Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy: Results of a National Multicenter survey

Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators

Research output: Contribution to journalArticle

Abstract

BACKGROUND: The number of patients with sickle cell disease (SCD) has increased in Italy in the past decade due to immigration. In spite of the established efficacy of hydroxyurea (HU) in childhood, population-based data regarding its prescription and effectiveness come mainly from studies performed in adults or outside Europe.

POPULATION AND METHODS: The Hydroxyurea in SCD: A Large Nation-wide Cohort Study from Italy was a retrospective cohort study of adult and pediatric patients with SCD attending 32 centers. Pediatric data are analyzed separately.

RESULTS: Out of 504 children followed in 11 centers, 206 (40%) were on HU (194 SS/Sβ°, 12 SC/Sß+); 74% came from Sub-Saharian Africa and 18% from Europe. HU therapy indications for SS/Sβ° patients were as follows: 57% painful vaso-occlusive crisis, acute chest syndrome or both, 24% anemia, 8% anemia, and other reasons (the majority had Hb ≤ 8-8.5 g/dl, revealing scarce acceptance of low Hb values by pediatric hematologist). Mean starting dose was 15.5 mg/kg, and dose at full regimen was 17.1 mg/kg. Mean age at HU therapy was 7.68 years, although it was lower for SS/Sβ° patients. Only 10% started HU before 3 years. In 92%, 500 mg capsule was used; in 6%, the galenic was used; and in 2%, 100 mg tablet was used. Significant reduction of clinical events and inpatients admissions, with improvement in hematological parameters, was observed for SS/Sβ° patients and a trend toward improvement for SC/Sß+ patients was also observed.

CONCLUSIONS: HU effectiveness is demonstrated in a national cohort of children with SCD living in Italy, even at a lower dose than recommended, revealing good adherence to a treatment program by a socially vulnerable group of patients such as immigrants.

Original languageEnglish
JournalPediatric Blood and Cancer
Volume65
Issue number2
DOIs
Publication statusPublished - Feb 2018

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Hydroxyurea
Sickle Cell Anemia
Italy
Prescriptions
Pediatrics
Anemia
Cohort Studies
Acute Chest Syndrome
Emigration and Immigration
Surveys and Questionnaires
Tablets
Capsules
Inpatients
Therapeutics
Retrospective Studies
Population

Keywords

  • Adolescent
  • Anemia, Sickle Cell/drug therapy
  • Child
  • Child, Preschool
  • Drug Prescriptions
  • Emigrants and Immigrants
  • Female
  • Follow-Up Studies
  • Health Services Accessibility
  • Humans
  • Hydroxyurea/administration & dosage
  • Infant
  • Italy/epidemiology
  • Male

Cite this

Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy : Results of a National Multicenter survey. / Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators.

In: Pediatric Blood and Cancer, Vol. 65, No. 2, 02.2018.

Research output: Contribution to journalArticle

Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators. / Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy : Results of a National Multicenter survey. In: Pediatric Blood and Cancer. 2018 ; Vol. 65, No. 2.
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title = "Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy: Results of a National Multicenter survey",
abstract = "BACKGROUND: The number of patients with sickle cell disease (SCD) has increased in Italy in the past decade due to immigration. In spite of the established efficacy of hydroxyurea (HU) in childhood, population-based data regarding its prescription and effectiveness come mainly from studies performed in adults or outside Europe.POPULATION AND METHODS: The Hydroxyurea in SCD: A Large Nation-wide Cohort Study from Italy was a retrospective cohort study of adult and pediatric patients with SCD attending 32 centers. Pediatric data are analyzed separately.RESULTS: Out of 504 children followed in 11 centers, 206 (40{\%}) were on HU (194 SS/Sβ°, 12 SC/S{\ss}+); 74{\%} came from Sub-Saharian Africa and 18{\%} from Europe. HU therapy indications for SS/Sβ° patients were as follows: 57{\%} painful vaso-occlusive crisis, acute chest syndrome or both, 24{\%} anemia, 8{\%} anemia, and other reasons (the majority had Hb ≤ 8-8.5 g/dl, revealing scarce acceptance of low Hb values by pediatric hematologist). Mean starting dose was 15.5 mg/kg, and dose at full regimen was 17.1 mg/kg. Mean age at HU therapy was 7.68 years, although it was lower for SS/Sβ° patients. Only 10{\%} started HU before 3 years. In 92{\%}, 500 mg capsule was used; in 6{\%}, the galenic was used; and in 2{\%}, 100 mg tablet was used. Significant reduction of clinical events and inpatients admissions, with improvement in hematological parameters, was observed for SS/Sβ° patients and a trend toward improvement for SC/S{\ss}+ patients was also observed.CONCLUSIONS: HU effectiveness is demonstrated in a national cohort of children with SCD living in Italy, even at a lower dose than recommended, revealing good adherence to a treatment program by a socially vulnerable group of patients such as immigrants.",
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author = "{Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators} and Raffaella Colombatti and Giovanni Palazzi and Nicoletta Masera and Notarangelo, {Lucia Dora} and Elisa Bonetti and Piera Samperi and Angelica Barone and Silverio Perrotta and Elena Facchini and Maurizio Miano and {Del Vecchio}, {Giovanni Carlo} and Guerzoni, {Maria Elena} and Paola Corti and Federica Menzato and Simone Cesaro and Maddalena Casale and Paolo Rigano and Forni, {Gian Luca} and Giovanna Russo and Laura Sainati",
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year = "2018",
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doi = "10.1002/pbc.26774",
language = "English",
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journal = "Pediatric Blood and Cancer",
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T1 - Hydroxyurea prescription, availability and use for children with sickle cell disease in Italy

