Patient and transplantation characteristics and outcome after first HCT in children with CGD (N = 55)
| . | Data . |
|---|---|
| Patient characteristics | |
| Year of transplant | |
| 1998-2007 | 17 (31) |
| 2008-2017 | 38 (69) |
| Males | 51 (93) |
| Inheritance | |
| X-linked CGD | 45 (82) |
| Autosomal recessive CGD | 10 (18) |
| Median age at transplant (range), y | 5.3 (0.6-18.0) |
| Donor characteristics | |
| Type of donor | |
| Matched family donor | 20 (36) |
| Unrelated donor | 31 (56) |
| Haploidentical donor | 4 (8) |
| Stem cell source | |
| Marrow | 29 (53) |
| Peripheral blood | 24 (43) |
| Cord blood | 2 (4) |
| Stem cell dose, median (range) | |
| Marrow | |
| TNC, × 108/kg | 5.0 (1.6-24.4) |
| CD34, × 106/kg | 4.3 (0.75-53.0) |
| PB | |
| TNC, × 108/kg | 10.3 (1.2-22.8) |
| CD34, × 106/kg | 8.0 (3.0-29.2) |
| Transplant characteristics | |
| Conditioning regimen | |
| MAC | |
| Busulfan-cyclophosphamide | 21 (38) |
| Fludarabine-treosulfan-thiotepa | 4 (7) |
| RTC | |
| Treosulfan-fludarabine | 24 (45) |
| Busulfan-fludarabine | 4 (7) |
| Treosulfan-cyclophosphamide | 1 (2) |
| Fludarabine-melphalan | 1 (2) |
| Serotherapy | |
| None | 9 (16) |
| ATG | 4 (8) |
| Alemtuzumab | 42 (76) |
| GVHD prophylaxis | |
| None | 3 (5) |
| CSA alone | 2 (4) |
| CSA + MTX | 16 (29) |
| CSA + MMF | 33 (60) |
| CSA + steroid (for cord blood) | 1 (2) |
| Hematopoietic recovery | |
| Days to neutrophil recovery, median (range) | 16 (9-37) |
| Days to platelet recovery, median (range) | 18 (10-139) |
| Transplant-related complications | |
| Acute GvHD | |
| Grade II-IV | 10 (20) |
| Grade III-IV | 5 (9) |
| Chronic GvHD2 | 0 |
| Veno-occlusive disease, n (%) | 1 (2) |
| Patient receiving second procedures for decreasing chimerism | 7 (13) |
| Patients receiving unconditioned stem cell boost infusion, n | 3 |
| Median myeloid chimerism prior to unconditioned stem cell boost infusion (range), % | 17 (15-23) |
| Patients receiving fully conditioned second transplant, n | 4 |
| Median myeloid chimerism prior to fully conditioned second transplant (range), % | 11 (0-13) |
| Cause of death (n = 5), n | |
| Multiorgan failure | 2 |
| Grade IV acute GvHD | 1 |
| Pulmonary hemorrhage | 1 |
| Posttransplant lymphoproliferative disease | 1 |
| . | Data . |
|---|---|
| Patient characteristics | |
| Year of transplant | |
| 1998-2007 | 17 (31) |
| 2008-2017 | 38 (69) |
| Males | 51 (93) |
| Inheritance | |
| X-linked CGD | 45 (82) |
| Autosomal recessive CGD | 10 (18) |
| Median age at transplant (range), y | 5.3 (0.6-18.0) |
| Donor characteristics | |
| Type of donor | |
| Matched family donor | 20 (36) |
| Unrelated donor | 31 (56) |
| Haploidentical donor | 4 (8) |
| Stem cell source | |
| Marrow | 29 (53) |
| Peripheral blood | 24 (43) |
| Cord blood | 2 (4) |
| Stem cell dose, median (range) | |
| Marrow | |
| TNC, × 108/kg | 5.0 (1.6-24.4) |
| CD34, × 106/kg | 4.3 (0.75-53.0) |
| PB | |
| TNC, × 108/kg | 10.3 (1.2-22.8) |
| CD34, × 106/kg | 8.0 (3.0-29.2) |
| Transplant characteristics | |
| Conditioning regimen | |
| MAC | |
| Busulfan-cyclophosphamide | 21 (38) |
| Fludarabine-treosulfan-thiotepa | 4 (7) |
| RTC | |
| Treosulfan-fludarabine | 24 (45) |
| Busulfan-fludarabine | 4 (7) |
| Treosulfan-cyclophosphamide | 1 (2) |
| Fludarabine-melphalan | 1 (2) |
| Serotherapy | |
| None | 9 (16) |
| ATG | 4 (8) |
| Alemtuzumab | 42 (76) |
| GVHD prophylaxis | |
| None | 3 (5) |
| CSA alone | 2 (4) |
| CSA + MTX | 16 (29) |
| CSA + MMF | 33 (60) |
| CSA + steroid (for cord blood) | 1 (2) |
| Hematopoietic recovery | |
| Days to neutrophil recovery, median (range) | 16 (9-37) |
| Days to platelet recovery, median (range) | 18 (10-139) |
| Transplant-related complications | |
| Acute GvHD | |
| Grade II-IV | 10 (20) |
| Grade III-IV | 5 (9) |
| Chronic GvHD2 | 0 |
| Veno-occlusive disease, n (%) | 1 (2) |
| Patient receiving second procedures for decreasing chimerism | 7 (13) |
| Patients receiving unconditioned stem cell boost infusion, n | 3 |
| Median myeloid chimerism prior to unconditioned stem cell boost infusion (range), % | 17 (15-23) |
| Patients receiving fully conditioned second transplant, n | 4 |
| Median myeloid chimerism prior to fully conditioned second transplant (range), % | 11 (0-13) |
| Cause of death (n = 5), n | |
| Multiorgan failure | 2 |
| Grade IV acute GvHD | 1 |
| Pulmonary hemorrhage | 1 |
| Posttransplant lymphoproliferative disease | 1 |
Unless otherwise noted, data are n (%).
ATG, anti-thymocyte globulin (Grafalon) for haploidentical transplant; MTX, methotrexate.