Patient and donor data, transplantation regimen, and outcome
| Patient no. . | Patient sex/age . | Donor sex/age . | Iron chelation . | Portal fibrosis . | Liver iron grade (ciot) . | Risk class . | Conditioning regimen . | Cellular dose NC × 108/kg . | Take . | aGVHD grade . | cGVHD grade . | Outcome . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1* | F/17 | F/42 | Ir | MO | III | III | BU14-CY160 | 3.9 | Yes | 0 | 0 | Alive with graft |
| 2† | M/9 | F/44 | R | MI | II | II | BU14-CY160 | 2.6 | No | NE | NE | Alive with thalassemia |
| 3* | M/4 | F/32 | R | – | – | I | BU14-CY200 | 1.9 | Yes | 0 | 0 | Alive with graft |
| 4 | M/7 | F/31 | Ir | MO | II | III | BU14-CY200 | 4.0 | No | NE | NE | Alive with thalassemia |
| 5† | F/9 | M/37 | Ir | MO | II | II | BU14-TT10-CY200 | 5.8 | Yes | II | 0 | Alive with graft |
| 6 | F/15 | M/36 | R | MI | II | II | BU14-TT10-CY200 | 2.6 | Yes | III | L | Alive with graft |
| 7† | M/16 | M/32 | Ir | MI | II | III | BU14-TT10-CY160 | 3.0 | Yes | II | 0 | Alive with graft |
| 8 | M/19 | M/35 | Ir | SE | IV | III | BU14-TT10-CY160 | 3.9 | Yes | II | E | Died (day + 470) |
| 9† | M/17 | M/36 | Ir | MO | III | III | BU14-TT10-CY160 | 2.3 | Yes | 0 | L | Alive with graft |
| 10* | F/12 | M/38 | R | MI | II | II | BU14-TT10-CY200 | 4.2 | Yes | 0 | 0 | Alive with graft |
| 11† | M/18 | F/40 | Ir | SE | IV | III | BU14-TT10-CY160 | 2.9 | Yes | II | 0 | Died (day + 125) |
| 12* | M/6 | M/43 | R | MI | I | II | BU14-TT10-CY200 | 5.8 | Yes | 0 | 0 | Alive with graft |
| 13* | M/14 | F/30 | R | MI | II | II | BU14-TT10-CY200 | 3.4 | Yes | 0 | NE | Died (day + 64) |
| 14 | M/6 | F/41 | Ir | SE | IV | III | BU14-TT10-CY160 | I0.0 | No‡ | NE | NE | Alive with thalassemia |
| 15 | M/25 | F/34 | Ir | SE | IV | III | BU14-TT10-CY120 | 3.5 | Yes | IV | L | Alive with graft |
| 16 | F/15 | F/39 | Ir | MO | III | III | BU14-TT10-CY160 | 1.8 | Yes | III | NE | Died (day + 66) |
| 17† | M/5 | F/45 | R | MO | II | II | BU14-TT10-CY200 | 3.9 | Yes | 0 | 0 | Alive with graft |
| 18† | F/2 | F/25 | R | NO | I | I | BU14-TT10-CY200 | 5.5 | Yes | II | 0 | Alive with graft |
| 19 | F/13 | M/36 | R | MO | II | II | BU14-TT10-CY200 | 2.6 | Yes | I | 0 | Alive with graft |
| 20* | M/15 | M/26 | R | MI | II | II | BU14-TT10-CY200 | 4.5 | No‡ | NE | NE | Alive with thalassemia |
| 21† | F/28 | F/31 | Ir | SE | III | III | BU14-TT10-CY120 | 3.7 | Yes | II | 0 | Alive with graft |
| 22† | F/26 | M/34 | Ir | SE | III | III | BU14-TT10-CY120 | 3.7 | Yes | NE | NE | Died (day + 17) |
| 23* | F/15 | M/36 | R | MI | I | II | BU14-TT10-CY200 | 4.1 | Yes | 0 | 0 | Alive with graft |
| 24 | M/26 | F/39 | Ir | MI | III | III | BU14-TT10-CY120 | 7.5 | Yes | 0 | 0 | Alive with graft |
| 25 | M/24 | M/50 | Ir | MO | III | III | BU14-TT10-CY120 | 3.3 | Yes | IV | E | Died (day + 269) |
| 26† | F/18 | M/33 | Ir | MO | III | III | BU14-TT10-CY160 | 3.8 | Yes | 0 | 0 | Alive with graft |
| 27† | M/11 | F/25 | R | MI | II | II | BU14-TT10-CY200 | 3.9 | Yes | 0 | 0 | Alive with graft |
| 28 | M/7 | M/30 | R | NO | I | I | BU14-TT10-CY200 | 7.0 | Yes | 0 | 0 | Alive with graft |
| 29† | M/14 | M/52 | R | MI | II | III | BU14-TT10-CY200 | 3.0 | Yes | III | L | Alive with graft |
| 30† | M/21 | M/36 | Ir | MO | III | III | BU14-TT10-CY120 | 3.8 | Yes | 0 | 0 | Alive with graft |
| 31† | M/4 | F/31 | R | NO | I | I | BU14-TT10-CY200 | 4.0 | Yes | 0 | 0 | Alive with graft |
| 32† | M/16 | F/43 | Ir | SE | IV | III | BU14-TT10-CY160 | I1.6 | Yes | 0 | 0 | Alive with graft |
