Overview of HSCT in the cohort of Colombian CGD patients
| Pt | Age at HSCT (months) | Transplant source and match | Conditioning | Regimen | Graft failure | a-GVHD | c-GVHD | Survival | Complications and follow-up after HSCT |
|---|---|---|---|---|---|---|---|---|---|
| P1 | 173 | UCB 5/6 | RIC | Busulfan/cyclophosphamide | No | II | II | Yes | Alive 11 years after HSCT |
| P2 | 32 | UCB 5/6 | RIC | Busulfan/fludarabine | No | No | No | Yes | Alive 8 years after HSCT |
| P3 | 56 | UCB 5/6 | RIC | Busulfan/cyclophosphamide | No | III | No | Yes | Alive 6 years after HSCT |
| P7 | 11 | UCB 5/6 | MAC | Busulfan/fludarabine | No | No | No | No | Died after HSCT due to GI infection, graft failure |
| P9 | 16 | Haplo-PtCy | MAC | Busulfan/fludarabine/cyclophosphamide/TBI | Yes | I | I | Yes | Alive 9 years after HSCT. Thyroid tumor after HSCT |
| P10 | 5 | Haplo-PtCy | MAC | Busulfan/fludarabine/cyclophosphamide/TBI | No | No | III | No | Alveolar hemorrhage. Died due to severe GVHD and sepsis, BCG-osis |
| P16 | 6 | Haplo-PtCy | MAC | Busulfan/fludarabine | Yes | NE | NE | NE | Graft failure, underwent second HSCT |
| 7 | Haplo-PtCy | MAC | Busulfan/fludarabine/cyclophosphamide/TBI | No | No | No | Yes | Alive 9 years after HSCT | |
| P18 | 71 | Haplo-PtCy | RIC | Busulfan/fludarabine | NE | NE | NE | No | Early death after HSCT, multiorgan dysfunction |
| P22 | 11 | Haplo-PtCy | RIC | Busulfan/fludarabine | No | No | No | Yes | Alive 11 mo after HSCT |
| P23 | 20 | UCB 5/6 | RIC | Busulfan/fludarabine | No | IV | II | No | Died due to severe GVHD and infection |
| P24 | 34 | UCB 5/6 | RIC | Busulfan/fludarabine | No | IV | III | No | Died due to lymphoproliferative disorder 3 years after HSCT, Burkitt lymphoma EBER-positive |
| P25 | 7 | Haplo-PtCy | MAC | Busulfan/fludarabine/melphalan | No | II | I | Yes | Laryngomalacia and tracheal granuloma. Alive 21 mo after HSCT |
| P26 | 24 | Haplo-PtCy | RIC | Busulfan/fludarabine | Yes | NE | NE | NE | Graft failure, underwent second HSCT |
| 36 | Haplo-PtCy | RIC | Busulfan/cyclophosphamide | No | III | II | Yes | Pseudomonas mendocina bacteremia. Probable lung aspergillosis. Clostridium difficile gastrointestinal infection. Gastrointestinal bleeding. Alive 1.7 years after HSCT | |
| P30 | 14 | UCB 5/6 | RIC | Busulfan/fludarabine | No | IV | NE | No | Died due to severe GVHD |
| P32 | 132 | UCB 5/6 | RIC | Busulfan/fludarabine | Yes | NE | NE | Yes | Graft failure. Alive with prophylaxis |
| P33 | 75 | Haplo-PtCy | RIC | Busulfan/fludarabine/melphalan | Yes | NE | NE | NE | Graft failure, underwent second HSCT |
| 90 | Haplo-PtCy | MAC | Busulfan/fludarabine | Yes | NE | NE | No | Died after HSCT due to invasive aspergillosis and multiorgan failure | |
| P35 | 96 | Matched sibling donor | RIC | Fludarabine/cyclophosphamide/TBI | No | No | No | Yes | Alive after HSCT |
