Review of publications on HSCT for patients with SCD
| Reference . | N . | Age, median (range), y . | Conditioning . | Stem-cell source (n) . | Follow-up, median (range), y . | OS, % . | EFS, % . | TRM, % . | Graft rejection, % . |
|---|---|---|---|---|---|---|---|---|---|
| 72 | 50 | 7.5 (0.9-23) | MAC | BM (48), UCB (2) | 5 (0.3-11) | 93 | 82 | 4 | 10 |
| 16 | 59 | 10.1 (3.3-15.9) | MAC | BM | 3.5 (1.0-9.6) | 93 | 84 | 6.7 | 10 |
| 14 | 7* | 9 (3.0-20) | RIC | BM (6), PBMC (1) | 2.3 (1.3-3.3) | 0 | 0 | 100 | |
| 7 | 87 | 9.5 (2-22) | MAC | BM (74), UCB (10), BM/UCB (2), PBMC (1) | 6.0 (2.0-17.9) | 93.1 | 86.1 | 7 | 22.6 (without ATG), 2.9 (with ATG) |
| 74 | 67 | 10 (2-27) | MAC | BM (54), UCB (4), PBSC (9) | 5.1(0.3-14.8) | 97 | 85 | 4 | 13 |
| 17 | 7 | 8 (6-18) | RIC | BM | 4.0 (2.0-8.5) | 86 | 0 | 14 | |
| 9 | 15 | 17 (16-27) | MAC | BM (14), PBMC (1) | 3.4 (1-16.1) | 93 | 93 | 7 | 0 |
| 18 | 17 | 30 (15-46) | RIC | BM | 1.9 (0.61-5.4) | 100 | 0 | 35 | |
| 73 | 8 | 9.1 (2-24) | RIC | BM (6), UCB (1), BM/PBSC (1) | 4 (1-7.7) | 100 | 100 | 0 | 0 |
| 19 | 30 | 28.5 (17-65) | RIC | PBSC | 3.4 (1-8.6) | 88 | 3 | 13 | |
| 75 | 18 | 8.9 (2.3-20.2) | MAC | BM (15), UCB (3) | 2.9 (0.37-7.48) | 100 | 100 | 0 | 0 |
| 76 | 13 | 30 (17-40) | RIC | PBSC | 1.8 (1.0-3.7) | 100 | 92 | 0 | 7.7 |
| 77 | 11 | 7 (2-13) | MAC | BM | 3.1 (1-5.7) | 90.9 | 81.9 | 9.1 | 9.1 |
| Reference . | N . | Age, median (range), y . | Conditioning . | Stem-cell source (n) . | Follow-up, median (range), y . | OS, % . | EFS, % . | TRM, % . | Graft rejection, % . |
|---|---|---|---|---|---|---|---|---|---|
| 72 | 50 | 7.5 (0.9-23) | MAC | BM (48), UCB (2) | 5 (0.3-11) | 93 | 82 | 4 | 10 |
| 16 | 59 | 10.1 (3.3-15.9) | MAC | BM | 3.5 (1.0-9.6) | 93 | 84 | 6.7 | 10 |
| 14 | 7* | 9 (3.0-20) | RIC | BM (6), PBMC (1) | 2.3 (1.3-3.3) | 0 | 0 | 100 | |
| 7 | 87 | 9.5 (2-22) | MAC | BM (74), UCB (10), BM/UCB (2), PBMC (1) | 6.0 (2.0-17.9) | 93.1 | 86.1 | 7 | 22.6 (without ATG), 2.9 (with ATG) |
| 74 | 67 | 10 (2-27) | MAC | BM (54), UCB (4), PBSC (9) | 5.1(0.3-14.8) | 97 | 85 | 4 | 13 |
| 17 | 7 | 8 (6-18) | RIC | BM | 4.0 (2.0-8.5) | 86 | 0 | 14 | |
| 9 | 15 | 17 (16-27) | MAC | BM (14), PBMC (1) | 3.4 (1-16.1) | 93 | 93 | 7 | 0 |
| 18 | 17 | 30 (15-46) | RIC | BM | 1.9 (0.61-5.4) | 100 | 0 | 35 | |
| 73 | 8 | 9.1 (2-24) | RIC | BM (6), UCB (1), BM/PBSC (1) | 4 (1-7.7) | 100 | 100 | 0 | 0 |
| 19 | 30 | 28.5 (17-65) | RIC | PBSC | 3.4 (1-8.6) | 88 | 3 | 13 | |
| 75 | 18 | 8.9 (2.3-20.2) | MAC | BM (15), UCB (3) | 2.9 (0.37-7.48) | 100 | 100 | 0 | 0 |
| 76 | 13 | 30 (17-40) | RIC | PBSC | 1.8 (1.0-3.7) | 100 | 92 | 0 | 7.7 |
| 77 | 11 | 7 (2-13) | MAC | BM | 3.1 (1-5.7) | 90.9 | 81.9 | 9.1 | 9.1 |
ATG, antithymocyte globulin; BM, bone marrow; EFS, event-free survival; MAC, myeloablative conditioning; PBMC, peripheral blood mononuclear cell; PBSC, peripheral blood stem cell; RIC, reduced-intensity conditioning; TRM, transplantation-related mortality; UCB, umbilical cord blood.
Includes 1 patient with β-thalassemia major.