Patient demographics and transplant characteristics
| Patient demographic . | No. (%) . |
|---|---|
| Sex | |
| Male | 35 (83.3%) |
| Female | 7 (16.7%) |
| Median age in years at first HSCT (range) | 6 (0.9–15.2 years) |
| Median age in years at last follow-up (range) | 16.8 (6.0–22.0 years) |
| Median follow-up time post first HSCT in years (range) | 8 (2.4–13.8 years) |
| CGD diagnosis | 42 |
| XL-CGD | 29 (69.0%) |
| p47 | 8 (19.0%) |
| p67 | 3 (7.1%) |
| p22 | 2 (4.8%) |
| Number of interventions | 45 |
| One HSCT | 42 (93.3%) |
| Post-HSCT DLI | 3 (6.6%) |
| DLI followed by second HSCT | 2 (4.4%) |
| Post-HSCT gene therapy | 1 (2.2%) |
| Donor | 45 |
| MUD | 23 (51.1%) |
| MSD | 11 (24.4%) |
| MMUD | 10 (22.2%) |
| MFD | 1 (2.2%) |
| Conditioning intensity | 45 |
| Myeloablative (MAC) | 11 (24.4%) |
| —Bu/Cy | 6 (13.3%) |
| —Bu/Flu | 3 (6.7%) |
| —Treo/Thio/Flu | 2 (4.4%) |
| Reduced intensity (RIC) | 34 (75.6%) |
| —Flu/Bu | 20 (44.4%) |
| —Flu/Treo | 12 (26.7%) |
| —Flu/Melph | 1 (2.2%) |
| —Bu/Cy | 1 (2.2%) |
| Transplant cells | 45 |
| Bone marrow | 25 (55.5%) |
| PBSC | 18 (40.0%) |
| Cord | 2 (4.4%) |
| GvHD prophylaxis | 45 |
| CSA + MMF | 35 (77.8%) |
| CSA + MTX | 6 (13.3%) |
| CSA + Methylprednisolone | 1 (2.2%) |
| CSA only | 2 (4.4%) |
| None | 1 (2.2%) |
| Patient demographic . | No. (%) . |
|---|---|
| Sex | |
| Male | 35 (83.3%) |
| Female | 7 (16.7%) |
| Median age in years at first HSCT (range) | 6 (0.9–15.2 years) |
| Median age in years at last follow-up (range) | 16.8 (6.0–22.0 years) |
| Median follow-up time post first HSCT in years (range) | 8 (2.4–13.8 years) |
| CGD diagnosis | 42 |
| XL-CGD | 29 (69.0%) |
| p47 | 8 (19.0%) |
| p67 | 3 (7.1%) |
| p22 | 2 (4.8%) |
| Number of interventions | 45 |
| One HSCT | 42 (93.3%) |
| Post-HSCT DLI | 3 (6.6%) |
| DLI followed by second HSCT | 2 (4.4%) |
| Post-HSCT gene therapy | 1 (2.2%) |
| Donor | 45 |
| MUD | 23 (51.1%) |
| MSD | 11 (24.4%) |
| MMUD | 10 (22.2%) |
| MFD | 1 (2.2%) |
| Conditioning intensity | 45 |
| Myeloablative (MAC) | 11 (24.4%) |
| —Bu/Cy | 6 (13.3%) |
| —Bu/Flu | 3 (6.7%) |
| —Treo/Thio/Flu | 2 (4.4%) |
| Reduced intensity (RIC) | 34 (75.6%) |
| —Flu/Bu | 20 (44.4%) |
| —Flu/Treo | 12 (26.7%) |
| —Flu/Melph | 1 (2.2%) |
| —Bu/Cy | 1 (2.2%) |
| Transplant cells | 45 |
| Bone marrow | 25 (55.5%) |
| PBSC | 18 (40.0%) |
| Cord | 2 (4.4%) |
| GvHD prophylaxis | 45 |
| CSA + MMF | 35 (77.8%) |
| CSA + MTX | 6 (13.3%) |
| CSA + Methylprednisolone | 1 (2.2%) |
| CSA only | 2 (4.4%) |
| None | 1 (2.2%) |
Patients (n = 42) and transplants (n = 45). Bu, busulfan; CSA, ciclosporin; Cy, cyclophosphamide; Flu, fludarabine; Melph, melphalan; MFD, matched family donor; MMF, mycophenolate; MTX, methotrexate; PBSC, peripheral blood stem cell; Thio, thiotepa; Treo, treosulfan.