Table 1.
Patient characteristics.
| Case | Age at SCT (y.o.) | Family history | Type of transplant | HLA compatibility for GVH direction at the allelic level | Conditioning regimen | GVHD prophylaxis | VLCFA before SCT |
Loes score |
NFS |
Engraftment (day) |
Outcome (months) | ||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| C24:0/C22:0 | C26:0/C22:0 | Before SCT | After SCT (months) | Before SCT | After SCT | 1st SCT | 2nd SCT | ||||||||
| 1 | 11 | − | UR-CBT | 8/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 1.184 | 0.020 | 14 (IC−) | 12 (52) | 1 | 3 | 19 | Alive (91) | |
| 2 | 9 | − | UR-CBT | 6/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 1.166 | 0.018 | 18 (IC−) | 14 (72) | 2 | 8 | 24 | Alive (88) | |
| 3 | 10 | + | ID-SIB-BMT | 6/6 | FLU+MEL+TBI 4Gy | CSA + MTX | 1.270 | 0.048 | 13 (IC+) | 21 (51) | 1 | 19 | 15 | Alive (86) | |
| 4 | 6 | + | UR-CBT | 6/8 | FLU+MEL+TBI 3Gy | TAC + MTX | 1.500 | 0.054 | 16 (IC−) | 14 (58) | 1 | 3 | − | 20 | Alive (76) |
| 5 | 6 | − | ID-SIB-BMT | 8/8 | FLU+MEL+TBI 4Gy | CSA + MTX | 1.727 | 0.204 | 11 (IC−) | 12 (46) | 1 | 2 | 16 | Alive (73) | |
| 6 | 10 | − | UR-CBT | 6/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 1.706 | 0.134 | 10 (IC−) | 14 (55) | 2 | 2 | 21 | Alive (69) | |
| 7 | 11 | − | UR-CBT | 6/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 1.149 | 0.020 | 15.5 (IC−) | 14 (39) | 1 | 11 | 22 | Alive (61) | |
| 8 | 4 | + | UR-CBT | 8/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 2.030 | 0.119 | 13 (IC+) | 24 (36) | 6 | 21 | 21 | Alive (46) | |
| 9 | 9 | − | UR-CBT | 8/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 1.690 | 0.089 | 14 (IC−) | 14 (26) | 2 | 3 | 20 | Alive (45) | |
| 10 | 10 | − | UR-CBT | 7/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 2.000 | 0.099 | 12 (IC−) | 23 (23) | 4 | 14 | 29 | Dead (32) | |
| 11 | 10 | − | UR-CBT | 6/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 1.429 | 0.026 | 15 (IC+) | 16.5 (1) | 2 | − | − | − | Alive (36) |
| 12 | 14 | + | UR-CBT | 6/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 1.628 | 0.023 | 13 (IC−) | 14 (18) | 0 | 1 | − | 25 | Alive (26) |
| 13 | 14 | + | UR-CBT | 6/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 1.438 | 0.022 | 0 (IC−) | 0 (8) | 0 | 0 | − | 20 | Alive (19) |
| 14 | 3 | + | UR-CBT | 7/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 2.320 | 0.149 | 0 (IC−) | 0 (14) | 0 | 0 | 23 | Alive (29) | |
| 15 | 5 | − | UR-CBT | 6/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 1.710 | 0.076 | 11 (IC+) | 15.5 (7) | 2 | 25 | − | 21 | Alive (20) |
| 16 | 5 | − | UR-CBT | 8/8 | FLU+MEL+TBI 4Gy | TAC + MTX | 1.930 | 0.116 | 20 (IC+) | 31 (7) | 2 | 22 | 19 | Alive (16) | |
SCT: stem cell transplantation, y.o.: years old, UR-CBT: unrelated cord blood transplantation, ID-SIB-BMT: bone marrow transplantation from an HLA identical sibling, HLA: human leukocyte antigen,
GVH: graft-versus-host, FLU: fludarabine 125 mg/m2, MEL: melphalan 140 mg/m2, TBI: total body irradiation, TAC: tacrolimus, MTX: methotrexate, CSA:cyclosporine,
IC−: No involvement of internal capsule, IC+: Involvement of internal capsule, VLCFA: very long chain fatty acid, NFS: Neurologic function score.