Multi-center Study of Hydroxyurea (MSH) trial and the Stroke Prevention Trial (STOP) trial.
| Study design . | Randomized, controlled trial . | Randomized, controlled trial . |
|---|---|---|
| No. patients | 299 | 130 |
| Inclusion criteria | HbSS | HbSS or HbS β0 thalassemia |
| Age ≥18 y | Age 2-16 y | |
| ≥3 painful episodes in the year prior For transfused patients, HbA ≤15% | TCD velocity ≥200 cm/second | |
| Exclusion criteria | Pregnancy | Pregnancy |
| HIV | HIV | |
| Chronic transfusion therapy | Chronic transfusion therapy | |
| Stroke within last 6 y | History of stroke | |
| Bone marrow suppression | History of seizures | |
| Narcotic abuse or overuse | Serum ferritin >500 ng/mL | |
| Prior hydroxyurea therapy | ||
| Use of another antisickling agent | ||
| Primary analysis | Reduction in painful episodes Intention to treat analysis | Redcution in stroke incidence Intention to treat analysis |
| Level of significance | .05 | .05 |
| Secondary outcome measures | Reduction in acute chest syndrome episodes, death, stroke, hepatic sequestration, blood transfusion requirements, and time to first and second painful episode | None |
| Level of significance | .01 | None |
| Study design . | Randomized, controlled trial . | Randomized, controlled trial . |
|---|---|---|
| No. patients | 299 | 130 |
| Inclusion criteria | HbSS | HbSS or HbS β0 thalassemia |
| Age ≥18 y | Age 2-16 y | |
| ≥3 painful episodes in the year prior For transfused patients, HbA ≤15% | TCD velocity ≥200 cm/second | |
| Exclusion criteria | Pregnancy | Pregnancy |
| HIV | HIV | |
| Chronic transfusion therapy | Chronic transfusion therapy | |
| Stroke within last 6 y | History of stroke | |
| Bone marrow suppression | History of seizures | |
| Narcotic abuse or overuse | Serum ferritin >500 ng/mL | |
| Prior hydroxyurea therapy | ||
| Use of another antisickling agent | ||
| Primary analysis | Reduction in painful episodes Intention to treat analysis | Redcution in stroke incidence Intention to treat analysis |
| Level of significance | .05 | .05 |
| Secondary outcome measures | Reduction in acute chest syndrome episodes, death, stroke, hepatic sequestration, blood transfusion requirements, and time to first and second painful episode | None |
| Level of significance | .01 | None |