TABLE I.
Patients, with cell therapies received and samples studied for TRB diversity
| Patient | Sex | Genotype | Treatment type (HCT/GT) | Time points studied (months posttreatment) | Donor type* | Conditioning regimen | CD4+ T cells/mm3 6 mo posttreatment* | CD4+ naive T cells/mm3 6 mo posttreatment* |
|---|---|---|---|---|---|---|---|---|
| 1 | F | RAG | HCT(a)† | 12, 24, 36 | MMRD | None | 183 | 0 |
| HCT(b) | ND | MMRD | MAC | 65 | 6 | |||
| 2 | F | RAG | HCT | 12, 36, 48 | URD | RIC | 576 | 65 |
| 3 | M | RAG | HCT | 24, 36, 48 | mmURD | MAC | 281 | 132 |
| 4 | M | RAG | HCT | 12, 36, 48 | MSD | None | 203 | 107 |
| 5 | F | RAG | HCT† | 6, 24, 36, 48 | MMRD | RIC | 1242 | 384 |
| 6 | F | RAG | HCT | 6, 12, 24, 36 | URD | RIC | 304 | 119 |
| 7 | M | RAG | HCT | 6, 12, 24 | URD | RIC | 912 | 511 |
| 8 | F | RAG | HCT | 6, 12 | URD | RIC | 194 | ND |
| 9 | F | DCLRE1C | HCT(a)† | 12, 24, 36, 48 | MMRD | None | 281 | 3 |
| GT(b) | ND | GT | RIC | 252 | 15 | |||
| 10 | F | DCLRE1C | HCT(a) | 3, 6, 12, 24, 36 | MMRD | RIC | 350 | 8 |
| HCT(b) | 3, 6, 24 | MMRD | RIC | 427 | 85 | |||
| 11 | F | DCLRE1C | HCT | 12 | MMRD | None | 161 | 1 |
| 12 | M | IL2RG | HCT(a)† | 12, 24, 36 | MMRD | None | 20 | 11 |
| GT(b) | 3, 6, 12 | GT | RIC | 187 | 60 | |||
| 13 | M | IL2RG | HCT(a) | 3 | MMRD | IS | Second therapy ‡ | Second therapy |
| HCT(b) | 6, 24 | mmURD | RIC | 570 | ND | |||
| 14 | M | IL2RG | HCT | 12, 36, 48 | MMRD | None | 627 | 74 |
| 15 | M | IL2RG | GT | 3 | GT | RIC | 1949 | 779 |
| 16 | M | IL2RG | HCT | 3, 6, 48 | URD | IS | >500 | 375 |
| 17 | M | IL2RG | HCT | 6, 12, 24, 36, 48 | MMRD | IS | 1417 | 879 |
| 18 | M | IL2RG | HCT | 3, 6, 12 | MMRD | IS | 920 | 626 |
| 19 | M | IL2RG | GT | 3, 6, 12 | GT | RIC | 1050 | 726 |
| 20 | M | IL2RG | GT | 3, 6 | GT | RIC | 2039 | 1305 |
| 21 | M | IL2RG | HCT† | 3, 12, 24, 36, 48 | MMRD | None | 289 | 87 |
| 22 | F | IL7R | HCT | 24 | URD | IS | 203 | 3 |
| 23 | F | IL7R | HCT | 6, 12, 24, 48 | MMRD | None | 1086 | 478 |
| 24 | F | IL7R | HCT (Boost) § | 3, 6, 12 | MMRD | None | 2291 | 226 |
| 25 | F | IL7R | HCT | 6, 12, 24, 36 | URD | IS | 217 | 91 |
| 26 | F | JAK3 | HCT | 3, 6 | MMRD | IS | 1296 | ND |
| 27 | M | BCL11B | HCT(a) | 3, 6 | URD | IS | Second therapy ‡ | Second therapy |
| HCT(b) | 36 | URD | RIC | 1120 | 44 |
Multiple cell therapies are indicated by letters following the treatment type.
F, Female; IS, immune suppression; M, male; MAC, myeloablative conditioning; MMRD, mismatched related donor; mmURD, mismatched unrelated donor; MSD, matched sibling donor; ND, not done; RIC, reduced-intensity conditioning; URD, unrelated (HLA-matched) donor.
*
Lower end of 10%–90% of healthy children aged 1–2 y 1300/mm3 for CD4; 950 for CD4/CD45RA/CD62L.11
†
Autoimmune complications.
‡
Value not available if second treatment occurred within 6 mo of initial treatment.
§
Boost, additional cells from previous donor source administered with no conditioning between 3 and 6 mo post-HCT.