Patient characteristics and treatment response
. | Visit . | Patient 1 . | Patient 2 . | Patient 3* . | Patient 4 . | Patient 5 . | Patient 6 . |
---|---|---|---|---|---|---|---|
Age, y | — | 22 | 40 | 38 | 31 | 50 | 54 |
Sex | — | Male | Male | Male | Female | Male | Male |
Splenectomy (age, y) | — | No | Yes (21) | Yes (30) | Yes (7) | Yes (4) | Yes (5) |
No. of transfusions in past year | — | 0 | 4 | 1 | 1 | 1 | 0 |
No. of injections (wk) | — | 24 (1-12) | 21 (1-11) | 4 (1-2) | 16 (1-8) | 24 (1-12) | 16 (1-8) |
Hemoglobin, g/dL | Baseline | 8.8 | 10.7 | 8 | 6.1 | 7.8 | 6 |
Peak† | 9.4 | 11.8 | — | 7.7 | 8.7 | 7.6 | |
Week12‡ | 9.4 | 11.8 | — | 6.4 | 8.7 | 7.5 | |
HbF, g/dL | Baseline | 7 | 0.1 | 1.7 | 2.8 | 4.8 | 3.6 |
Peak† | 7.5 | 0.7 | — | 3.5 | 5.6 | 4.1 | |
Week12‡ | 7.1 | 0.7 | — | 3.4 | 5.6 | 3.6 | |
Indirect bilirubin, μM | Baseline | 25.7 | 104.3 | 47.9 | 89 | 33 | 20 |
Nadir‡ | 27.4 | 83.8 | — | 48 | 18 | 8 | |
Week12 | 30.8 | 83.8 | — | 48 | 33 | 10 | |
Serum lactate dehydrogenase, U/L | Baseline | 893 | 596 | 543 | 458 | 192 | 258 |
Nadir | 660 | 541 | — | 260 | 149 | 204 | |
Week12 | 660 | 556 | — | 324 | 167 | 204 | |
Platelet count, × 109/L | Baseline | 233 | 625 | 536 | 720 | 627 | 721 |
Peak† | 296 | 1038 | — | 1265 | 801 | 1301 | |
Week12‡ | 281 | 1038 | — | 1265 | 756 | 1301 | |
Neutrophil count, × 109/L | Baseline | 5.57 | 5 | 5.4 | 5.6 | 5.1 | 11.3 |
Nadir | 2.82 | 2.2 | — | 5.2 | 2.1 | 4.5 | |
Week12 | 4.62 | 2.2 | — | 7.9 | 10.3 | 5.7 | |
Absolute reticulocyte count, × 109/L | Baseline | 165 | 155 | 165 | 163 | 196 | 106 |
Nadir‡ | 135 | 160 | — | 116 | 125 | 79 | |
Week12‡ | 135 | 160 | — | 116 | 145 | 79 |
. | Visit . | Patient 1 . | Patient 2 . | Patient 3* . | Patient 4 . | Patient 5 . | Patient 6 . |
---|---|---|---|---|---|---|---|
Age, y | — | 22 | 40 | 38 | 31 | 50 | 54 |
Sex | — | Male | Male | Male | Female | Male | Male |
Splenectomy (age, y) | — | No | Yes (21) | Yes (30) | Yes (7) | Yes (4) | Yes (5) |
No. of transfusions in past year | — | 0 | 4 | 1 | 1 | 1 | 0 |
No. of injections (wk) | — | 24 (1-12) | 21 (1-11) | 4 (1-2) | 16 (1-8) | 24 (1-12) | 16 (1-8) |
Hemoglobin, g/dL | Baseline | 8.8 | 10.7 | 8 | 6.1 | 7.8 | 6 |
Peak† | 9.4 | 11.8 | — | 7.7 | 8.7 | 7.6 | |
Week12‡ | 9.4 | 11.8 | — | 6.4 | 8.7 | 7.5 | |
HbF, g/dL | Baseline | 7 | 0.1 | 1.7 | 2.8 | 4.8 | 3.6 |
Peak† | 7.5 | 0.7 | — | 3.5 | 5.6 | 4.1 | |
Week12‡ | 7.1 | 0.7 | — | 3.4 | 5.6 | 3.6 | |
Indirect bilirubin, μM | Baseline | 25.7 | 104.3 | 47.9 | 89 | 33 | 20 |
Nadir‡ | 27.4 | 83.8 | — | 48 | 18 | 8 | |
Week12 | 30.8 | 83.8 | — | 48 | 33 | 10 | |
Serum lactate dehydrogenase, U/L | Baseline | 893 | 596 | 543 | 458 | 192 | 258 |
Nadir | 660 | 541 | — | 260 | 149 | 204 | |
Week12 | 660 | 556 | — | 324 | 167 | 204 | |
Platelet count, × 109/L | Baseline | 233 | 625 | 536 | 720 | 627 | 721 |
Peak† | 296 | 1038 | — | 1265 | 801 | 1301 | |
Week12‡ | 281 | 1038 | — | 1265 | 756 | 1301 | |
Neutrophil count, × 109/L | Baseline | 5.57 | 5 | 5.4 | 5.6 | 5.1 | 11.3 |
Nadir | 2.82 | 2.2 | — | 5.2 | 2.1 | 4.5 | |
Week12 | 4.62 | 2.2 | — | 7.9 | 10.3 | 5.7 | |
Absolute reticulocyte count, × 109/L | Baseline | 165 | 155 | 165 | 163 | 196 | 106 |
Nadir‡ | 135 | 160 | — | 116 | 125 | 79 | |
Week12‡ | 135 | 160 | — | 116 | 145 | 79 |