Details of definitive therapy for Wiskott-Aldrich syndrome in patients
| Patient no. . | DT . | Declining donor chimerism after HSCT (% donor) . | IMs occurrence after DT? (% donor myeloid cells at IMs onset) . | 2nd HSCT . | Current status and FU duration after last DT . |
|---|---|---|---|---|---|
| 1 | HSCT (twice) | Yes, after 1st HSCT (CD3, 85%; CD14, 1%) | Yes, recurrence (CD14, 1%) | Yes | CR after 2nd HSCT, full donor chimerism (3 y FU) |
| 2 | HSCT (twice) | Yes, after 1st HSCT (CD3, 90%; CD15, 0%) | Yes, 1st appearance (CD15, 0%) | Yes | CR after 2nd HSCT, full donor chimerism (4 y FU) |
| 3 | HSCT | Yes, after HSCT (CD3, 80%; CD14, 30%) | Yes, 1st appearance (CD14, 30%) | No | IMs well-controlled on canakinumab |
| 4 | HSCT | No | No | No | CR after HSCT, full donor chimerism (3 y FU) |
| 5 | HSCT | Yes, after HSCT (CD3, 93%; CD15, 15%) | Yes, recurrence (CD15, 40%) | No | IMs well-controlled on canakinumab |
| 6 | GT | NA | No | No | CR (5 y FU) |
| 7 | GT | NA | No | No | CR (4.4 y FU) |
| 8 | GT | NA | No | No | CR (4.1 y FU) |
| 9 | GT | NA | No | No | CR (8 y FU) |
| Patient no. . | DT . | Declining donor chimerism after HSCT (% donor) . | IMs occurrence after DT? (% donor myeloid cells at IMs onset) . | 2nd HSCT . | Current status and FU duration after last DT . |
|---|---|---|---|---|---|
| 1 | HSCT (twice) | Yes, after 1st HSCT (CD3, 85%; CD14, 1%) | Yes, recurrence (CD14, 1%) | Yes | CR after 2nd HSCT, full donor chimerism (3 y FU) |
| 2 | HSCT (twice) | Yes, after 1st HSCT (CD3, 90%; CD15, 0%) | Yes, 1st appearance (CD15, 0%) | Yes | CR after 2nd HSCT, full donor chimerism (4 y FU) |
| 3 | HSCT | Yes, after HSCT (CD3, 80%; CD14, 30%) | Yes, 1st appearance (CD14, 30%) | No | IMs well-controlled on canakinumab |
| 4 | HSCT | No | No | No | CR after HSCT, full donor chimerism (3 y FU) |
| 5 | HSCT | Yes, after HSCT (CD3, 93%; CD15, 15%) | Yes, recurrence (CD15, 40%) | No | IMs well-controlled on canakinumab |
| 6 | GT | NA | No | No | CR (5 y FU) |
| 7 | GT | NA | No | No | CR (4.4 y FU) |
| 8 | GT | NA | No | No | CR (4.1 y FU) |
| 9 | GT | NA | No | No | CR (8 y FU) |
Abbreviations: CR, complete remission; FU, follow-up; GT, gene therapy; NA, not applicable.