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. 2021 Jun;68(6):e29008.
doi: 10.1002/pbc.29008. Epub 2021 Mar 19.

Implementation of near-universal hydroxyurea uptake among children with sickle cell anemia: A single-center experience

Kristine Karkoska  1 , Kevin Todd  1 , Omar Niss  1   2 , Kelly Clapp  1 , Lynette Fenchel  1 , Theodosia A Kalfa  1   2 , Punam Malik  1   2 , Charles T Quinn  1   2 , Russell E Ware  1   2 , Patrick T McGann  1   2
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Implementation of near-universal hydroxyurea uptake among children with sickle cell anemia: A single-center experience

Kristine Karkoska et al. Pediatr Blood Cancer. 2021 Jun.

Abstract

Background: Without early initiation of disease-modifying therapy, the acute and chronic complications of sickle cell anemia (SCA) begin early in childhood and progress throughout life. Hydroxyurea is a safe and effective medication that reduces or prevents most SCA-related complications. Despite recommendations to prescribe hydroxyurea for all children with SCA as young as 9 months, utilization remains low.

Procedure: We completed a retrospective review of hydroxyurea-prescribing practices and associated clinical outcomes at our institution over a 10-year period before and after the 2014 National Heart, Lung, and Blood Institute (NHLBI) recommendations to use hydroxyurea for all children with SCA.

Results: Hydroxyurea use more than doubled within our pediatric SCA population from 43% in 2010 to 95% in 2019. The age of hydroxyurea initiation was significantly younger during 2014-2019 compared to 2010-2013 (median 2 years vs. 6 years, p ≤ .001). With this change in clinical practice, nearly all (69/71 = 97%) children born after 2013 received disease-modifying therapy by the end of 2019, primarily hydroxyurea (93%). Concurrently, the number of SCA-related admissions significantly decreased from 67/100 patient-years in 2010 to 39/100 patient-years in 2019 (p < .001).

Conclusion: The early and universal prescription of hydroxyurea for children with SCA is the standard of care. Here, we demonstrate that a careful and deliberate commitment to follow this guideline in clinical practice is feasible and results in measurable improvements in clinical outcomes. Our approach and improved outcomes can serve as a model for other programs to expand their hydroxyurea use for more children with SCA.

Keywords: hydroxyurea; pediatrics; prescriptions; sickle cell anemia.

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References

REFERENCES

    1. Brousseau D, Panepinto J, Nimmer M, Hoffmann R. The number of people with sickle-cell disease in the United States: national and state estimates. Am J Hematol. 2010;85:77-78.
    1. Therrell BL Jr, Lloyd-Puryear MA, Eckman JR, Mann MY. Newborn screening for sickle cell diseases in the United States: a review of data spanning 2 decades. Semin Perinatol. 2015;39:238-251.
    1. Piel FB, Patil AP, Howes RE, et al. Global epidemiology of sickle haemoglobin in neonates: a contemporary geostatistical model-based map and population estimates. Lancet. 2013;381:142-151.
    1. Telfer P, Coen P, Chakravorty S, et al. Clinical outcomes in children with sickle cell disease living in England: a neonatal cohort in East London. Haematologica. 2007;92:905-912.
    1. Quinn CT, Rogers ZR, McCavit TL, Buchanan GR. Improved survival of children and adolescents with sickle cell disease. Blood. 2010;115:3447-3452.

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