Patient, donor, and transplant characteristics of patients given BM or CBT for hemoglobinopathies
| . | BMT n = 389 . | CBT n = 96 . | P value . |
|---|---|---|---|
| Median age at transplantation, y (range) | 8.1 (0.7-24) | 5.9 (2-20) | .02 |
| Patient gender, male/female | 200/189 | 48/48 | .82 |
| Median body weight, kg (range) | 23 (7-71) | 19 (10-60) | .01 |
| Not available | 3 | 0 | |
| Donor gender, male/female | 196/193 | 48/48 | .87 |
| Median donor age, y (range) | 9.0 (0.2-30) | — | <.01 |
| Diagnosis | |||
| TM | 259 (67%) | 66 (69%) | .55 |
| SCD | 130 (33%) | 30 (31%) | |
| Pesaro class for TM patients | |||
| Class 1 | 86 (33%) | 40 (61%) | <.01 |
| Class 2 | 122 (31%) | 23 (35%) | |
| Class 3 | 51 (13%) | 2 (4%) | |
| Previous CNS involvement at time of transplantation in patients with SCD | |||
| Yes | 57 (56%) | 16 (54%) | .83 |
| No | 73 (44%) | 14 (46%) | |
| Patient HCMV serology | |||
| Positive | 142 (37%) | 30 (35%) | .70 |
| Negative | 239 (63%) | 61 (65%) | |
| Not available | 8 | 5 | |
| Median number of nucleated cells infused ×108/kg of recipient body weight (range) | 4.1 (0.1-46) | 0.39 (1.5-14) | <.01 |
| Not available | 46 | 2 | |
| ABO compatibility | |||
| Compatible | 248 (66%) | 63 (70%) | .44 |
| Minor incompatibility | 48 (13%) | 10 (12%) | |
| Major incompatibility | 77 (21%) | 22 (28%) | |
| Not available | 17 | 1 | |
| Median year of transplantation (range) | 1999 (94-05) | 2001 (94-05) | <.01 |
| Conditioning regimen | |||
| Bu/Cy | 345 (89%) | 53 (56%) | <.01 |
| Bu/Cy/Flu | 16 (4%) | 5 (5%) | |
| Bu/Flu/TT | 27 (7%) | 21 (22%) | |
| Bu/Flu/ | 0 | 4 (4%) | |
| Bu/Cy/TT | 0 | 10 (11%) | |
| Not available | 1 | ||
| Use of ATG/ALG | |||
| Yes | 259 (67%) | 49 (54%) | <.01 |
| No | 130 (33%) | 43 (46%) | |
| Not available | 5 | ||
| GVHD prophylaxis | |||
| Scheme including MTX | 296 (76%) | 21 (30%) | <.01 |
| Scheme without MTX | 93 (24%) | 73 (70%) | |
| Not available | 2 | ||
| . | BMT n = 389 . | CBT n = 96 . | P value . |
|---|---|---|---|
| Median age at transplantation, y (range) | 8.1 (0.7-24) | 5.9 (2-20) | .02 |
| Patient gender, male/female | 200/189 | 48/48 | .82 |
| Median body weight, kg (range) | 23 (7-71) | 19 (10-60) | .01 |
| Not available | 3 | 0 | |
| Donor gender, male/female | 196/193 | 48/48 | .87 |
| Median donor age, y (range) | 9.0 (0.2-30) | — | <.01 |
| Diagnosis | |||
| TM | 259 (67%) | 66 (69%) | .55 |
| SCD | 130 (33%) | 30 (31%) | |
| Pesaro class for TM patients | |||
| Class 1 | 86 (33%) | 40 (61%) | <.01 |
| Class 2 | 122 (31%) | 23 (35%) | |
| Class 3 | 51 (13%) | 2 (4%) | |
| Previous CNS involvement at time of transplantation in patients with SCD | |||
| Yes | 57 (56%) | 16 (54%) | .83 |
| No | 73 (44%) | 14 (46%) | |
| Patient HCMV serology | |||
| Positive | 142 (37%) | 30 (35%) | .70 |
| Negative | 239 (63%) | 61 (65%) | |
| Not available | 8 | 5 | |
| Median number of nucleated cells infused ×108/kg of recipient body weight (range) | 4.1 (0.1-46) | 0.39 (1.5-14) | <.01 |
| Not available | 46 | 2 | |
| ABO compatibility | |||
| Compatible | 248 (66%) | 63 (70%) | .44 |
| Minor incompatibility | 48 (13%) | 10 (12%) | |
| Major incompatibility | 77 (21%) | 22 (28%) | |
| Not available | 17 | 1 | |
| Median year of transplantation (range) | 1999 (94-05) | 2001 (94-05) | <.01 |
| Conditioning regimen | |||
| Bu/Cy | 345 (89%) | 53 (56%) | <.01 |
| Bu/Cy/Flu | 16 (4%) | 5 (5%) | |
| Bu/Flu/TT | 27 (7%) | 21 (22%) | |
| Bu/Flu/ | 0 | 4 (4%) | |
| Bu/Cy/TT | 0 | 10 (11%) | |
| Not available | 1 | ||
| Use of ATG/ALG | |||
| Yes | 259 (67%) | 49 (54%) | <.01 |
| No | 130 (33%) | 43 (46%) | |
| Not available | 5 | ||
| GVHD prophylaxis | |||
| Scheme including MTX | 296 (76%) | 21 (30%) | <.01 |
| Scheme without MTX | 93 (24%) | 73 (70%) | |
| Not available | 2 | ||
Bu, busulfan; CNS, central nervous system; HCMV, human cytomegalovirus; Treo, treosulfan.