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

Paolo Rigano, Lucia De Franceschi, Laura Sainati, Antonio Piga, Frédéric B. Piel, Maria Domenica Cappellini, Carmelo Fidone, Nicoletta Masera, Giovanni Palazzi, Barbara Gianesin, Gian Luca Forni

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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).

Original languageEnglish
Pages (from-to)82-89
JournalBlood Cells, Molecules, and Diseases
Volume69
DOIs
Publication statusPublished - 2018

Fingerprint

Hydroxyurea
Sickle Cell Anemia
Multicenter Studies
Therapeutics
Acute Chest Syndrome
Sickle Hemoglobin
Fetal Hemoglobin
Osteonecrosis
Cerebral Infarction
Platelet Count
L-Lactate Dehydrogenase
Italy
Hospitalization
Cohort Studies
Stroke
Genotype
Clinical Trials
Pediatrics
Safety
Incidence

Keywords

  • Complications
  • Hydroxycarbamide
  • Hydroxyurea
  • Management
  • Real-world
  • Sickle cell disease

ASJC Scopus subject areas

  • Molecular Medicine
  • Molecular Biology
  • Hematology
  • Cell Biology

Cite this

Real-life experience with hydroxyurea in sickle cell disease : A multicenter study in a cohort of patients with heterogeneous descent. / Rigano, Paolo; De Franceschi, Lucia; Sainati, Laura; Piga, Antonio; Piel, Frédéric B.; Cappellini, Maria Domenica; Fidone, Carmelo; Masera, Nicoletta; Palazzi, Giovanni; Gianesin, Barbara; Forni, Gian Luca.

In: Blood Cells, Molecules, and Diseases, Vol. 69, 2018, p. 82-89.

Research output: Contribution to journalArticle

Rigano, Paolo ; De Franceschi, Lucia ; Sainati, Laura ; Piga, Antonio ; Piel, Frédéric B. ; Cappellini, Maria Domenica ; Fidone, Carmelo ; Masera, Nicoletta ; Palazzi, Giovanni ; Gianesin, Barbara ; Forni, Gian Luca. / Real-life experience with hydroxyurea in sickle cell disease : A multicenter study in a cohort of patients with heterogeneous descent. In: Blood Cells, Molecules, and Diseases. 2018 ; Vol. 69. pp. 82-89.
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AU - Piga, Antonio

AU - Piel, Frédéric B.

AU - Cappellini, Maria Domenica

AU - Fidone, Carmelo

AU - Masera, Nicoletta

AU - Palazzi, Giovanni

AU - Gianesin, Barbara

AU - Forni, Gian Luca

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