Table 6.
Outcome of the largest cohort of patients with FA who underwent transplantation from haploidentical donors
| Reference | Patients (n) | Prevalently using conditioning regimen | Follow-up | aGVHD | cGVHD | TRM/NRM | OS | Malignancies, % |
|---|---|---|---|---|---|---|---|---|
| Bonfim et al53 (2017) | 26 | Flu-mini TBI ± ATG + PTCY | 30 months | 75% no ATG 14.2% ATG |
83% no ATG 14.2% ATG |
41.6% no ATG 21.4% ATG |
72.6% at 1 year | NR |
| Ayas et al54 (2019) | 19 | Flu-mini TBI-ATG + PTCY | 38.3 + 5.8 months | Grades III-IV 15.7% | Extensive in 1 patient (5.3%) | 5.2% | 89.2% + 7.2% at 5 years | None |
| Bonfim et al34 (2022) | 49 | Flu-mini TBI-ATG or alemtuzumab | 3.8 years | Grades II-IV 28% | 26% | 8/49 (16.3%) | 82% at 2 years | 4% |
| Strocchio et al33 (2021) | 24 | Flu-CY-mini TBI Alfa/beta/CD19 depletion |
5.2 years | Grades I-II 17.4% | 5.5% | 0 | 100% at 5 years | 4.1% |
ATG, antithymocyte globulin; Cy, cyclophosphamide; Flu, fludarabine; NR, not reported; PTCY, posttransplantation cyclophosphamide; TBI, total body irradiation; TRM/NRM, transplant-related mortality/nonrelapse mortality.