Study

Impact of Early and Universal Hydroxyurea on Cerebrovascular Outcomes in Sickle Cell Disease


Study Dates
2024 - Present
Principal Investigator
Affiliation
Sponsor Site
Children's Hospital of Philadelphia
PEDSnet Study ID
2024.DAIS.PF.CHOP.551

Study to identify the impact of the 2014 NHBLI guidelines recommending early and universal hydroxyurea (HU) on HU prescriptions and cerebrovascular outcomes in a national cohort of children and young adults born between 2009-2023, and to determine the relationship between timely HU initiation before 2 years of age and the occurence of cerebrovascular disease in children and young adults with sickle cell disease.

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Funding

This research was made possible through the generous support of American Society of Hematology.

Creative Commons license

Except where otherwised noted, this item's license is described as a CC-BY 4.0 Attribution license.

Publisher

PEDSnet

Project Description

This study was intended to determine whether timely initiation of hydroxyurea (HU) prevents the development of early cerebrovascular changes in children with sickle cell disease (SCD). This project seeks to establish a link between early HU and cerebrovascular protection, promoting timely HU initiation, reducing the need for CRCT, and lowering stroke rates in children with SCD.

Study Aims

  1. Identify the impact of the 2014 NHLBI guidelines recommending early and universal HU on 1A) HU prescriptions and 1B) cerebrovascular outcomes in a national cohort of children and young adults born between 2009-2023. We hypothesize that the incidence of abnormal TCD and AIS has decreased since 2014, and that this occurred alongside an increase in HU prescriptions.
  2. Determine the relationship between timely HU initiation before 2 years of age and the occurrence of cerebrovascular disease in children and young adults with SCD. We hypothesize that timely HU initiation by 2 years of age leads to a lower risk of abnormal TCD or AIS.

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