Table 3.
Outcomes in the Japanese patients with XIAP deficiency post-HSCT
| Patient | ID | Engraftment (days) | Chimerism | Virus reactivation | Adverse event | IBD post-HSCT | HLH post-HSCT | Outcome | Months after HSCT |
|---|---|---|---|---|---|---|---|---|---|
| 1 | 2.1 | 22 | N/A | – | ARDS | – | + | Dead | 27 days |
| 2 | 8 | 16 | 98.8% | – | Catheter infection (Staphylocuccus epidermidis) | – | – | PS0 | 45 months |
| 3 | 1 | 18 | >95% | – | ATG-anaphylaxis | – | + (Eto, DP) | PS0 | 36 months |
| 4 | 11 | 23 | 100% | – | Adrenal failure | – | – | PS0 | 25 months |
| 5 | 13 | 11 | 98.6% | HHV6-encephalitis, BK virus cystitis | – | – | + (Eto, PSL) | PS1 with mechanical ileus | 23 months |
| 6 | 5.2 | 26 | 100% | BK viremia JC viremia | Sepsis (Escherichia coli) | – | + (Eto, DP) | PS0 | 19 months |
| 7 | 2.3 | 20 | >95% | – | TMA, PAH | – | – | PS1 with TMA | 14 months |
| 8 | 14 | 26 | 100% | CMV-emia | – | – | – | PS1 with LPD | 12 months |
| 9 | 15.2 | 22 | 99.7% | – | MOF | – | + (Eto, DP) | PS0 | 12 months |
| 10 | 18 | 19 | 83% | BK virus cystitis, CMV-emia | Enteritis (Enterobacter) | – | – | PS1 | 5 months |
HSCT hematopoietic stem cell transplantation, HLH hemophagocytic lymphohistiocytosis, N/A not available, ARDS acute respiratory distress syndrome, ATG anti-thymocyte globulin, Eto etoposide, DP dexamethasone palmitate, HHV6 human herpesvirus 6, TMA thrombotic microangiopathy, PAH pulmonary artery hypertension, CMV cytomegalovirus, LPD lymphoproliferative disease, MOF multiple organ failure, DEX dexamethasone, PSL prednisolone, PS performance status