Children and adolescents with SCA have similar laboratory efficacy using hydroxyurea at MTD as adults
| . | Pediatric (phase 1/2 trial) . | Adult (phase 1/2 trial) . |
|---|---|---|
| No. of patients | 66 | 32 |
| MTD, mg/kg/d | 25.6 | 21.3 |
| Hemoglobin, g/dL | 1.2 | 1.2 |
| MCV, fL | 14 | 23 |
| HbF, percentage | 9.6 | 11.2 |
| Reticulocytes, ×109/L | −146 | −158 |
| WBC, ×109/L | −4.2 | −5.0 |
| ANC, ×109/L | −2.2 | −2.8 |
| Bilirubin, mg/dL | −1.0 | −2.0 |
| LDH, U/L | −310 | Not provided |
| . | Pediatric (phase 1/2 trial) . | Adult (phase 1/2 trial) . |
|---|---|---|
| No. of patients | 66 | 32 |
| MTD, mg/kg/d | 25.6 | 21.3 |
| Hemoglobin, g/dL | 1.2 | 1.2 |
| MCV, fL | 14 | 23 |
| HbF, percentage | 9.6 | 11.2 |
| Reticulocytes, ×109/L | −146 | −158 |
| WBC, ×109/L | −4.2 | −5.0 |
| ANC, ×109/L | −2.2 | −2.8 |
| Bilirubin, mg/dL | −1.0 | −2.0 |
| LDH, U/L | −310 | Not provided |
Data are from published phase 1/2 trials for children21 and adults15 who were taking hydroxyurea at the end of study.
SCA indicates sickle cell anemia; MTD, maximum tolerated dose; MCV, mean corpuscular volume; HbF, fetal hemoglobin; WBC, white blood cell; ANC, absolute neutrophil count; and LDH, lactate dehydrogenase.