T2 - Results of a National Multicenter survey

AU - Italian Multicenter Study of Hydroxyurea in Sickle Cell Anemia Investigators

AU - Colombatti, Raffaella

AU - Palazzi, Giovanni

AU - Masera, Nicoletta

AU - Notarangelo, Lucia Dora

AU - Bonetti, Elisa

AU - Samperi, Piera

AU - Barone, Angelica

AU - Perrotta, Silverio

AU - Facchini, Elena

AU - Miano, Maurizio

AU - Del Vecchio, Giovanni Carlo

AU - Guerzoni, Maria Elena

AU - Corti, Paola

AU - Menzato, Federica

AU - Cesaro, Simone

AU - Casale, Maddalena

AU - Rigano, Paolo

AU - Forni, Gian Luca

AU - Russo, Giovanna

AU - Sainati, Laura

N1 - © 2017 Wiley Periodicals, Inc.

PY - 2018/2

Y1 - 2018/2

N2 - BACKGROUND: The number of patients with sickle cell disease (SCD) has increased in Italy in the past decade due to immigration. In spite of the established efficacy of hydroxyurea (HU) in childhood, population-based data regarding its prescription and effectiveness come mainly from studies performed in adults or outside Europe.POPULATION AND METHODS: The Hydroxyurea in SCD: A Large Nation-wide Cohort Study from Italy was a retrospective cohort study of adult and pediatric patients with SCD attending 32 centers. Pediatric data are analyzed separately.RESULTS: Out of 504 children followed in 11 centers, 206 (40%) were on HU (194 SS/Sβ°, 12 SC/Sß+); 74% came from Sub-Saharian Africa and 18% from Europe. HU therapy indications for SS/Sβ° patients were as follows: 57% painful vaso-occlusive crisis, acute chest syndrome or both, 24% anemia, 8% anemia, and other reasons (the majority had Hb ≤ 8-8.5 g/dl, revealing scarce acceptance of low Hb values by pediatric hematologist). Mean starting dose was 15.5 mg/kg, and dose at full regimen was 17.1 mg/kg. Mean age at HU therapy was 7.68 years, although it was lower for SS/Sβ° patients. Only 10% started HU before 3 years. In 92%, 500 mg capsule was used; in 6%, the galenic was used; and in 2%, 100 mg tablet was used. Significant reduction of clinical events and inpatients admissions, with improvement in hematological parameters, was observed for SS/Sβ° patients and a trend toward improvement for SC/Sß+ patients was also observed.CONCLUSIONS: HU effectiveness is demonstrated in a national cohort of children with SCD living in Italy, even at a lower dose than recommended, revealing good adherence to a treatment program by a socially vulnerable group of patients such as immigrants.

AB - BACKGROUND: The number of patients with sickle cell disease (SCD) has increased in Italy in the past decade due to immigration. In spite of the established efficacy of hydroxyurea (HU) in childhood, population-based data regarding its prescription and effectiveness come mainly from studies performed in adults or outside Europe.POPULATION AND METHODS: The Hydroxyurea in SCD: A Large Nation-wide Cohort Study from Italy was a retrospective cohort study of adult and pediatric patients with SCD attending 32 centers. Pediatric data are analyzed separately.RESULTS: Out of 504 children followed in 11 centers, 206 (40%) were on HU (194 SS/Sβ°, 12 SC/Sß+); 74% came from Sub-Saharian Africa and 18% from Europe. HU therapy indications for SS/Sβ° patients were as follows: 57% painful vaso-occlusive crisis, acute chest syndrome or both, 24% anemia, 8% anemia, and other reasons (the majority had Hb ≤ 8-8.5 g/dl, revealing scarce acceptance of low Hb values by pediatric hematologist). Mean starting dose was 15.5 mg/kg, and dose at full regimen was 17.1 mg/kg. Mean age at HU therapy was 7.68 years, although it was lower for SS/Sβ° patients. Only 10% started HU before 3 years. In 92%, 500 mg capsule was used; in 6%, the galenic was used; and in 2%, 100 mg tablet was used. Significant reduction of clinical events and inpatients admissions, with improvement in hematological parameters, was observed for SS/Sβ° patients and a trend toward improvement for SC/Sß+ patients was also observed.CONCLUSIONS: HU effectiveness is demonstrated in a national cohort of children with SCD living in Italy, even at a lower dose than recommended, revealing good adherence to a treatment program by a socially vulnerable group of patients such as immigrants.

KW - Adolescent

KW - Anemia, Sickle Cell/drug therapy

KW - Child

KW - Child, Preschool

KW - Drug Prescriptions

KW - Emigrants and Immigrants

KW - Female

KW - Follow-Up Studies

KW - Health Services Accessibility

KW - Humans

KW - Hydroxyurea/administration & dosage

KW - Infant

KW - Italy/epidemiology

KW - Male

U2 - 10.1002/pbc.26774

DO - 10.1002/pbc.26774

M3 - Article

C2 - 28868627

VL - 65

JO - Pediatric Blood and Cancer

JF - Pediatric Blood and Cancer

SN - 1545-5009

IS - 2

ER -