Imperial College London

Dr Frédéric B. Piel

Faculty of MedicineSchool of Public Health

Senior Lecturer



+44 (0)20 7594 3346f.piel




Praed StreetSt Mary's Campus






BibTex format

author = {Piel, FBJ and Rigano, P and De, Francesci L and Sainati, L and Piga, A and Cappellini, MD and Fidone, C and Masera, N and Palazzi, G and Gianesin, B and Forni, GL},
doi = {10.1016/j.bcmd.2017.08.017},
journal = {Blood Cells, Molecules and Diseases},
pages = {82--89},
title = {Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent},
url = {},
volume = {69},
year = {2017}

RIS format (EndNote, RefMan)

AB - 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 (NCT02709681).
AU - Piel,FBJ
AU - Rigano,P
AU - De,Francesci L
AU - Sainati,L
AU - Piga,A
AU - Cappellini,MD
AU - Fidone,C
AU - Masera,N
AU - Palazzi,G
AU - Gianesin,B
AU - Forni,GL
DO - 10.1016/j.bcmd.2017.08.017
EP - 89
PY - 2017///
SN - 1079-9796
SP - 82
TI - Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent
T2 - Blood Cells, Molecules and Diseases
UR -
UR -
VL - 69
ER -