TY - JOUR T1 - Effect of hydroxyurea on mortality and morbidity in adult sickle cell anemia: risks and benefits up to 9 years of treatment. AU - Steinberg,Martin H, AU - Barton,Franca, AU - Castro,Oswaldo, AU - Pegelow,Charles H, AU - Ballas,Samir K, AU - Kutlar,Abdullah, AU - Orringer,Eugene, AU - Bellevue,Rita, AU - Olivieri,Nancy, AU - Eckman,James, AU - Varma,Mala, AU - Ramirez,Gloria, AU - Adler,Brian, AU - Smith,Wally, AU - Carlos,Timothy, AU - Ataga,Kenneth, AU - DeCastro,Laura, AU - Bigelow,Carolyn, AU - Saunthararajah,Yogen, AU - Telfer,Margaret, AU - Vichinsky,Elliott, AU - Claster,Susan, AU - Shurin,Susan, AU - Bridges,Kenneth, AU - Waclawiw,Myron, AU - Bonds,Duane, AU - Terrin,Michael, PY - 2003/4/4/pubmed PY - 2003/4/16/medline PY - 2003/4/4/entrez SP - 1645 EP - 51 JF - JAMA JO - JAMA VL - 289 IS - 13 N2 - CONTEXT: Hydroxyurea increases levels of fetal hemoglobin (HbF) and decreases morbidity from vaso-occlusive complications in patients with sickle cell anemia (SCA). High HbF levels reduce morbidity and mortality. OBJECTIVE: To determine whether hydroxyurea attenuates mortality in patients with SCA. DESIGN: Long-term observational follow-up study of mortality in patients with SCA who originally participated in the randomized, double-blind, placebo-controlled Multicenter Study of Hydroxyurea in Sickle Cell Anemia (MSH), conducted in 1992-1995, to determine if hydroxyurea reduces vaso-occlusive events. In the MSH Patients' Follow-up, conducted in 1996-2001, patients could continue, stop, or start hydroxyurea. Data were collected during the trial and in the follow-up period. SETTING: Inpatients and outpatients in 21 sickle cell referral centers in the United States and Canada. PATIENTS: Two-hundred ninety-nine adult patients with frequent painful episodes enrolled in the follow-up. Follow-up data through May 2001 were complete for 233 patients. INTERVENTION: In the MSH, patients were randomly assigned to receive hydroxyurea (n = 152) or placebo (n = 147). MAIN OUTCOME MEASURE: Mortality, HbF levels, painful episodes, acute chest syndrome, and blood cell counts. The randomized trial was not designed to detect specified differences in mortality. RESULTS: Seventy-five of the original 299 patients died, 28% from pulmonary disease. Patients with reticulocyte counts less than 250 000/mm3 and hemoglobin levels lower than 9 g/dL had increased mortality (P =.002). Cumulative mortality at 9 years was 28% when HbF levels were lower than 0.5 g/dL after the trial was completed compared with 15% when HbF levels were 0.5 g/dL or higher (P =.03). Individuals who had acute chest syndrome during the trial had 32% mortality compared with 18% of individuals without acute chest syndrome (P =.02). Patients with 3 or more painful episodes per year during the trial had 27% mortality compared with 17% of patients with less frequent episodes (P =.06). Taking hydroxyurea was associated with a 40% reduction in mortality (P =.04) in this observational follow-up with self-selected treatment. There were 3 cases of cancer, 1 fatal. CONCLUSIONS: Adult patients taking hydroxyurea for frequent painful sickle cell episodes appear to have reduced mortality after 9 of years follow-up. Survival was related to HbF levels and frequency of vaso-occlusive events. Whether indications for hydroxyurea treatment should be expanded is unknown. SN - 0098-7484 UR - https://www.unboundmedicine.com/medline/citation/12672732/Effect_of_hydroxyurea_on_mortality_and_morbidity_in_adult_sickle_cell_anemia:_risks_and_benefits_up_to_9_years_of_treatment_ DB - PRIME DP - Unbound Medicine ER -