| Pt | Age at HSCT (months) | Transplant source and match | Conditioning | Regimen | Graft failure | a-GVHD | c-GVHD | Survival | Complications and follow-up after HSCT |
|---|---|---|---|---|---|---|---|---|---|
| P1 | 173 | UCB 5/6 | RIC | Busulfan/cyclophosphamide | No | II | II | Yes | Alive 11 years after HSCT |
| P2 | 32 | UCB 5/6 | RIC | Busulfan/fludarabine | No | No | No | Yes | Alive 8 years after HSCT |
| P3 | 56 | UCB 5/6 | RIC | Busulfan/cyclophosphamide | No | III | No | Yes | Alive 6 years after HSCT |
| P7 | 11 | UCB 5/6 | MAC | Busulfan/fludarabine | No | No | No | No | Died after HSCT due to GI infection, graft failure |
| P9 | 16 | Haplo-PtCy | MAC | Busulfan/fludarabine/cyclophosphamide/TBI | Yes | I | I | Yes | Alive 9 years after HSCT. Thyroid tumor after HSCT |
| P10 | 5 | Haplo-PtCy | MAC | Busulfan/fludarabine/cyclophosphamide/TBI | No | No | III | No | Alveolar hemorrhage. Died due to severe GVHD and sepsis, BCG-osis |
| P16 | 6 | Haplo-PtCy | MAC | Busulfan/fludarabine | Yes | NE | NE | NE | Graft failure, underwent second HSCT |
| 7 | Haplo-PtCy | MAC | Busulfan/fludarabine/cyclophosphamide/TBI | No | No | No | Yes | Alive 9 years after HSCT | |
| P18 | 71 | Haplo-PtCy | RIC | Busulfan/fludarabine | NE | NE | NE | No | Early death after HSCT, multiorgan dysfunction |
| P22 | 11 | Haplo-PtCy | RIC | Busulfan/fludarabine | No | No | No | Yes | Alive 11 mo after HSCT |
| P23 | 20 | UCB 5/6 | RIC | Busulfan/fludarabine | No | IV | II | No | Died due to severe GVHD and infection |
| P24 | 34 | UCB 5/6 | RIC | Busulfan/fludarabine | No | IV | III | No | Died due to lymphoproliferative disorder 3 years after HSCT, Burkitt lymphoma EBER-positive |
| P25 | 7 | Haplo-PtCy | MAC | Busulfan/fludarabine/melphalan | No | II | I | Yes | Laryngomalacia and tracheal granuloma. Alive 21 mo after HSCT |
| P26 | 24 | Haplo-PtCy | RIC | Busulfan/fludarabine | Yes | NE | NE | NE | Graft failure, underwent second HSCT |
| 36 | Haplo-PtCy | RIC | Busulfan/cyclophosphamide | No | III | II | Yes | ||
| P30 | 14 | UCB 5/6 | RIC | Busulfan/fludarabine | No | IV | NE | No | Died due to severe GVHD |
| P32 | 132 | UCB 5/6 | RIC | Busulfan/fludarabine | Yes | NE | NE | Yes | Graft failure. Alive with prophylaxis |
| P33 | 75 | Haplo-PtCy | RIC | Busulfan/fludarabine/melphalan | Yes | NE | NE | NE | Graft failure, underwent second HSCT |
| 90 | Haplo-PtCy | MAC | Busulfan/fludarabine | Yes | NE | NE | No | Died after HSCT due to invasive aspergillosis and multiorgan failure | |
| P35 | 96 | Matched sibling donor | RIC | Fludarabine/cyclophosphamide/TBI | No | No | No | Yes | Alive after HSCT |
a-GVHD, acute graft vs. host disease; c-GVHD, chronic graft vs. host disease; EBER, Epstein-Barr virus-encoded small RNAs; GI, gastrointestinal; Haplo-PtCy, haploidentical hematopoietic stem cell transplantation with posttransplant cyclophosphamide; MAC, myeloablative conditioning; NE, not evaluated; Pt, patient; RIC, reduced intensity conditioning; TBI, total-body irradiation.