Characteristics of 8 patients treated with beti-cel
| Patient . | 1 . | 2 . | 3 . | 4 . | 5 . | 6 . | 7 . | 8 . |
|---|---|---|---|---|---|---|---|---|
| Age at the time of beti-cel infusion, y | 15 | 29 | 18 | 17 | 14 | 22 | 26 | 13 |
| Sex | Male | Male | Male | Female | Female | Male | Female | Male |
| Country of origin | Ukraine | Turkey | Syria | Afghanistan | Lebanon | Syria | Thailand | Lebanon |
| Functional genotype | β°/β⁺ | β°/β⁺ | β⁺/β⁺ | β⁺/β⁺ | β⁺/β⁺ | β°/β⁺ | β°/β+E | β⁺/β⁺ |
| Molecular genotype (HGVS nomenclature) | HBB:c.92+1G>A/HBB:c.92+6T>C | HBB:c.92+1G>A/HBB:c.92+6T>C | HBB:c.93–21G>A/HBB:c.93–21G>A | HBB:c.92+5G>C/HBB:c.92+5G>C | HBB:c.93–21G>A/HBB:c.92+6T>C | HBB:c.92+1G>A/HBB:c.92+6T>C | HBB:c.27_28insAGAA/HBB:c.79G>A | HBB:c.92+6T>C/HBB:c.93–21G>A |
| Age at initiation of regular RBC transfusions, y | 9.8 | 2 | 1.5 | 0.6 | 1.5 | 1 | 11 | 3 |
| Average no. of RBC transfusions per year over last 2 years | 17.3 | 20.8 | 17.3 | 17.3 | 13.0 | 26.0 | 17.3 | 12.0 |
| Deferasirox, mg/kg per day | 28.2 | 22.0 | 15.0 | 23.0 | 12.5 | 21.1 | 14.9 | |
| Deferiprone, mg/kg per day | 56.9 | 76.0 | ||||||
| Splenectomy | No | Yes | Yes | No | No | No | No | No |
| LIC, mg/g dw | 2 | 0.7 | 6.9 | 3.5 | 7.9 | 7 | 2.3 | 1.9 |
| Cardiac T2∗, ms | 35 | 38 | 37 | 25 | 44 | 37 | 41 | >20 |
| Serum ferritin, μg/L | 1059 | 282 | 1338 | 967 | 2342 | 1315 | 392 | 1268 |
| Spleen volume, mL | 801 | Post splenectomy | Post splenectomy | 534 | 547 | 691 | 784 | 623 |
| Patient . | 1 . | 2 . | 3 . | 4 . | 5 . | 6 . | 7 . | 8 . |
|---|---|---|---|---|---|---|---|---|
| Age at the time of beti-cel infusion, y | 15 | 29 | 18 | 17 | 14 | 22 | 26 | 13 |
| Sex | Male | Male | Male | Female | Female | Male | Female | Male |
| Country of origin | Ukraine | Turkey | Syria | Afghanistan | Lebanon | Syria | Thailand | Lebanon |
| Functional genotype | β°/β⁺ | β°/β⁺ | β⁺/β⁺ | β⁺/β⁺ | β⁺/β⁺ | β°/β⁺ | β°/β+E | β⁺/β⁺ |
| Molecular genotype (HGVS nomenclature) | HBB:c.92+1G>A/HBB:c.92+6T>C | HBB:c.92+1G>A/HBB:c.92+6T>C | HBB:c.93–21G>A/HBB:c.93–21G>A | HBB:c.92+5G>C/HBB:c.92+5G>C | HBB:c.93–21G>A/HBB:c.92+6T>C | HBB:c.92+1G>A/HBB:c.92+6T>C | HBB:c.27_28insAGAA/HBB:c.79G>A | HBB:c.92+6T>C/HBB:c.93–21G>A |
| Age at initiation of regular RBC transfusions, y | 9.8 | 2 | 1.5 | 0.6 | 1.5 | 1 | 11 | 3 |
| Average no. of RBC transfusions per year over last 2 years | 17.3 | 20.8 | 17.3 | 17.3 | 13.0 | 26.0 | 17.3 | 12.0 |
| Deferasirox, mg/kg per day | 28.2 | 22.0 | 15.0 | 23.0 | 12.5 | 21.1 | 14.9 | |
| Deferiprone, mg/kg per day | 56.9 | 76.0 | ||||||
| Splenectomy | No | Yes | Yes | No | No | No | No | No |
| LIC, mg/g dw | 2 | 0.7 | 6.9 | 3.5 | 7.9 | 7 | 2.3 | 1.9 |
| Cardiac T2∗, ms | 35 | 38 | 37 | 25 | 44 | 37 | 41 | >20 |
| Serum ferritin, μg/L | 1059 | 282 | 1338 | 967 | 2342 | 1315 | 392 | 1268 |
| Spleen volume, mL | 801 | Post splenectomy | Post splenectomy | 534 | 547 | 691 | 784 | 623 |
HGVS, Human Genome Variation Society.