Table 1.
Patient characteristics at transplantation for 31 thalassemia patients
| UPN | Age (years) |
Sex | Donor, HLA- match |
Anti-HLA antibody |
Ferritin (ng/mL) |
Risk | CD34+ cell dose (×106/kg) |
Outcome | Chimerism (%) | F/U time (months) |
Off IS |
|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 19.9 | F | Father, 5/10 | Negative | 1495 | Class 3 HR | 8.9 | Aa | 100% donor | 33 | Yes |
| 2 | 15.8 | F | Mother, 7/10 | Negative | 3613 | Class 3 HR | 11.6 | A | 100% donor | 28 | Yes |
| 3 | 1.7 | M | Mother, 7/10 | Negative | 1466 | Class 1 | 10.3 | A | 100% donor | 24 | Yes |
| 4 | 19.5 | M | Mother, 6/10 | Negative | 2552 | Class 3 HR | 11.6 | A | 100% donor | 23 | Yes |
| 5 | 14.3 | F | Mother, 5/10 | Negative | 2276 | Class 3 HR | 14.8 | A | 100% donor | 22 | Yes |
| 6 | 4.3 | M | Father, 7/10 | Negative | 1915 | Class 1 | 13.0 | A | 100% donor | 22 | Yes |
| 7 | 17.4 | F | Father, 5/10 | Positive | 2645 | Class 3 HR | 4.0 | Da | 100% donorb | 8 | N/A |
| 8 | 7.4 | M | Mother, 5/10 | Negative | 994 | Class 2 | 11.5 | A | 100% donor | 19 | Yes |
| 9 | 9.6 | M | Mother, 6/10 | Negative | 1599 | Class 2 | 13.0 | A | 100% donor | 19 | Yes |
| 10 | 18.6 | F | Mother, 6/10 | Positive | 3200 | Class 3 HR | 10.0 | A | 100% donor | 18 | Yes |
| 11 | 2.4 | F | Father, 6/10 | Negative | 956 | Class 1 | 15.0 | A | 100% donor | 15 | Yes |
| 12 | 7.5 | M | Mother, 5/10 | Positive | 1799 | Class 2 | 19.0 | A | Graft failure- autologous reconstitution |
15 | Rejected |
| 13 | 13.3 | M | Father, 5/10 | Negative | 3116 | Class 3 HR | 10.0 | A | 100% donor | 14 | Yes |
| 14c | 12.9 | F | Father, 5,10 | Negative | 2948 | Class 3 HR | 11.5 | A | 100% donor | 14 | Yes |
| 15 | 7.2 | M | Mother, 6/10 | Negative | 887 | Class 2 | 10.4 | A | 100% donor | 13 | Yes |
| 16 | 11.7 | M | Mother, 5/10 | Negative | 1540 | Class 3 HR | 8.5 | A | 100% donor | 12 | Yes |
| 17 | 10.8 | F | Mother, 5/10 | Negative | 2915 | Class 3 HR | 13.8 | A | 100% donor | 12 | Yes |
| 18 | 6.9 | F | Mother, 5/10 | Negative | 2487 | Class 1 | 15.0 | A | 100% donor | 11 | Yes |
| 19 | 14.2 | M | Mother, 7/10 | Negative | 1729 | Class 3 HR | 12.6 | A | 100% donor | 10 | Yes |
| 20 | 9.7 | M | Mother, 5/10 | Negative | 1756 | Class 2 | 13.0 | A | 100% donor | 10 | Yes |
| 21 | 7.3 | F | Mother, 5/10 | Negative | 1669 | Class 2 | 9.1 | A | 100% donor | 10 | Yes |
| 22 | 6.8 | F | Father, 6/10 | Negative | 1695 | Class 2 | 11.7 | A | 100% donor | 10 | Yes |
| 23 | 7.7 | F | Mother, 7/10 | Negative | 4341 | Class 2 | 12.4 | A | 100% donor | 10 | Yes |
| 24 | 9.8 | M | Father, 5/10 | Negative | 1131 | Class 3 HR | 10.6 | A | 100% donor | 9 | Yes |
| 25 | 6.6 | F | Father, 6/10 | Negative | 2894 | Class 2 | 13.9 | A | 100% donor | 9 | Yes |
| 26 | 11.0 | M | Mother, 6/10 | Negative | 1796 | Class 3 HR | 10.6 | A | 100% donor | 7 | Yes |
| 27 | 3.8 | M | Mother, 5/10 | Negative | 1460 | Class 1 | 11.6 | A | 100% donor | 7 | No |
| 28 | 2.1 | M | Father, 8/10 | Negative | 905 | Class 1 | 13.3 | A | 100% donor | 7 | No |
| 29 | 3.1 | M | Mother, 7/10 | Negative | 729 | Class 1 | 12.4 | A | 100% donor | 7 | No |
| 30 | 16.0 | F | Mother, 7/10 | Negative | 5100 | Class 3 HR | 9.2 | A | 100% donor | 7 | No |
| 31 | 15.0 | M | Father, 5/10 | Negative | 1518 | Class 3 HR | 11.5 | A | 100% donor | 7 | No |
Abbreviations: Class 3 HR = Class 3 high risk (age ≥ 7 years and hepatomegaly (liver ≥ 5 cms below right costal margin);9,22 F = female; IS = immunosuppression; M = male; N/A = not applicable.
a
A = Alive; D = dead.
b
100% donor chimerism; initial graft failure, engrafted after additional CD34 + cells were administered, please see text for details.
c
Haploidentical transplantation as a second transplantation.