| Patient no. . | Patient sex/age . | Donor sex/age . | Iron chelation . | Portal fibrosis . | Liver iron grade (ciot) . | Risk class . | Conditioning regimen . | Cellular dose NC × 108/kg . | Take . | aGVHD grade . | cGVHD grade . | Outcome . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1* | F/17 | F/42 | Ir | MO | III | III | BU14-CY160 | 3.9 | Yes | 0 | 0 | Alive with graft |
| 2† | M/9 | F/44 | R | MI | II | II | BU14-CY160 | 2.6 | No | NE | NE | Alive with thalassemia |
| 3* | M/4 | F/32 | R | – | – | I | BU14-CY200 | 1.9 | Yes | 0 | 0 | Alive with graft |
| 4 | M/7 | F/31 | Ir | MO | II | III | BU14-CY200 | 4.0 | No | NE | NE | Alive with thalassemia |
| 5† | F/9 | M/37 | Ir | MO | II | II | BU14-TT10-CY200 | 5.8 | Yes | II | 0 | Alive with graft |
| 6 | F/15 | M/36 | R | MI | II | II | BU14-TT10-CY200 | 2.6 | Yes | III | L | Alive with graft |
| 7† | M/16 | M/32 | Ir | MI | II | III | BU14-TT10-CY160 | 3.0 | Yes | II | 0 | Alive with graft |
| 8 | M/19 | M/35 | Ir | SE | IV | III | BU14-TT10-CY160 | 3.9 | Yes | II | E | Died (day + 470) |
| 9† | M/17 | M/36 | Ir | MO | III | III | BU14-TT10-CY160 | 2.3 | Yes | 0 | L | Alive with graft |
| 10* | F/12 | M/38 | R | MI | II | II | BU14-TT10-CY200 | 4.2 | Yes | 0 | 0 | Alive with graft |
| 11† | M/18 | F/40 | Ir | SE | IV | III | BU14-TT10-CY160 | 2.9 | Yes | II | 0 | Died (day + 125) |
| 12* | M/6 | M/43 | R | MI | I | II | BU14-TT10-CY200 | 5.8 | Yes | 0 | 0 | Alive with graft |
| 13* | M/14 | F/30 | R | MI | II | II | BU14-TT10-CY200 | 3.4 | Yes | 0 | NE | Died (day + 64) |
| 14 | M/6 | F/41 | Ir | SE | IV | III | BU14-TT10-CY160 | I0.0 | No‡ | NE | NE | Alive with thalassemia |
| 15 | M/25 | F/34 | Ir | SE | IV | III | BU14-TT10-CY120 | 3.5 | Yes | IV | L | Alive with graft |
| 16 | F/15 | F/39 | Ir | MO | III | III | BU14-TT10-CY160 | 1.8 | Yes | III | NE | Died (day + 66) |
| 17† | M/5 | F/45 | R | MO | II | II | BU14-TT10-CY200 | 3.9 | Yes | 0 | 0 | Alive with graft |
| 18† | F/2 | F/25 | R | NO | I | I | BU14-TT10-CY200 | 5.5 | Yes | II | 0 | Alive with graft |
| 19 | F/13 | M/36 | R | MO | II | II | BU14-TT10-CY200 | 2.6 | Yes | I | 0 | Alive with graft |
| 20* | M/15 | M/26 | R | MI | II | II | BU14-TT10-CY200 | 4.5 | No‡ | NE | NE | Alive with thalassemia |
| 21† | F/28 | F/31 | Ir | SE | III | III | BU14-TT10-CY120 | 3.7 | Yes | II | 0 | Alive with graft |
| 22† | F/26 | M/34 | Ir | SE | III | III | BU14-TT10-CY120 | 3.7 | Yes | NE | NE | Died (day + 17) |
| 23* | F/15 | M/36 | R | MI | I | II | BU14-TT10-CY200 | 4.1 | Yes | 0 | 0 | Alive with graft |
| 24 | M/26 | F/39 | Ir | MI | III | III | BU14-TT10-CY120 | 7.5 | Yes | 0 | 0 | Alive with graft |
| 25 | M/24 | M/50 | Ir | MO | III | III | BU14-TT10-CY120 | 3.3 | Yes | IV | E | Died (day + 269) |
| 26† | F/18 | M/33 | Ir | MO | III | III | BU14-TT10-CY160 | 3.8 | Yes | 0 | 0 | Alive with graft |
| 27† | M/11 | F/25 | R | MI | II | II | BU14-TT10-CY200 | 3.9 | Yes | 0 | 0 | Alive with graft |
| 28 | M/7 | M/30 | R | NO | I | I | BU14-TT10-CY200 | 7.0 | Yes | 0 | 0 | Alive with graft |
| 29† | M/14 | M/52 | R | MI | II | III | BU14-TT10-CY200 | 3.0 | Yes | III | L | Alive with graft |
| 30† | M/21 | M/36 | Ir | MO | III | III | BU14-TT10-CY120 | 3.8 | Yes | 0 | 0 | Alive with graft |
| 31† | M/4 | F/31 | R | NO | I | I | BU14-TT10-CY200 | 4.0 | Yes | 0 | 0 | Alive with graft |
| 32† | M/16 | F/43 | Ir | SE | IV | III | BU14-TT10-CY160 | I1.6 | Yes | 0 | 0 | Alive with graft |
Patient shared 2 extended haplotypes.
Patient shared one extended haplotype.
Take followed by rejection.
aGVHD indicates acute graft-versus-host disease; cGVHD, chronic graft-versus-host disease; R, regular; Ir, irregular; NO, none; MI, mild; MO, moderate; SE, severe; BU, busulphan; CY, cyclophosphamide; TT, thiotepa; NC, nucleated cells; NE, not evaluable; L, limited; E, extensive.