Table 1.
Hematologic dataa
| Treatment | Patient/cohort | Number of specimens/individuals | Age interval | Time since infusion/HU treatment | Hb (g/dL) | MCH (pg) | MCHC (g/dL) | MCV (fL) | HbF (%) | F-cell (%) |
|---|---|---|---|---|---|---|---|---|---|---|
| n | Years | Months | ||||||||
| Gene therapy | 2 | 7 | 21–25 | 49 | 10.9 (10.4,11.5) | 31.2 (30.9,31.8) | 35.0 (34.2,36.1) | 89.1 (88.3,91.6) | 23.3 (20.8,26.0) | 67.5 (59.7,76.5) |
| Gene therapy | 3 | 10 | 26–30 | 39 | 9.9 (9.1,11.1) | 30.0 (26.7,31.8) | 32.8 (32.3,35.7) | 89.2 (81.4,92.5) | 23.4 (16.5,30.1) | 62.6 (48.2,72.7) |
| Gene therapy | 4 | 9 | 21–25 | 40 | 11.1 (10.0,11.9) | 31.0 (30.2,32.2) | 36.4 (35.1,36.4) | 85.5 (84.2,88.3) | 28.9 (27.7,30.9) | 75.3 (70.2,81.9) |
| Gene therapy | 6b | 7 | 16–20 | 36 | 10.6 (10.5,11.1) | 25.8 (25.5,26.6) | 33.8 (32.2,34.1) | 77.8 (74.9,82.4) | 38.6 (36.1,40.1) | 71.8 (68.4,74.8) |
| Gene therapy | 7 | 8 | 11–15 | 32 | 11.8 (11.3,12.4) | 30.0 (30.0,30.1) | 36.1 (35.6,36.3) | 83.0 (82.8,84.4) | 25.5 (25.4,26.5) | 64.0 (63.1,67.0) |
| Gene therapy | 8 | 6 | 6–10 | 27 | 9.4 (9.0, 9.7) | 32.2 (31.3,32.7) | 36.5 (35.3,37.2) | 88.5 (86.9,95.8) | 37.8 (31.1,42.2) | 86.1 (80.8,91.1) |
| Gene therapy | 9 | 6 | 11–15 | 22 | 11.1 (9.6,11.4) | 38.4 (36.9,39.2) | 35.0 (34.8,35.5) | 109.8 (106.2,111.9) | 27.9 (25.3,28.9) | 75.4 (70.6,76.4) |
| Gene therapy | 10 | 6 | 16–20 | 23 | 7.6 (7.3,10.5) | 28.8 (28.2,30.3) | 35.0 (34.7,35.6) | 82.7 (81.5,85.2) | 11.1 (9.9,14.1) | 37.3 (30.0,45.4) |
| Gene therapy | 11 | 3 | 21–25 | 17 | 8.6 (8.4, 8.7) | 25.3 (25.1,26.0) | 35.3 (35.2,35.5) | 71.7 (70.8,74.0) | 27.6 (27.2,27.7) | 68.6 (64.9,69.8) |
| Gene therapy | Untransf. BCLshmiR c | 9 | 16 (7,24) | 32 (17,49) | 10.9 (8.6,11.8) | 31.0 (25.3,38.4) | 35.3 (33.8,36.5) | 85.5 (71.7,109.8) | 27.9 (23.3,38.6) | 71.8 (64.0,86.1) |
| Hydroxyurea | HU High Responder | 10 | 7 (2,16) | 46 (10,156) | 10.2 (8.5,11.1) | 35.6 (25.8,40.7) | 36.0 (34.1,37.0) | 98.8 (75.5,110.4) | 27.0 (23.0,33.2) | 87.6 (78.1,93.6) |
| Hydroxyurea | HU Low Responder | 8 | 8.5 (3,23) | 66 (24,144) | 7.3 (6.1, 9.0) | 33.2 (26.0,36.8) | 35.0 (34.1,35.7) | 94.3 (75.2,104.2) | 11.7 (6.3,19.8) | 48.6 (29.9,75.2) |
aValues shown are median (minimum,maximum). Gene Therapy includes data ≥ 5 months after treatment. Subject ages are reported as 5-year intervals to maintain privacy. Source data are provided as a Source Data file.
bHbS/beta zero thalassemia.
cUntransf. BCLshmiR corresponds to the seven untransfused BCLshmiR patients: 2, 4, 6, 7, 8, 9, and 11.