Table 2.
HSCT studies with Reduced or Low Intensity Conditioning HCT for children with SCD in the last decade (n>5 patients)
| Author/year | Matthes-Martin, 2013 (4) |
Bhatia, 2014 (5) | King et al. 2015, (6) |
Guilcher, 2019 (7) | Ngwube A, 2020 (8) |
|---|---|---|---|---|---|
| n | 8 | 18 | 43 | 16 | 14 |
|
Age at transplant:
median (range) |
9 (2.1 - 17) | 8.9 (2.3–20.2) | 11.5 (0.8 -20.3) | 12 (3 -18) | 13 (7-21) |
| Donor type | MSD | MSD | MSD | MSD | MSD*/URD |
| Graft source | BM/CB | BM/CB | BM/CB | PBSC | BM/CB |
| Conditioning regimen | Flu/Mel/TT or TLI/ATG or Alemtuzumab |
BU/Flu/Alemtuzu mab |
Flu/Mel/Alemtuzu mab |
TBI(300 cGy)/Alemtuzuma b |
Flu/Mel/Alemtuzumab+thi otepa(UD) |
| GvHdprophylaxis | CsA or TAC/MMF |
TAC/MMF | CsA or TAC+/- MTX or MMF +/- PRED |
Sirolimus | Abatacept/TAC and MTX |
| OS (%) | 100% | 100% | 93% | 100% | 100% |
| aGvHD (%) (n) | 0 | 17% | 23% | 0 | 7% (III- IV)-28% (I-II) |
| cGvHD (%) (n) | 0 | 11% | 13% | 0 | 57% |
| Graft Rejection | 0 | 0 | 0 | 0 | 7,1% |
|
Neutrophil engraftment:
median day (range) |
19 (17– 27) | 16 (0–41) | 13 (5–21) | 22 (20.5-25) | 14(10-24) |
| DFS | 100% | 100% | 90.7% | 100% | 92.9% |
| TRM | 0 | 0 | 5.7% | 0 | 0 |
Abbreviations: MSD, matched sibling donor; URD, unrelated donors; BM, Bone marrow; CB, cord blood; PBSC, Peripheral Blood Stem Cell; Flu, fludarabine; Mel, melphalan; TT, Thiotepa; TLI, total lymphoid irradiation; ATG, anti-thymocyte globulin; Bu, busulfan; TBI, total body irradiation; CsA, cyclosporine A, TAC, tacrolimus ; MMF, mycophenolat emofetil; OS, overall survival; aGvHD, acute graft-versus-host disease; cGVHD, chronic graft-versus-host disease; DFS, disease-free survival; TRM, transplant related mortality