Table 5.
Outcome of the largest cohort of patients with FA who underwent transplantation from alternative donors
| Reference | Patients (n) | Donor type | Prevalently using conditioning | Follow-up | aGVHD | cGVHD | TRM/NRM | OS | Malignancies, No. (%) |
|---|---|---|---|---|---|---|---|---|---|
| MacMillan et al50 (2015) | 130 | 73 MUD (TCD) 39 1-2 MM (no TCD) CB 18 1Ag MM Unrelated (TCD) |
Flu + Cy + ATG + TBI (in the most recent patients) with thymic shielding | 7.7 years | 20% (grades II-IV) | 10% | NR | 58% at 5 years | 11/130 (8.4%) |
| Mehta et al51 (2017) | 45 | 25 MUD 14 MMUD 6 MMRD All TCD |
Bu-Flu-Cy-ATG | 41 months | 6.7% (0 grades III-IV) | 3/45 (6.7%) limited, 0 extensive | NR | 80% at 3 years | 1/45 (2.2%) |
| Ebens et al52 (2018) | 57 | All TCD 32 (m)MUD BM 20 (m)MUD UCB 2 MMRD 1 8/8 HLA parental BM 1 MMSD UCB + BM boost |
Flu-Cy-TBI-ATG | 6.2 years | 12% grades II-IV | 4/57 (7%) | NR | 86% at 5 years | NR |
| Zubicaray et al35 (2021) | 123 | MMUD (all TCD) | Flu-Cy-TBI-ATG | 44.8 months | 41% grades II-IV | 5% limited 10% extensive |
NR | 62% at 2 years | NR |
| Modified from Pierri et al.13 | |||||||||
| ATG, antithymocyte globulin; BM, bone marrow; Bus, busulfan; CB, cord blood; Cy, cyclophosphamide; Flu, fludarabine; HLA, human leukocyte antigen; IRR, irradiation in conditioning regimen; MM, mismatched; MMRD, mismatched related donor; MMUD, mismatched unrelated donor; MUD, matched unrelated donor; NR, not reported; TBI, total body irradiation; TRM/NRM, transplant-related mortality/nonrelapse mortality; UCB, unrelated cord blood. | |||||||||