Hydroxyurea for the treatment of sickle cell disease: Efficacy, barriers, toxicity, and management in children

John J. Strouse; Matthew M. Heeney

doi:10.1002/pbc.24178

Hydroxyurea for the treatment of sickle cell disease: Efficacy, barriers, toxicity, and management in children

John J. Strouse, Matthew M. Heeney

Research output: Contribution to journal › Review article › peer-review

73 Scopus citations

Abstract

Hydroxyurea is the only approved medication in the United States for the treatment of sickle cell anemia (HbSS) and is widely used in children despite an indication limited to adults. We review the evidence of efficacy and safety in children with reference to pivotal adult studies. This evidence and expert opinion form the basis for recommended guidelines for the use of hydroxyurea in children including indications, dosing, therapeutic and safety monitoring, and interventions to improve adherence. However, there are substantial gaps in our knowledge to be addressed by on-going and planned studies in children.

Original language	English (US)
Pages (from-to)	365-371
Number of pages	7
Journal	Pediatric Blood and Cancer
Volume	59
Issue number	2
DOIs	https://doi.org/10.1002/pbc.24178
State	Published - Aug 2012
Externally published	Yes

Keywords

Children
Efficacy
Hydroxyurea
Sickle cell disease

ASJC Scopus subject areas

Pediatrics, Perinatology, and Child Health
Hematology
Oncology

Access to Document

10.1002/pbc.24178

Cite this

@article{f8ee728df30948e094db2ff5f79ad2e0,

title = "Hydroxyurea for the treatment of sickle cell disease: Efficacy, barriers, toxicity, and management in children",

abstract = "Hydroxyurea is the only approved medication in the United States for the treatment of sickle cell anemia (HbSS) and is widely used in children despite an indication limited to adults. We review the evidence of efficacy and safety in children with reference to pivotal adult studies. This evidence and expert opinion form the basis for recommended guidelines for the use of hydroxyurea in children including indications, dosing, therapeutic and safety monitoring, and interventions to improve adherence. However, there are substantial gaps in our knowledge to be addressed by on-going and planned studies in children.",

keywords = "Children, Efficacy, Hydroxyurea, Sickle cell disease",

author = "Strouse, {John J.} and Heeney, {Matthew M.}",

year = "2012",

month = aug,

doi = "10.1002/pbc.24178",

language = "English (US)",

volume = "59",

pages = "365--371",

journal = "Pediatric Blood and Cancer",

issn = "1545-5009",

publisher = "Wiley-Liss Inc.",

number = "2",

}

TY - JOUR

T1 - Hydroxyurea for the treatment of sickle cell disease

T2 - Efficacy, barriers, toxicity, and management in children

AU - Strouse, John J.

AU - Heeney, Matthew M.

PY - 2012/8

Y1 - 2012/8

N2 - Hydroxyurea is the only approved medication in the United States for the treatment of sickle cell anemia (HbSS) and is widely used in children despite an indication limited to adults. We review the evidence of efficacy and safety in children with reference to pivotal adult studies. This evidence and expert opinion form the basis for recommended guidelines for the use of hydroxyurea in children including indications, dosing, therapeutic and safety monitoring, and interventions to improve adherence. However, there are substantial gaps in our knowledge to be addressed by on-going and planned studies in children.

AB - Hydroxyurea is the only approved medication in the United States for the treatment of sickle cell anemia (HbSS) and is widely used in children despite an indication limited to adults. We review the evidence of efficacy and safety in children with reference to pivotal adult studies. This evidence and expert opinion form the basis for recommended guidelines for the use of hydroxyurea in children including indications, dosing, therapeutic and safety monitoring, and interventions to improve adherence. However, there are substantial gaps in our knowledge to be addressed by on-going and planned studies in children.

KW - Children

KW - Efficacy

KW - Hydroxyurea

KW - Sickle cell disease

UR - http://www.scopus.com/inward/record.url?scp=84862254136&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=84862254136&partnerID=8YFLogxK

U2 - 10.1002/pbc.24178

DO - 10.1002/pbc.24178

M3 - Review article

C2 - 22517797

AN - SCOPUS:84862254136

SN - 1545-5009

VL - 59

SP - 365

EP - 371

JO - Pediatric Blood and Cancer

JF - Pediatric Blood and Cancer

IS - 2

ER -

Hydroxyurea for the treatment of sickle cell disease: Efficacy, barriers, toxicity, and management in children

Abstract

Keywords

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this