6/28/23, 11:19 PM MSH Pathway Feedback Search Clinical Topics Home Studies MSH MSH Disease Sickle cell disease Trial question What is the role of hydroxyurea in adult patients with sickle cell anemia? Study design Multi-center Double blinded RCT Population Characteristics of study participants 51.0% female N = 299 49.0% male 299 patients (151 female, 148 male) Inclusion criteria: adult patients with sickle cell anemia Key exclusion criteria: sickle cell-beta+-thalassemia and sickle cell- -thalassemia, pregnancy, narcotic addiction or regular consumption of > 30 oxycodone capsules (or the equivalent) every two weeks; indication of marrow depression; or prior hydroxyurea therapy; Interventions N=152 hydroxyurea (initial dose of 15 mg/kg body weight and adjusted according to marrow depression, platelet count, and Hgb level) N=147 placebo (Starch 1500, with the same adjustments of the dose) Primary outcome https://web.pathway.md/studies/recgPETAFbH3EUNoH 1/2 6/28/23, 11:19 PM MSH Pathway Painful crises 4.5 % 4.5 3.4 % 2.5 2.3 % Significant decrease 1.1 % NNT = 50 0.0 % Hydroxyurea Placebo Significant decrease in painful crises (2.5% vs. 4.5%; RR 0.56, 95% CI 0.23 to 0.89) Secondary outcomes Significant decrease in chest syndrome (25 vs. 51; RR 0.49, 95% CI 0.2 to 0.78) Significant decrease in underwent transfusions (48 vs. 73; RR 0.66, 95% CI 0.27 to 1.05) Conclusion In adult patients with sickle cell anemia, hydroxyurea was superior to placebo with respect to painful crises. Reference Charache S, Terrin ML, Moore RD et al. Effect of hydroxyurea on the frequency of painful crises in sickle cell anemia. Investigators of the Multicenter Study of Hydroxyurea in Sickle Cell Anemia. N Engl J Med. 1995 May 18;332(20):1317-22. Open reference URL https://web.pathway.md/studies/recgPETAFbH3EUNoH 2/2