Table 3:
Treatment characteristics and outcome of Massachusetts infants with SCID
| Case # | Dx | Gene | Age at treatment (months) | Infectious Status at treatment | Donor | Conditioning | Years post | Status | Ig replacement status |
|---|---|---|---|---|---|---|---|---|---|
| 594 | SCID | JAK3 | 2.5 | None | Unrelated HCT | MAC bu/cy | 9.3 | Alive | Off |
| 935 | SCID | TTC7A | 3.4 | None | Sibling HCT | ATG | 8.3 | Alive | On (post multivisceral transplant) |
| 1015 | SCID | IL2RG | 2.4 | None | Unrelated HCT | MAC bu/flu/ATG | 8.2 | Alive | Off |
| 1050 | SCID | CD3D | 2.4 | None | Unrelated HCT | MAC bu/flu/ATG | 8.1 | Alive | Off |
| 2053 | leaky SCID | JAK3 | 4.6 | None | Unrelated HCT | MAC bu/flu/ATG | 5.4 | Alive | Off |
| 3023 | SCID | ADA | 1.4 | Aspergillus pneumonia, controlled | Sibling HCT | none | 3.4 | Alive | Off |
| 3840 | SCID | IL2RG | 5.4 | None | Autologous Gene Therapy | RIC bu | 1.8 | Alive | Off |
| 4435 | leaky SCID | IL7R | 4.2 | CMV viremia, active, pertussis treated | Unrelated HCT | Campath | 1.0 | Alive | On |
| 4580 | SCID | RAG1 | 4.0 | None | Paternal haploidentical HCT | RIC bu/flu/thio/ATG | 0.8 | Alive | Off |
(note: calculated to 1y post end of 10y screening period i.e. Jan 31, 2020)