconi . |
|---|---|---|
| Number | 137 | 15 |
| Gender F:M, n | 63:74 | 9:6 |
| Etiology, n | ||
| Idiopathic | 122 | – |
| Hepatitis associated | 10 | – |
| PNH | 2 | – |
| Insecticide | 3 | – |
| Age at diagnosis, median (range) | 10.7 (0.1-17.8) | 6.3 (3.0-13.3) |
| Time from diagnosis to transplantation, median (range) | 0.1 (0.1-9.1) | 2.0 (0.1-6.6) |
| Age at transplantation, median (range) | 11.1 (0.8-17.9) | 9.0 (5.1-15.9) |
| Length of follow-up, median (range) | 21.8 (1-38.1) | 20 (2-30) |
| First transplantation preparative regimen | ||
| Single-fraction TBI, n | 2 | – |
| Fractionated TBI | ||
| 12.0 Gy | 10 | – |
| 200-600 cGy | 12 | 4 |
| Cyclophosphamide ± ATG | 106 | 11 |
| Procarbazine ATG cyclophosphamide | 5 | – |
| None | 2 | – |
| Second/third preparative regimens, n | 15/4 | – |
| Donor, | ||
| Related | 118 | 12 |
| Unrelated | 16 | 3 |
| Syngenic | 3 | – |
| HLA-matched donor, yes:no | 128:19 | 15:0 |
| Source of stem cells | ||
| BM | 135 | 12 |
| Peripheral blood | – | 3 |
| Cord blood | 2 | – |
| Acute GVHD, grades 2-3, n (%) | 41 (30%) | 9 (60%) |
| Chronic GVHD, Clinical extensive, n | 36 | 5 |
ATG indicates antithymocyte globulin; PNH, paroxysmal nocturnal hemoglobinuria; and TBI, total body irradiation.