RCTs
| Study/authors/location . | Inclusion criteria . | Intervention . | Age mean/median (range) . | N . | Follow-up period . | Outcomes for hydroxyurea treatment vs no hydroxyurea . |
|---|---|---|---|---|---|---|
| MSH*/Charache et al, 1995/North America | SCA; age >18 y; ≥3 crises/y | HU vs placebo; HU: 15 mg/kg per day escalated to MTD | 30 y (18-59) | 299 (HU: 152) | 21 mo (mean); 134/299 with 2 y | 1°: decreased VOC (P < .001); 2°: decreased ACS (P < .001), decreased transfusions (P = .001) |
| BABY HUG/Wang et al, 2011/United States | SCA; 9-18 mo; no requirement for clinical severity | HU vs placebo; HU: fixed at 20 mg/kg per day | 13.6 mo (HU); 13.5 mo (placebo) | 193 (HU: 96) | 24 mo | 1°: no difference in splenic function (P = .21) or renal function (P = .84); 2°: decreased VOC (P = .002), ACS (P = .02), dactylitis (P < .0001) |
| CHAMPS/Wang et al, 2011/United States | HbSC ; age ≥5 y; 1 VOC in previous y | 4 groups: HU+placebo, Mg+placebo, HU+Mg, placebo+placebo; HU: fixed at 20 mg/kg per day | 13.6 y (5-53) | 44 (HU: 22) | 36 patients: 8 wk; 22 patients: 44 wk | Early trial termination due to poor accrual; 1°: no difference in hyperdense red cells; 2°: no difference in frequency of VOC (HU: increased HbF, week 8: P < .05 and week 24: P < .001) |
| Jain et al, 2012/India | SCA; >3 VOC/y; requiring hospitalization or transfusion >3/y | HU vs placebo; HU: fixed at 10 mg/kg per day | 12.7 ± 4.4 y (HU); 11.7 ± 4.1 y (placebo) (5-18) | 60 (HU: 30) | 18 mo | 1°: decreased VOC (P < .001); 2°: decreased transfusions (P < .001); decreased hospitalizations (P < .001) |
| SWiTCH/Ware et al, 2012/United States | SCA | HU/phlebotomy vs txn/chelation (noninferiority trial design); HU: 20 mg/kg per day escalated to MTD | 13y (5-18) | 133 (HU: 67) | 24 mo (mean) | Study closed at first interim analysis; 1°: equivalent liver iron content at interim analysis for both arms; rate of stroke with HU arm within noninferiority margin |
| Study/authors/location . | Inclusion criteria . | Intervention . | Age mean/median (range) . | N . | Follow-up period . | Outcomes for hydroxyurea treatment vs no hydroxyurea . |
|---|---|---|---|---|---|---|
| MSH*/Charache et al, 1995/North America | SCA; age >18 y; ≥3 crises/y | HU vs placebo; HU: 15 mg/kg per day escalated to MTD | 30 y (18-59) | 299 (HU: 152) | 21 mo (mean); 134/299 with 2 y | 1°: decreased VOC (P < .001); 2°: decreased ACS (P < .001), decreased transfusions (P = .001) |
| BABY HUG/Wang et al, 2011/United States | SCA; 9-18 mo; no requirement for clinical severity | HU vs placebo; HU: fixed at 20 mg/kg per day | 13.6 mo (HU); 13.5 mo (placebo) | 193 (HU: 96) | 24 mo | 1°: no difference in splenic function (P = .21) or renal function (P = .84); 2°: decreased VOC (P = .002), ACS (P = .02), dactylitis (P < .0001) |
| CHAMPS/Wang et al, 2011/United States | HbSC ; age ≥5 y; 1 VOC in previous y | 4 groups: HU+placebo, Mg+placebo, HU+Mg, placebo+placebo; HU: fixed at 20 mg/kg per day | 13.6 y (5-53) | 44 (HU: 22) | 36 patients: 8 wk; 22 patients: 44 wk | Early trial termination due to poor accrual; 1°: no difference in hyperdense red cells; 2°: no difference in frequency of VOC (HU: increased HbF, week 8: P < .05 and week 24: P < .001) |
| Jain et al, 2012/India | SCA; >3 VOC/y; requiring hospitalization or transfusion >3/y | HU vs placebo; HU: fixed at 10 mg/kg per day | 12.7 ± 4.4 y (HU); 11.7 ± 4.1 y (placebo) (5-18) | 60 (HU: 30) | 18 mo | 1°: decreased VOC (P < .001); 2°: decreased transfusions (P < .001); decreased hospitalizations (P < .001) |
| SWiTCH/Ware et al, 2012/United States | SCA | HU/phlebotomy vs txn/chelation (noninferiority trial design); HU: 20 mg/kg per day escalated to MTD | 13y (5-18) | 133 (HU: 67) | 24 mo (mean) | Study closed at first interim analysis; 1°: equivalent liver iron content at interim analysis for both arms; rate of stroke with HU arm within noninferiority margin |
1°, primary outcomes; 2°, secondary outcomes; BABY HUG, Pediatric Hydroxyurea in Sickle Cell Anemia; CHAMPS, Hydroxyurea and Magnesium Pidolate to Treat People with Hemoglobin Sickle Cell Disease; HU, hydroxyurea; Mg, magnesium pidolate; MTD, maximum tolerated dose; txn, transfusion.
Secondary analyses of MSH included in the current review.