Patient, disease, and graft characteristics
| Characteristics, n = 26 patients . | n (%) or median (IQR) . |
|---|---|
| Patients | |
| Age at transplantation, y | 16 (9-23) |
| Children, age <18 y | 15 (58) |
| Weight at transplantation, kg | 55.5 (28.5-69.3) |
| Males | 15 (58) |
| Recipient CMV+ | 16 (62) |
| Disease | |
| PNH clone | 8 (31) |
| Posthepatitis SAA | 1 (4) |
| Disease history and transfusions* | |
| 2 courses or more previous immunosuppressive treatment | 11 (42.3) |
| Time interval from diagnosis of SAA to transplantation, mo | 11.9 (8.7-17.8) |
| >20 red blood cell units infused | 18 (69) |
| >20 platelet unit transfusions | 22 (85) |
| Graft | |
| Single CB vs double-unit CB | 16 (62) |
| No. of HLA disparities for single CB† | |
| 6/6 match | 0 |
| 5/6 match | 8 (50) |
| 4/6 match | 7 (44) |
| 3/6 match | 1 (6) |
| No. of HLA disparities for double CB‡ | |
| 2 units 5/6 match | 3 (30) |
| 2 units 4/6 match | 4 (40) |
| 1 unit 5/6 and 1 unit 4/6 match | 2 (20) |
| 1 unit 6/6 and 1 unit 5/6 match | 1 (10) |
| No. of frozen nucleated cells, ×107/kg | |
| Single CB | 5.8 (4.9-7.2) |
| Double-unit CB | 5.7 (5.3-7.1) |
| No. of infused nucleated cells, ×107/kg | |
| Single CB | 4.3 (3.6-5.6) |
| Double-unit CB | 3 (2.8-3.8) |
| No. of frozen CD34+cells, ×105/kg | |
| Single CB | 2.0 (1.3-2.6) |
| Double-unit CB | 2.2 (1.4-2.9) |
| No. of infused CD34+cells, ×105/kg | |
| Single CB | 1.2 (1.1-1.8) |
| Double-unit CB | 1.7 (1.0-2.4) |
| Characteristics, n = 26 patients . | n (%) or median (IQR) . |
|---|---|
| Patients | |
| Age at transplantation, y | 16 (9-23) |
| Children, age <18 y | 15 (58) |
| Weight at transplantation, kg | 55.5 (28.5-69.3) |
| Males | 15 (58) |
| Recipient CMV+ | 16 (62) |
| Disease | |
| PNH clone | 8 (31) |
| Posthepatitis SAA | 1 (4) |
| Disease history and transfusions* | |
| 2 courses or more previous immunosuppressive treatment | 11 (42.3) |
| Time interval from diagnosis of SAA to transplantation, mo | 11.9 (8.7-17.8) |
| >20 red blood cell units infused | 18 (69) |
| >20 platelet unit transfusions | 22 (85) |
| Graft | |
| Single CB vs double-unit CB | 16 (62) |
| No. of HLA disparities for single CB† | |
| 6/6 match | 0 |
| 5/6 match | 8 (50) |
| 4/6 match | 7 (44) |
| 3/6 match | 1 (6) |
| No. of HLA disparities for double CB‡ | |
| 2 units 5/6 match | 3 (30) |
| 2 units 4/6 match | 4 (40) |
| 1 unit 5/6 and 1 unit 4/6 match | 2 (20) |
| 1 unit 6/6 and 1 unit 5/6 match | 1 (10) |
| No. of frozen nucleated cells, ×107/kg | |
| Single CB | 5.8 (4.9-7.2) |
| Double-unit CB | 5.7 (5.3-7.1) |
| No. of infused nucleated cells, ×107/kg | |
| Single CB | 4.3 (3.6-5.6) |
| Double-unit CB | 3 (2.8-3.8) |
| No. of frozen CD34+cells, ×105/kg | |
| Single CB | 2.0 (1.3-2.6) |
| Double-unit CB | 2.2 (1.4-2.9) |
| No. of infused CD34+cells, ×105/kg | |
| Single CB | 1.2 (1.1-1.8) |
| Double-unit CB | 1.7 (1.0-2.4) |
Sixteen patients received 1 CB unit and 10 received 2 CB units. Median number of nucleated cells infused was 3.7 × 107/kg (IQR, 3-4.9) and CD34+ cells infused was 1.4 × 105/kg (IQR, 1.0-1.9).
CMV indicates cytomegalovirus; PNH, paroxysmal nocturnal hemoglobinuria.
Fifteen patients received transplants because of refractory disease after immunosuppressive treatment (ATG plus CSA) at a median time of 9.2 months after diagnosis, although 11 patients received transplants because of relapse following 2 courses of immunosuppressive treatment (ATG plus CSA) at a median time of 9 months after relapse.
One unit, antigen-level HLA-A and -B and allele-level HLA-RDB1 typing.
Two units, antigen-level HLA-A and -B and allele-level HLA-RDB1.