Patients with ADA SCID: treatment parameters and outcomes
| UPN . | Age at GT (mo)* . | CD34+ cells (×106/kg)* . | VCN* . | Bu AUC (μM × min)* . | Follow-up (y after GT) . | ERT status . | IgRT status . |
|---|---|---|---|---|---|---|---|
| 401 | 184 | 0.6 | 0.6 | Not measured | 11 | Resumed ERT 6.5 mo post-GT | On |
| 402 | 4 | 1.7 | 0.71 | 5437 | 11 | Off ERT since GT | On |
| 403 | 97 | 0.92 | 0.185 | 3871 | 11 | Off ERT since GT | On |
| 404 | 3 | 7.1 | 1.96 | 3532 | 10 | Off ERT since GT | Off |
| 405 | 8 | 7.62 | 2 | 5469 | 9 | Off ERT since GT | Off |
| 406 | 17 | 8 | 2.6 | 2427 | 9 | Off ERT since GT | On |
| 407 | 14 | 4, 1.6 | 1.0, 2.3† | 3232 | 9 | Off ERT since GT | On |
| 408 | 3 | 6.85 | 2.68 | 5344 | 8 | Off ERT since GT | Off |
| 409 | 20 | 2.86 | 1.22 | 5608 | 8 | Off ERT since GT | On |
| 410 | 3 | 8.41 | 2.38 | 6714 | 8 | Off ERT since GT | Off |
| UPN . | Age at GT (mo)* . | CD34+ cells (×106/kg)* . | VCN* . | Bu AUC (μM × min)* . | Follow-up (y after GT) . | ERT status . | IgRT status . |
|---|---|---|---|---|---|---|---|
| 401 | 184 | 0.6 | 0.6 | Not measured | 11 | Resumed ERT 6.5 mo post-GT | On |
| 402 | 4 | 1.7 | 0.71 | 5437 | 11 | Off ERT since GT | On |
| 403 | 97 | 0.92 | 0.185 | 3871 | 11 | Off ERT since GT | On |
| 404 | 3 | 7.1 | 1.96 | 3532 | 10 | Off ERT since GT | Off |
| 405 | 8 | 7.62 | 2 | 5469 | 9 | Off ERT since GT | Off |
| 406 | 17 | 8 | 2.6 | 2427 | 9 | Off ERT since GT | On |
| 407 | 14 | 4, 1.6 | 1.0, 2.3† | 3232 | 9 | Off ERT since GT | On |
| 408 | 3 | 6.85 | 2.68 | 5344 | 8 | Off ERT since GT | Off |
| 409 | 20 | 2.86 | 1.22 | 5608 | 8 | Off ERT since GT | On |
| 410 | 3 | 8.41 | 2.38 | 6714 | 8 | Off ERT since GT | Off |
Data for age at gene therapy, CD34+ cell dose, cell product vector copy number, and busulfan area-under-the-curve are from Shaw et al. (ADA gene mutations for these patients are listed in Table 1).13
Because of a manufacturing deviation, subject 407 received 2 separate cell products.
Bu AUC, busulfan area-under-the-curve; UPN, unique patient number.