Analysis of survival outcome in proband and sibling cohorts following different conditioning regimes, donor sources, and molecular diagnosis
| . | Proband cohort . | Mortality . | Sibling cohort . | Mortality . | Significance (P) . |
|---|---|---|---|---|---|
| Conditioning | |||||
| Unconditioned | 11 | 5 | 20 | 2 | < .05* |
| RIC | 7 | 2 | 20 | 0 | < .05* |
| Myeloablative | 12 | 5 | 18 | 3 | NS |
| Donor | |||||
| MSD | 6 | 0 | 11 | 1 | NS |
| MFD | 6 | 4 | 8 | 0 | < .05* |
| MUD | 2 | 0 | 5 | 1 | NS |
| mMUD | 1 | 0 | 3 | 0 | NS |
| Cord | 0 | 0 | 6 | 0 | NS |
| Haplo | 16 | 8 | 24 | 3 | < .01* |
| GT-autologous | 0 | 0 | 1 | 0 | NS |
| Genetic defect | |||||
| γ-c | 10 | 6 | 14 | 1 | < .01* |
| JAK3 | 3 | 2 | 3 | 1 | NS |
| IL-7RA | 1 | 0 | 3 | 1 | NS |
| RAG1/2 | 6 | 5 | 10 | 1 | < .01* |
| Artemis | 2 | 1 | 4 | 0 | NS |
| Omenn | 2 | 2 | 1 | 0 | NS |
| ADA | 8 | 5 | 8 | 1 | < .05* |
| Undefined | 16 | 8 | 17 | 1 | < .05* |
| . | Proband cohort . | Mortality . | Sibling cohort . | Mortality . | Significance (P) . |
|---|---|---|---|---|---|
| Conditioning | |||||
| Unconditioned | 11 | 5 | 20 | 2 | < .05* |
| RIC | 7 | 2 | 20 | 0 | < .05* |
| Myeloablative | 12 | 5 | 18 | 3 | NS |
| Donor | |||||
| MSD | 6 | 0 | 11 | 1 | NS |
| MFD | 6 | 4 | 8 | 0 | < .05* |
| MUD | 2 | 0 | 5 | 1 | NS |
| mMUD | 1 | 0 | 3 | 0 | NS |
| Cord | 0 | 0 | 6 | 0 | NS |
| Haplo | 16 | 8 | 24 | 3 | < .01* |
| GT-autologous | 0 | 0 | 1 | 0 | NS |
| Genetic defect | |||||
| γ-c | 10 | 6 | 14 | 1 | < .01* |
| JAK3 | 3 | 2 | 3 | 1 | NS |
| IL-7RA | 1 | 0 | 3 | 1 | NS |
| RAG1/2 | 6 | 5 | 10 | 1 | < .01* |
| Artemis | 2 | 1 | 4 | 0 | NS |
| Omenn | 2 | 2 | 1 | 0 | NS |
| ADA | 8 | 5 | 8 | 1 | < .05* |
| Undefined | 16 | 8 | 17 | 1 | < .05* |
RIC indicates reduced-intensity conditioning; MSD, matched sibling donor; MFD, matched family donor; MUD, matched unrelated donor; mMUD, mismatched unrelated donor; Cord, cord blood; Haplo, haploidentical; GT, gene therapy; ADA, adenosine deaminase; and NS, no statistical difference.
Statistically significant difference.