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

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Title: Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent
Authors: Piel, FBJ
Rigano, P
De Francesci, L
Sainati, L
Piga, A
Cappellini, MD
Fidone, C
Masera, N
Palazzi, G
Gianesin, B
Forni, GL
Item Type: Journal Article
Abstract: 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 ± 15 years, 51.4% males). Hydroxyurea median treatment duration was 7 years (range: < 1 year to 29 years) at a mean therapeutic dose of 18 ± 4.7 mg/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 ≥ 10 years). 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).
Issue Date: 9-Oct-2017
Date of Acceptance: 8-Aug-2017
URI: http://hdl.handle.net/10044/1/51635
DOI: https://dx.doi.org/10.1016/j.bcmd.2017.08.017
ISSN: 1079-9796
Publisher: Elsevier
Start Page: 82
End Page: 89
Journal / Book Title: Blood Cells, Molecules and Diseases
Volume: 69
Copyright Statement: © 2017, Elsevier. Licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International http://creativecommons.org/licenses/by-nc-nd/4.0/
Keywords: 1103 Clinical Sciences
Immunology
Publication Status: Published
Appears in Collections:Faculty of Medicine
Epidemiology, Public Health and Primary Care



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