Table 5.
Transplant outcomes for thalassemia
| Study | N | Conditioning | Graft failure |
aGVHD Grade 2–4 | cGVHD | OS (%) | DFS (%) |
|---|---|---|---|---|---|---|---|
| MSD | |||||||
| Lucarelli et al.106 | <17 years: Class 1–2: 515 Class 3: 73 Adult: 107 |
BU/CY ± TT BU/CY BU/CY | 4% 7% 4% |
NA | NA | Class 1–2: 88% Class 3: 87% Adult: 66% |
Class 1–2: 85% Class 3: 82% Adult: 62% |
| URD | |||||||
| Hongeng et al.32 | 49 (21 URD) | BU/CY/ATG | 14% | Related:32% Unrelated: 42% |
14% | 89% (all patients) | Related: 82% Unrelated: 71% |
| La Nasa et al.33 | 68 URD | BU/CY or BU/Flu ± TT | 14% | 40% (17% Gr 3–4) |
18% | 79% (class 1–2: 97% Class 3 66%) | 66% (class 1–2: 80% Class 3 55%) |
| La Nasa et al.34 | 27 | BU/CY or BU/TT/CY | 30% | 37% | 27% | 70% | 70% |
| Bernardo et al.30 | 40 URD | Treosulfan | 9% | 14% | 1 | 93% | 94% |
| Gaziev et al.67 | 16 | 0% | 6% | 19% | 13% | 94% | 94% |
| UCB | |||||||
| Jaing et al.69 | 51 | BU, CY, ATG | 21.6% | NA | NA | 74.5% | NA |
| Ruggeri et al.107 | 35 | MAC (30) RIC (5) |
15/35 | 23% (including sickle cell patients) | 16% | 8/35 | 21% |
| Jaing et al.68 | 45 (32 thalassemia) | BU, CY, ATG | 6/32 (19%) | 76% (all patients) | 35% (all patients) | 88% (allpatients) | 77% (all patients) |
| Haplo | |||||||
| Sodani, personal communication | 43 | Megadose CD34+ PBSCs + haplo-BM MNCs G-CSF/EPO |
NA | NA | NA | 89% | 58% EFS |
Abbreviations: aGVHD =acute GVHD; ATG= anti-thymocyte globulin; cGVHD =chronic GVHD; DFS=disease-free survival; G-CSF = granulocyte CSF; MAC=myeloablative; MNCs = mononuclear cells; MSD= matched sibling donor; N=number; NA= not available; PBSCs =PB stem cells; RIC= reduced intensity conditioning; TT =thiotepa; UCB=umbilical cord blood; URD=unrelated donor.