Table 1.
Patient characteristics
| Patient no. |
Age at GT (y) | Mutation (protein effect) | WASp expression (flow cytometry) | Revertant cells | WAS clinical score |
Platelet count (×103 cells per μL) |
Cell product |
Bu AUC (mg × h/L) | F/U (y) | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Without romi-plostim | With romi-plostim | Source of CD34+ cells | CD34+ dose (× 106/kg) | CD34+ bulk VCN (copies per cell) | Positive CFUs (%) | Positive CFU VCN (mean) | Transduced CD34+ (× 106/kg) | ||||||||
| 1 | 1.8 | Intron 3 -2A>G |
↓↓↓ | − | 3 | 24-29 | MPB | 24.91 | 3.37 | 97 | 2.35 | 24.16 | 81.2 | 8.8 | |
| 2 | 3.6 | Exon 1 c.35delG (p.Gly12Alafs33) | Null | + CD8+ (52%) DNT (72%) NK (69%) |
4 | <10 | 15-20 | MPB | 9.34 | 1.34 | 94 | 1.3 | 8.78 | 48.8 | 8.3 |
| 3 | 1.4 | Exon 2 c.256C>T (p.Arg86Cys) | ↓↓↓ | − | 3 | 10-15 | 24-66 | MPB | 9.8 | 0.54 | 51 | 0.93 | 5.00 | 77.2 | 7.6 |
| 4 | 8 | Exon 2 c.224T>C (p.Trp64Arg) | Null | + CD4+ (9%) CD8+ (10%) DNT (4%) |
5 | 19-35 | MPB ×2 and BM | 6.32 | 1.01∗ | 28-74 | 1.81∗ | 3.06 | 84.5 | 7.5 | |
| MPB† | 2.52 | 0.25 | 28 | 1.13 | 0.71 | ||||||||||
| MPB† | 2.29 | 0.78 | 54 | 1.3 | 1.24 | ||||||||||
| BM | 1.51 | 2.61 | 74 | 2.8 | 1.12 | ||||||||||
| 5 | 1.4 | Exon 1 c.91G>A (p.Glu31Lys) | Null | − | 5 | 12-59 | MPB | 15.06 | 1.49 | 69 | 2.45 | 10.39 | 69.0 | 5.3 | |
Italics indicate the characteristics of the 3 different manufactured products that patient 4 received to distinguish from the the estimate of the total MPs infused. AUC, area under the curve; BM, bone marrow; Bu, busulfan; DNT, double-negative T cells; F/U, follow-up; MPB, mobilized peripheral blood; NK, natural killer cell.
∗
Weighed average of multiple products.
†
Products were each transduced separately, frozen, then thawed and infused together with the fresh BM product.