Date of Award
2027
Document Type
Capstone
Degree Name
Master of Science (MS)
Department
Physician Assistant Studies
Committee Chair
Ashley Kernicky
Abstract
Introduction: Sickle cell disease (SCD) is one of the most common inherited hemoglobinopathies worldwide and is associated with significant chronic morbidity and early mortality in children, underscoring the need for effective long-term disease-modifying therapies. The purpose of this review is to evaluate the current evidence comparing hydroxyurea therapy with chronic blood transfusion therapy in children with sickle cell disease, with particular emphasis on their effectiveness in reducing the frequency of vaso-occlusive crises.
Methods: PubMed was searched with a specific search strategy using key terms “children with sickle cell disease”, “chronic blood transfusion therapy”, and “hydroxyurea therapy”. MeSH terms and Boolean operators were added to produce a final search of “sickle cell disease”, “reduce vaso-occlusive crises”, “hydroxyurea therapy”, and “chronic blood transfusion therapy”.
Results: A review of the literature identified studies that directly addressed the comparative effectiveness of hydroxyurea therapy and chronic blood transfusion therapy in children with sickle cell disease. The evidence included two systematic reviews, two randomized controlled trials, and one large systematic review evaluating hydroxyurea outcomes in more than 3,800 patients. Overall, the evidence consistently demonstrated that hydroxyurea substantially reduces vaso-occlusive crises (VOCs), hospitalizations, and transfusion requirements, whereas chronic blood transfusion therapy remains the standard therapy for primary and secondary stroke prevention in children at highest neurologic risk.
Discussion: Although both hydroxyurea and chronic blood transfusion therapy have demonstrated benefits in children with sickle cell disease, the current evidence does not clearly establish a single superior strategy for reducing recurrent vaso-occlusive crises. Most comparative studies focus on stroke prevention rather than pain-related outcomes, limiting direct evaluation of vaso-occlusive crisis reduction. Consequently, treatment decisions should be individualized based on patient characteristics, stroke risk, clinical presentation, and available healthcare resources.
Recommended Citation
Pertiller SL. Hydroxyurea Versus Chronic Blood Transfusion Therapy for Reducing Vaso-Occlusive Crises in Children With Sickle Cell Disease: A Review of Current Evidence. The PA Department Journal of Medical Science. 2026. https://digitalcommons.gardner-webb.edu/pa-department-journal-of-medical-science/77
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