Patient, transplantation, and graft characteristics
| Variable . | Patients (n = 71) . |
|---|---|
| Patient sex, male/female | 42/29 |
| Median patient age, y (range) | 9 (1.6-27) |
| Mean patient age, y (± SD) | 10.2 (± 5.2) |
| Patients older than 18 y, n | 4 |
| Mean patient weight ± SD, kg (range) | 26.4 ± 11.15 (10-60) |
| Median donor age, y (range) | 11 (1.3-52) |
| Diagnosis | |
| Thalassemia | 68 |
| Sickle cell anemia | 3 |
| Risk class (1/2/3/not applicable) | 6/23/39/3 |
| Median AST, IU/L (range) | 33 (13-216) |
| Median ALT, IU/L (range) | 38 (8-247) |
| Median bilirubin, mg/dL (range) | 0.9 (0.3-2.8) |
| Median serum ferritin, ng/mL (range) | 2126 (279-9458) |
| Serum ferritin > 2000 ng/mL, n | 38 |
| Median liver iron concentration, mg/g dw (range) | 17 (0.6-47.8) |
| Liver iron concentration > 7 mg/g dw, n | 55 |
| Median packed RBC units received before transplantation, n (range) | 95 (2-500) |
| Liver size ≤ 3 cm, n | 55 |
| Liver size > 3 cm, n | 16 |
| Splenectomy, yes/no | 17/54 |
| Liver fibrosis score (Ishak et al23 ; staging 0-6) | 1 (0-6) |
| Incomplete cirrhosis | 4 |
| Cirrhosis | 2 |
| Hepatitis C (HCV-RNA–positive) | 7 |
| Hepatitis B (HBs antigen–positive) | 1 |
| HLA-identical siblings | 62 |
| HLA-phenotypically identical parents or relatives | 9 |
| Donor/patient CMV serology | |
| Both positive | 60 |
| Any positive | 9 |
| Both negative | 2 |
| Conditioning regimen | |
| IV Bu/CY200 | 11 |
| IV Bu/TT10/CY200 | 14 |
| IV Bu/CY160 preceded by HU AZA FL20 | 15 |
| IV Bu/TT10/CY160 preceded by HU AZA FL30 | 15 |
| IV Bu/TT10/CY90 preceded by HU AZA FL30 | 3 |
| IV Bu/TT10/CY200/ATG10 preceded by HU AZA FL30 | 10 |
| IV Bu/CY200/ATG10 | 3 |
| GVHD prophylaxis | |
| CSA + methylprednisolone + short MTX 10 mg/m2 (d+1, d+3, d+6) | 39 |
| CSA + methylprednisolone + CY7.5 mg/kg (d+1) + MTX 10 mg/m2 (d+3, d+6) | 32 |
| Median nucleated cell dose, ×108/kg (range) | 4.4 (1.4-10.8) |
| Median CD34+ cell dose, ×106/kg (range) | 7.1 (1.4-26) |
| Variable . | Patients (n = 71) . |
|---|---|
| Patient sex, male/female | 42/29 |
| Median patient age, y (range) | 9 (1.6-27) |
| Mean patient age, y (± SD) | 10.2 (± 5.2) |
| Patients older than 18 y, n | 4 |
| Mean patient weight ± SD, kg (range) | 26.4 ± 11.15 (10-60) |
| Median donor age, y (range) | 11 (1.3-52) |
| Diagnosis | |
| Thalassemia | 68 |
| Sickle cell anemia | 3 |
| Risk class (1/2/3/not applicable) | 6/23/39/3 |
| Median AST, IU/L (range) | 33 (13-216) |
| Median ALT, IU/L (range) | 38 (8-247) |
| Median bilirubin, mg/dL (range) | 0.9 (0.3-2.8) |
| Median serum ferritin, ng/mL (range) | 2126 (279-9458) |
| Serum ferritin > 2000 ng/mL, n | 38 |
| Median liver iron concentration, mg/g dw (range) | 17 (0.6-47.8) |
| Liver iron concentration > 7 mg/g dw, n | 55 |
| Median packed RBC units received before transplantation, n (range) | 95 (2-500) |
| Liver size ≤ 3 cm, n | 55 |
| Liver size > 3 cm, n | 16 |
| Splenectomy, yes/no | 17/54 |
| Liver fibrosis score (Ishak et al23 ; staging 0-6) | 1 (0-6) |
| Incomplete cirrhosis | 4 |
| Cirrhosis | 2 |
| Hepatitis C (HCV-RNA–positive) | 7 |
| Hepatitis B (HBs antigen–positive) | 1 |
| HLA-identical siblings | 62 |
| HLA-phenotypically identical parents or relatives | 9 |
| Donor/patient CMV serology | |
| Both positive | 60 |
| Any positive | 9 |
| Both negative | 2 |
| Conditioning regimen | |
| IV Bu/CY200 | 11 |
| IV Bu/TT10/CY200 | 14 |
| IV Bu/CY160 preceded by HU AZA FL20 | 15 |
| IV Bu/TT10/CY160 preceded by HU AZA FL30 | 15 |
| IV Bu/TT10/CY90 preceded by HU AZA FL30 | 3 |
| IV Bu/TT10/CY200/ATG10 preceded by HU AZA FL30 | 10 |
| IV Bu/CY200/ATG10 | 3 |
| GVHD prophylaxis | |
| CSA + methylprednisolone + short MTX 10 mg/m2 (d+1, d+3, d+6) | 39 |
| CSA + methylprednisolone + CY7.5 mg/kg (d+1) + MTX 10 mg/m2 (d+3, d+6) | 32 |
| Median nucleated cell dose, ×108/kg (range) | 4.4 (1.4-10.8) |
| Median CD34+ cell dose, ×106/kg (range) | 7.1 (1.4-26) |
RBC indicates red blood cell; IV, intravenous; TT, thiotepa; HU, hydroxyurea; AZ, azathioprine; FLU, fludarabine; ATG, thymoglobulin; MTX, methotrexate; CSA, cyclosporine A; and dw, dry weight.