Description of patients needed second interventions, including genetic background, comorbidities, second intervention characteristics, and outcomes
| . | P10 . | P12 . | P21 . | P25 . | P40 . | P41 . | |||
|---|---|---|---|---|---|---|---|---|---|
| CGD | gp91 | p67 | gp91 | gp91 | gp91 | p22 | |||
| Comorbidities | Colitis Perianal disease Lung involvement | Colitis | Lung involvement | Lung involvement Liver abscess | Colitis Perianal disease | Lung involvement Colitis Perianal disease | |||
| Age at first HSCT (years) | 11.4 | 3.2 | 4.4 | 7.2 | 6.4 | 13.2 | |||
| Donor | MUD | MUD | MUD | MSD | MUD | MUD | |||
| Source | BM | PBSC | PBSC | BM | PBSC | PBSC | |||
| Conditioning | Camp/Bu/Cy (MAC) | Camp/Flu/Treo (RIC) | Camp/Flu/Melph (RIC) | Bu/Cy (RIC) | Camp/Flu/Treo (RIC) | Camp/Flu/Treo (RIC) | |||
| aGvHD/cGvHD | No/Yes | No/No | Yes/Yes | No/No | Yes/yes | No/No | |||
| Type of second intervention | DLI (1 × 107/kg) | Second HSCT | DLI (1 × 106/kg) | Second HSCT | Gene therapy | Second HSCT | DLI (1, 5, 10 × 107/kg) | DLI (1 × 107/kg) | DLI (1, 5 × 107/kg) |
| Time from first HSCT to second intervention (years) | 1.7 | 3.3 | 0.3 | 1.6 | 1.3 | 3.9 | 9.8 | 1 | 1.4 |
| Cause of second intervention | Declined in donor chimerism | Declined in donor chimerism | Declined in donor chimerism | Declined in donor chimerism | Secondary graft failure | Persistent graft failure post gene therapy | Declined in donor chimerism | Declined in donor chimerism | Declined in donor chimerism |
| CGD-related disease before second intervention | Relapse of colitis | Relapse of colitis | None | None | De novo colitis Recurrence of lung involvement | None | De novo perianal fistulizing disease | No | No |
| Chimerism at second intervention | WB 19% | WB 11% | CD3 61% CD15 29% | CD3 58% CD15 0% | WB 0% | WB 0% | CD3 48% CD15 23% | CD3 59% CD15 19% | CD3 57% CD15 11% |
| Age at second intervention (years) | 13.1 | 14.8 | 3.5 | 4.7 | 5.7 | 8.3 | 17 | 7.4 | 14.6 |
| If second HSCT, specify donor-source-conditioning-aGvHD/cGvHD | MMUD-PBSC- Camp/Flu/Treo (RIC) No/Yes | MUD-PBSC-Camp/Flu/Bu (MAC) Yes (stage-II skin)/No | MUD-PBSC-Camp/Bu/Cy (RIC) Yes/Yes (eyes and skin-limited sclerodermatous) | / | / | / | |||
| CGD-related disease after second intervention | None | None | None | Fistulizing disease | None | None | |||
| On IS at last FU | No | No | No | No | No | No | |||
| Chimerism at last FU | WB 100%, CD15 100% | CD3 100%, CD15 100% | CD3 100%, CD15 100% | CD3 51%, CD15 100% | CD3 100%, CD15 100% | CD3 59%, CD15 10%; patient declined second transplant | |||
| . | P10 . | P12 . | P21 . | P25 . | P40 . | P41 . | |||
|---|---|---|---|---|---|---|---|---|---|
| CGD | gp91 | p67 | gp91 | gp91 | gp91 | p22 | |||
| Comorbidities | Colitis Perianal disease Lung involvement | Colitis | Lung involvement | Lung involvement Liver abscess | Colitis Perianal disease | Lung involvement Colitis Perianal disease | |||
| Age at first HSCT (years) | 11.4 | 3.2 | 4.4 | 7.2 | 6.4 | 13.2 | |||
| Donor | MUD | MUD | MUD | MSD | MUD | MUD | |||
| Source | BM | PBSC | PBSC | BM | PBSC | PBSC | |||
| Conditioning | Camp/Bu/Cy (MAC) | Camp/Flu/Treo (RIC) | Camp/Flu/Melph (RIC) | Bu/Cy (RIC) | Camp/Flu/Treo (RIC) | Camp/Flu/Treo (RIC) | |||
| aGvHD/cGvHD | No/Yes | No/No | Yes/Yes | No/No | Yes/yes | No/No | |||
| Type of second intervention | DLI (1 × 107/kg) | Second HSCT | DLI (1 × 106/kg) | Second HSCT | Gene therapy | Second HSCT | DLI (1, 5, 10 × 107/kg) | DLI (1 × 107/kg) | DLI (1, 5 × 107/kg) |
| Time from first HSCT to second intervention (years) | 1.7 | 3.3 | 0.3 | 1.6 | 1.3 | 3.9 | 9.8 | 1 | 1.4 |
| Cause of second intervention | Declined in donor chimerism | Declined in donor chimerism | Declined in donor chimerism | Declined in donor chimerism | Secondary graft failure | Persistent graft failure post gene therapy | Declined in donor chimerism | Declined in donor chimerism | Declined in donor chimerism |
| CGD-related disease before second intervention | Relapse of colitis | Relapse of colitis | None | None | De novo colitis Recurrence of lung involvement | None | De novo perianal fistulizing disease | No | No |
| Chimerism at second intervention | WB 19% | WB 11% | CD3 61% CD15 29% | CD3 58% CD15 0% | WB 0% | WB 0% | CD3 48% CD15 23% | CD3 59% CD15 19% | CD3 57% CD15 11% |
| Age at second intervention (years) | 13.1 | 14.8 | 3.5 | 4.7 | 5.7 | 8.3 | 17 | 7.4 | 14.6 |
| If second HSCT, specify donor-source-conditioning-aGvHD/cGvHD | MMUD-PBSC- Camp/Flu/Treo (RIC) No/Yes | MUD-PBSC-Camp/Flu/Bu (MAC) Yes (stage-II skin)/No | MUD-PBSC-Camp/Bu/Cy (RIC) Yes/Yes (eyes and skin-limited sclerodermatous) | / | / | / | |||
| CGD-related disease after second intervention | None | None | None | Fistulizing disease | None | None | |||
| On IS at last FU | No | No | No | No | No | No | |||
| Chimerism at last FU | WB 100%, CD15 100% | CD3 100%, CD15 100% | CD3 100%, CD15 100% | CD3 51%, CD15 100% | CD3 100%, CD15 100% | CD3 59%, CD15 10%; patient declined second transplant | |||
Bu, busulfan; Camp, Campath; Cy, cyclophosphamide; Flu, fludarabine; FU, follow-up; Melph, melphalan; Treo, treosulfan.