Patient, transplant, and graft characteristics
| Variables . | RD group . | MSD group . | P . |
|---|---|---|---|
| Number of patients | 16 | 66 | — |
| Median patient age, y | 9.6 (1.4-24) | 10 (1.8-27) | .68 |
| Patient sex | |||
| Male, n | 10 | 37 | .78 |
| Female, n | 6 | 29 | — |
| Risk class | |||
| Class 1, n | 1 | 0 | .20 |
| Class 2, n | 5 | 31 | .27 |
| Class 3, n | 10 | 35 | .58 |
| Median donor age, y | 37.5 (2-53) | 13 (1-34) | .11 |
| Median AST, IU/L | 38 (13-120) | 34 (19-216) | .28 |
| Median ALT, IU/L | 48 (13-122) | 38 (11-224) | .35 |
| Median bilirubin, mg/dL | 0.95 (0.4-1.5) | 1.2 (0.3-5.0) | .12 |
| Median serum ferritin, ng/mL | 1867 (782-7944) | 2219 (682-10 222) | .48 |
| Median liver iron concentration, g/dry weight | 14.7 (1.7-28.2) | 16 (2-44) | .50 |
| Median packed RBC units received pretransplantation | 96 (13-350) | 105 (11-400) | .86 |
| Liver size >2 cm, n | 11 | 41 | .77 |
| Splenectomy, n | 4 | 20 | .77 |
| Median liver fibrosis score (Ishak et al24 ) | 2 (0-6) | 2 (0-5) | .11 |
| Hepatitis C (HCV-RNA positive), n | 2 | 6 | .65 |
| Donor–recipient HLA matching | |||
| Phenotypically matched, n | 11 | ||
| One-antigen mismatched, n | 5 | ||
| Matched siblings, n | 66 | ||
| Donor–recipient CMV status | |||
| Both positive, n | 16 | 55 | .11 |
| Any positive, n | 7 | .33 | |
| Both negative | 4 | .58 | |
| Donor–recipient sex match | |||
| Matched, n | 9 | 30 | |
| Mismatched, n | 7 | 36 | .57 |
| ABO blood group compatibility | |||
| Identical, n | 7 | 40 | .27 |
| Major and/or minor incompatibility, n | 9 | 26 | |
| Preparation for transplant | |||
| Preconditioning cytoreduction/immunosuppression | 16 | BU14 /i.v.BU/CY200 | |
| d −45 to d −12 | |||
| HU 30 mg/kg/d | ±TT10 (n = 31) | ||
| AZ 3 mg/kg/d | BU14 /i.v.BU/CY160 | ||
| d −16 to d −12 | |||
| Flu 30 mg/m2/d | Preceded by HU, AZ, Flu | ||
| Conditioning regimen | n = 26 | ||
| d −10 to d −7 | |||
| Oral BU14/weight-based i.v. Busilvex | BU14 /i.v.BU/CY90 ±TT10 | ||
| d −6 | |||
| TT 10 mg/kg/d | Preceded by HU, AZ, Flu | ||
| d −6 to d −3 | |||
| Thymoglobulin 2.5 mg/kg/d | n = 9 | ||
| d −5 to d −2 | |||
| CY 50 mg/kg/d | — | ||
| GVHD prophylaxis with CSA + methylprednisolone + CY or MTX (d 1) + MTX (d 3 and 6), n | 16 | 66 | |
| Median nucleated cells, × 108/kg | 4.2 (1.8-10) | 4.3 (1.3-8.7) | .75 |
| Median CD34+ cells, × 106/kg | 4 (1-13) | 6.3 (0.8-35) | .68 |
| Variables . | RD group . | MSD group . | P . |
|---|---|---|---|
| Number of patients | 16 | 66 | — |
| Median patient age, y | 9.6 (1.4-24) | 10 (1.8-27) | .68 |
| Patient sex | |||
| Male, n | 10 | 37 | .78 |
| Female, n | 6 | 29 | — |
| Risk class | |||
| Class 1, n | 1 | 0 | .20 |
| Class 2, n | 5 | 31 | .27 |
| Class 3, n | 10 | 35 | .58 |
| Median donor age, y | 37.5 (2-53) | 13 (1-34) | .11 |
| Median AST, IU/L | 38 (13-120) | 34 (19-216) | .28 |
| Median ALT, IU/L | 48 (13-122) | 38 (11-224) | .35 |
| Median bilirubin, mg/dL | 0.95 (0.4-1.5) | 1.2 (0.3-5.0) | .12 |
| Median serum ferritin, ng/mL | 1867 (782-7944) | 2219 (682-10 222) | .48 |
| Median liver iron concentration, g/dry weight | 14.7 (1.7-28.2) | 16 (2-44) | .50 |
| Median packed RBC units received pretransplantation | 96 (13-350) | 105 (11-400) | .86 |
| Liver size >2 cm, n | 11 | 41 | .77 |
| Splenectomy, n | 4 | 20 | .77 |
| Median liver fibrosis score (Ishak et al24 ) | 2 (0-6) | 2 (0-5) | .11 |
| Hepatitis C (HCV-RNA positive), n | 2 | 6 | .65 |
| Donor–recipient HLA matching | |||
| Phenotypically matched, n | 11 | ||
| One-antigen mismatched, n | 5 | ||
| Matched siblings, n | 66 | ||
| Donor–recipient CMV status | |||
| Both positive, n | 16 | 55 | .11 |
| Any positive, n | 7 | .33 | |
| Both negative | 4 | .58 | |
| Donor–recipient sex match | |||
| Matched, n | 9 | 30 | |
| Mismatched, n | 7 | 36 | .57 |
| ABO blood group compatibility | |||
| Identical, n | 7 | 40 | .27 |
| Major and/or minor incompatibility, n | 9 | 26 | |
| Preparation for transplant | |||
| Preconditioning cytoreduction/immunosuppression | 16 | BU14 /i.v.BU/CY200 | |
| d −45 to d −12 | |||
| HU 30 mg/kg/d | ±TT10 (n = 31) | ||
| AZ 3 mg/kg/d | BU14 /i.v.BU/CY160 | ||
| d −16 to d −12 | |||
| Flu 30 mg/m2/d | Preceded by HU, AZ, Flu | ||
| Conditioning regimen | n = 26 | ||
| d −10 to d −7 | |||
| Oral BU14/weight-based i.v. Busilvex | BU14 /i.v.BU/CY90 ±TT10 | ||
| d −6 | |||
| TT 10 mg/kg/d | Preceded by HU, AZ, Flu | ||
| d −6 to d −3 | |||
| Thymoglobulin 2.5 mg/kg/d | n = 9 | ||
| d −5 to d −2 | |||
| CY 50 mg/kg/d | — | ||
| GVHD prophylaxis with CSA + methylprednisolone + CY or MTX (d 1) + MTX (d 3 and 6), n | 16 | 66 | |
| Median nucleated cells, × 108/kg | 4.2 (1.8-10) | 4.3 (1.3-8.7) | .75 |
| Median CD34+ cells, × 106/kg | 4 (1-13) | 6.3 (0.8-35) | .68 |
ALT, alanine aminotransferase; AST, aspartate aminotransferase; CSA, cyclosporine; CY, cyclophosphamide; MTX, methotrexate; RBC, red blood cell; RD, HLA phenotypically identical or 1-antigen–mismatched RDs; TT, thiotepa.