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

Dain, Sarah; Bailey, Charles

Study

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

Study Dates

2024

Publisher

PEDSnet

Abstract

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.

Affiliation(s)

Children's Hospital of Philadelphia

Funder(s)

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

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

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.
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.

Cohort Description

Patients ages 0 to 21 years with homozygous sickle cell disease who initiate hematology care at a PEDSnet institution in the first year of life between 2009 and 2023. Patients with a history of malignancy or prematurity (less than 28 weeks) are excluded.