Effect of Hydroxyurea on Mortality and Morbidity in Adult Sickle Cell Anemia

摘要: ContextHydroxyurea increases levels of fetal hemoglobin (HbF) and decreases morbidity from vaso-occlusive complications in patients with sickle cell anemia (SCA). High HbF reduce and mortality.ObjectiveTo determine whether hydroxyurea attenuates mortality in patients with SCA.DesignLong-term observational follow-up study SCA who originally participated the randomized, double-blind, placebo-controlled Multicenter Study Hydroxyurea in Sickle Cell Anemia (MSH), conducted 1992-1995, to determine if reduces events. In MSH Patients' Follow-up, 1996-2001, patients could continue, stop, or start hydroxyurea. Data were collected during trial and in the period.SettingInpatients outpatients 21 referral centers United States Canada.PatientsTwo-hundred ninety-nine adult frequent painful episodes enrolled follow-up. Follow-up data through May 2001 complete for 233 patients.InterventionIn MSH, randomly assigned to receive (n = 152) placebo 147).Main Outcome MeasureMortality, levels, painful episodes, acute chest syndrome, blood counts. The randomized was not designed to detect specified differences mortality.ResultsSeventy-five original 299 died, 28% from pulmonary disease. Patients reticulocyte counts less than 250 000/mm3 lower than 9 g/dL had increased (P .002). Cumulative at years was when lower than 0.5 g/dL after completed compared 15% HbF levels higher (P .03 ). Individuals had acute chest syndrome 32% 18% of individuals without acute syndrome (P .02). 3 more painful per year 27% compared with 17% frequent episodes (P .06). Taking was associated a 40% reduction mortality (P .04) this with self-selected treatment. There cases cancer, 1 fatal.ConclusionsAdult taking painful sickle appear have reduced years follow-up. Survival related frequency of vaso-occlusive Whether indications treatment should be expanded is unknown.