Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Real-life experience with hydroxyurea in sickle cell disease: A multicenter study in a cohort of patients with heterogeneous descent
source: Blood Cells, Molecules and Diseases
authors: Rigano P, De Franceschi L, Sainati L, Piga A, Piel FB, Cappellini MD, Fidone C, Masera N, Palazzi G, Gianesin B, Forni GLsummary/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±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
read more full text
How sickled red blood cells stick to blood vesselsOne of the most common complications o...
Unanticipated consequences identified after implementation of a pediatric emergency department (PED)-based intranasa...Background: The 2014 National Heart Lun...
Access to Care for Individuals With Sickle Cell Anemia – SCDAASickle cell disease (SCD) is a genetic b...
Vanderbilt University Medical CenterThe Vanderbilt-Meharry-Matthew Walker Ce...
Santosh L. Saraf, MDDr. Santosh Saraf is an assistant profes...
Dana Carroll, PhDDana Carroll, PhD, is a distinguished pr...
Ask Me Anything Blood Donor Educational WebinarJoin us for our next 1-hour virtual educ...
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.