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scientific articles

Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent

key information

source: Blood Cells, Molecules and Diseases

year: 2018

authors: Rigano P, De Franceschi L, Sainati L, Piga A, Piel FB, Cappellini MD, Fidone C, Masera N, Palazzi G, Gianesin B, Forni GL


We conducted the first nation-wide cohort study of sickle cell disease (SCD) in Italy, a Southern European country exposed to intense recent flux migration from endemic areas for SCD. We evaluate the impact of hydroxyurea on a total of 652 pediatric and adult patients from 33 Reference Centers for SCD (mean age 24.5±15years, 51.4% males). Hydroxyurea median treatment duration was 7years (range: <1year to 29years) at a mean therapeutic dose of 18±4.7mg/kg/day. Hydroxyurea was associated with a significant increase in mean total and fetal hemoglobin and a significant decrease in mean hemoglobin S, white blood and platelet counts, and lactate dehydrogenase levels. Hydroxyurea was associated with a significant reduction in the incidence of acute chest syndrome (-29.3%, p<0.001), vaso-occlusive crisis (-34.1%, p<0.001), hospitalization (-53.2%, p<0.001), and bone necrosis (-6.9%, p<0.001). New silent cerebral infarction (SCI) occurred during treatment (+42.4%, p<0.001) but not stroke (+0.5%, p=0.572). These observations were generally consistent upon stratification for age, descent (Caucasian or African), genotype (βS/βS, βS/β0 or βS/β+) and duration of treatment (< or >=10years). There were no new safety concerns observed compared to those commonly reported in the literature. Our study, conducted on a large population of patients with different descent and compound state supports the benefits of hydroxyurea therapy as a treatment option. Registered at clinical trials.gov (NCT02709681).

organization: Azienda Ospedaliera-Università di Padova, Padua, Italy; University of Verona, AOUI Verona, Policlinico GB Rossi, Verona, Italy; University of Turin, Italy; Imperial College London, UK; "Ca' Granda" Ospedale Maggiore Policlinico Foundation IRCCS, Italy; Campus of Haematology Franco e Piera Cutino-A.O.O.R. "Villa Sofia-Cervello", Palermo, Italy; SIMTI Ragusa, Italy; San Gerardo Hospital, Monza, Italy; Azienda Ospedaliera-Università di Modena, Italy; Galliera Hospital, Italy

DOI: 10.1016/j.bcmd.2017.08.017

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