HSCT and outcomes
| Patient . | Age at HSCT, y . | Conditioning regimen . | Donor . | Amount of CD34+ cells, × 106 cells/kg . | T-cell depletion . | Engraftment . | Acute GVHD . | Chronic GVHD . | Other complications . | Infections . | Follow-up after HSCT, days . | Outcome/clinical status . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 (UPN 389) | 0.5 (1st HSCT) | Bu (by mouth), Cy, anti-LFA1, anti-CD2 | URD 10/10 | nd | + | No | 0 | HLH | ||||
| 1 (UPN 389) | 0.8 (2nd HSCT) | ATG, Bu (by mouth), Cy anti-LFA1, anti-CD2 | URD 10/10 | 10.4 | + | Yes | III | Extensive | 4343 (12 years) | Alive, impaired cognitive abilities | ||
| 2 (UPN 483) | 2.3 | ATG, Bu (by mouth), Cy | HLA id | 2.5 | − | Yes | 0 | 0 | 3414 (9 years) | Alive, well | ||
| 3 (UPN 533) | 6.8 | ATG, Bu (by mouth), Cy | HLA id | 3.4 | − | Yes | II | 0 | VOD, AH, DIC | Adenovirus, CMV, bacterial sepsis | 2894 (8 years) | Alive, impaired cognitive abilities, necrosis of femoral head |
| 4 (UPN 556) | 11.5 | ATG, Bu, Cy | HLA id | 5.4 | − | Yes | I | NA | VOD, CLS | 99 | Death (ARDS) | |
| 5 (UPN 643) | 7.2 | ATG, Bu, Cy | URD 10/10 | 5.4 | − | Yes | II | 0 | LPD, pancreatitis | VZV, EBV, Aspergillus nidulans pneumonia | 1585 (4 years) | Alive, well |
| 6 (UPN 635) | 1.0 | ATG, Bu, Cy | Phenoid parent | 1.5 | − | Yes | 0 | NA | VOD, LPD, HLH | EBV | 105 | Death (LPD, HLH) |
| 7 (UPN 688) | 7.4 | ATG, Bu, Cy | URD 9/10 | 2.5 | − | Yes | III | NA | HC (BK-virus), AH | Adenovirus, CMV, Aspergillus, HSV, BK-virus | 110 | Death (ARDS) |
| 8 (UPN 716) | 2.9 | ATG, Bu, Cy | URD 9/10 | 5.8 | + | Yes | II | 0 | VOD, CLS | Adenovirus | 651 (20 months) | Alive, epilepsy |
| 9 (UPN728) | 5.3 | ATG, Bu, Cy | Haploid | 3.0 | + | Yes | II | 0 | VOD | Parvovirus B19, VZV, Bacterial sepsis | 519 (17 months) | Alive, well |
| 10 (UPN 744) | 4.3 | ATG, Bu, Cy | Haploid | 7.3 | + | Yes | II | 0 | Severe mucositis | Bacterial sepsis | 309 (10 months) | Alive, well |
| Patient . | Age at HSCT, y . | Conditioning regimen . | Donor . | Amount of CD34+ cells, × 106 cells/kg . | T-cell depletion . | Engraftment . | Acute GVHD . | Chronic GVHD . | Other complications . | Infections . | Follow-up after HSCT, days . | Outcome/clinical status . |
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 (UPN 389) | 0.5 (1st HSCT) | Bu (by mouth), Cy, anti-LFA1, anti-CD2 | URD 10/10 | nd | + | No | 0 | HLH | ||||
| 1 (UPN 389) | 0.8 (2nd HSCT) | ATG, Bu (by mouth), Cy anti-LFA1, anti-CD2 | URD 10/10 | 10.4 | + | Yes | III | Extensive | 4343 (12 years) | Alive, impaired cognitive abilities | ||
| 2 (UPN 483) | 2.3 | ATG, Bu (by mouth), Cy | HLA id | 2.5 | − | Yes | 0 | 0 | 3414 (9 years) | Alive, well | ||
| 3 (UPN 533) | 6.8 | ATG, Bu (by mouth), Cy | HLA id | 3.4 | − | Yes | II | 0 | VOD, AH, DIC | Adenovirus, CMV, bacterial sepsis | 2894 (8 years) | Alive, impaired cognitive abilities, necrosis of femoral head |
| 4 (UPN 556) | 11.5 | ATG, Bu, Cy | HLA id | 5.4 | − | Yes | I | NA | VOD, CLS | 99 | Death (ARDS) | |
| 5 (UPN 643) | 7.2 | ATG, Bu, Cy | URD 10/10 | 5.4 | − | Yes | II | 0 | LPD, pancreatitis | VZV, EBV, Aspergillus nidulans pneumonia | 1585 (4 years) | Alive, well |
| 6 (UPN 635) | 1.0 | ATG, Bu, Cy | Phenoid parent | 1.5 | − | Yes | 0 | NA | VOD, LPD, HLH | EBV | 105 | Death (LPD, HLH) |
| 7 (UPN 688) | 7.4 | ATG, Bu, Cy | URD 9/10 | 2.5 | − | Yes | III | NA | HC (BK-virus), AH | Adenovirus, CMV, Aspergillus, HSV, BK-virus | 110 | Death (ARDS) |
| 8 (UPN 716) | 2.9 | ATG, Bu, Cy | URD 9/10 | 5.8 | + | Yes | II | 0 | VOD, CLS | Adenovirus | 651 (20 months) | Alive, epilepsy |
| 9 (UPN728) | 5.3 | ATG, Bu, Cy | Haploid | 3.0 | + | Yes | II | 0 | VOD | Parvovirus B19, VZV, Bacterial sepsis | 519 (17 months) | Alive, well |
| 10 (UPN 744) | 4.3 | ATG, Bu, Cy | Haploid | 7.3 | + | Yes | II | 0 | Severe mucositis | Bacterial sepsis | 309 (10 months) | Alive, well |
AH indicates alveolar hemorrhage; ARDS, acute respiratory stress syndrome; ATG, anti-thymoglobulin; Bu, busulfan; CLS, capillary leak syndrome; CMV, cytomegalovirus; Cy, cyclophosphamide; DIC, disseminated intravascular coagulation; EBV, Epstein-Barr virus; GVHD, graft-versus-host disease; HC, hemorrhagic cystitis; HSV, herpes simplex virus; LPD, lymphoproliferative disease; NA, not applicable; nd, not determined; URD, matched unrelated donor; VOD, veno-occlusive disease; and VZV, varicella zoster virus.