Patient, disease, and transplantation characteristics
| Variable . | N (%) or median (range) . |
|---|---|
| No. of patients | 179 |
| Age at transplantation, years | |
| < 5 | 57 (32) |
| 6-9 | 55 (31) |
| 10-20 | 67 (37) |
| Male | 98 (55) |
| Karnofsky-Lansky score before transplantation | |
| < 90% | 7 (4) |
| ≥ 90% | 169 (94) |
| Not reported | 3 (2) |
| CMV serostatus | |
| Donor negative/recipient negative | 28 (16) |
| Donor negative/recipient positive | 16 (9) |
| Donor positive/recipient negative | 11 (6) |
| Donor positive/recipient positive | 100 (56) |
| Not reported | 24 (13) |
| Total serum bilirubin before conditioning, μM | |
| Median (range) | 14 (3-104) |
| < 35 | 151 (84) |
| 35-50 | 9 (5) |
| 50-100 | 6 (3) |
| ≥ 100 | 2 (1) |
| Not reported | 11 (6) |
| Presence of portal fibrosis on liver biopsy | |
| Absent | 63 (35) |
| Present | 92 (51) |
| Not reported | 24 (13) |
| Liver size | |
| > 2 cm below costal margin | 83 (46) |
| ≤ 2 cm below costal margin | 78 (44) |
| Not reported | 18 (10) |
| Iron chelation therapy | |
| Inadequate | 163 (94) |
| Adequate* | 10 (6) |
| Not reported | 6 (3) |
| Pesaro risk class | |
| I | 3 (2) |
| II | 75 (42) |
| III | 64 (36) |
| Not reported | 37 (21) |
| Serum ferritin level before conditioning, μg/L | |
| < 2500 | 73 (41) |
| ≥ 2500 | 63 (35) |
| Not reported | 43 (24) |
| ALT/AST before conditioning | |
| Normal (< 2 times upper limit of normal) | 101 (56) |
| Elevated (≥ 2 times upper limit of normal) | 45 (25) |
| Not reported | 33 (18) |
| No. of blood transfusions before transplantation | |
| ≤ 20 | 13 (7) |
| 20-50 | 41 (23) |
| > 50 | 111 (62) |
| Not reported | 14 (8) |
| Interval from diagnosis to transplantation | |
| ≤ 5 years | 73 (41) |
| > 5 years | 102 (57) |
| Not reported | 4 (2) |
| Conditioning regimen | |
| Busulfan + cyclophosphamide + ATG | 77 (43) |
| Busulfan + cyclophosphamide without ATG | 102 (57) |
| Donor-recipient gender match | |
| Male donor/male recipient | 42 (23) |
| Male donor/female recipient | 37 (21) |
| Female donor/male recipient | 56 (31) |
| Female donor/female recipient | 44 (25) |
| GVHD prophylaxis | |
| Cyclosporine + methotrexate | 152 (85) |
| Cyclosporine with or without other (not methotrexate) | 23 (13) |
| Methorexate with or without other (not cyclosporine) | 2 (1) |
| None | 2 (1) |
| Year of transplantation | |
| 1995-1997 | 101 (56) |
| 1998-2001 | 78 (44) |
| Median follow-up of survivors, months | 73 (6-127) |
| Variable . | N (%) or median (range) . |
|---|---|
| No. of patients | 179 |
| Age at transplantation, years | |
| < 5 | 57 (32) |
| 6-9 | 55 (31) |
| 10-20 | 67 (37) |
| Male | 98 (55) |
| Karnofsky-Lansky score before transplantation | |
| < 90% | 7 (4) |
| ≥ 90% | 169 (94) |
| Not reported | 3 (2) |
| CMV serostatus | |
| Donor negative/recipient negative | 28 (16) |
| Donor negative/recipient positive | 16 (9) |
| Donor positive/recipient negative | 11 (6) |
| Donor positive/recipient positive | 100 (56) |
| Not reported | 24 (13) |
| Total serum bilirubin before conditioning, μM | |
| Median (range) | 14 (3-104) |
| < 35 | 151 (84) |
| 35-50 | 9 (5) |
| 50-100 | 6 (3) |
| ≥ 100 | 2 (1) |
| Not reported | 11 (6) |
| Presence of portal fibrosis on liver biopsy | |
| Absent | 63 (35) |
| Present | 92 (51) |
| Not reported | 24 (13) |
| Liver size | |
| > 2 cm below costal margin | 83 (46) |
| ≤ 2 cm below costal margin | 78 (44) |
| Not reported | 18 (10) |
| Iron chelation therapy | |
| Inadequate | 163 (94) |
| Adequate* | 10 (6) |
| Not reported | 6 (3) |
| Pesaro risk class | |
| I | 3 (2) |
| II | 75 (42) |
| III | 64 (36) |
| Not reported | 37 (21) |
| Serum ferritin level before conditioning, μg/L | |
| < 2500 | 73 (41) |
| ≥ 2500 | 63 (35) |
| Not reported | 43 (24) |
| ALT/AST before conditioning | |
| Normal (< 2 times upper limit of normal) | 101 (56) |
| Elevated (≥ 2 times upper limit of normal) | 45 (25) |
| Not reported | 33 (18) |
| No. of blood transfusions before transplantation | |
| ≤ 20 | 13 (7) |
| 20-50 | 41 (23) |
| > 50 | 111 (62) |
| Not reported | 14 (8) |
| Interval from diagnosis to transplantation | |
| ≤ 5 years | 73 (41) |
| > 5 years | 102 (57) |
| Not reported | 4 (2) |
| Conditioning regimen | |
| Busulfan + cyclophosphamide + ATG | 77 (43) |
| Busulfan + cyclophosphamide without ATG | 102 (57) |
| Donor-recipient gender match | |
| Male donor/male recipient | 42 (23) |
| Male donor/female recipient | 37 (21) |
| Female donor/male recipient | 56 (31) |
| Female donor/female recipient | 44 (25) |
| GVHD prophylaxis | |
| Cyclosporine + methotrexate | 152 (85) |
| Cyclosporine with or without other (not methotrexate) | 23 (13) |
| Methorexate with or without other (not cyclosporine) | 2 (1) |
| None | 2 (1) |
| Year of transplantation | |
| 1995-1997 | 101 (56) |
| 1998-2001 | 78 (44) |
| Median follow-up of survivors, months | 73 (6-127) |
CMV indicates cytomegalovirus; and ALT/AST, alanine aminotransferase/aspartate aminotransferase.
The adequacy of iron chelation is defined as the chelation therapy was initiated within 18 months of the first transfusion and administered subcutaneously for 8 to 10 hours per day for at least 5 days/week; otherwise, the iron chelation is deemed inadequate.