A Retrospective Analysis of Sociodemographic and Hematologic Characteristics Associated with Achieving Optimal Hydroxyurea Therapy in Children with Sickle Cell Disease

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George P.E.
Bazo-Alvarez J.C.
Sheehan V.A.
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Lippincott Williams and Wilkins
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Hydroxyurea (HU) has proven hematologic and clinical benefits, especially when escalated to the maximum tolerated dose (MTD). We reviewed clinical data from patients with sickle cell disease (January 2011 to 2016) to determine baseline sociodemographic and laboratory parameters associated with reaching HU MTD without significant delays. In total, 210 patients (mean HU start age, 6.6 y) were included. Initial Kaplan-Meier event analysis showed 1 year to be an inflection point for reaching MTD. In total, 116 patients (55%) reached MTD in <1 year, with 56 (27%) taking >1 year to reach MTD and 38 (18%) patients not successfully reaching MTD during follow-up. In both crude and adjusted analyses, age at HU start was found to be significantly and inversely associated with reaching MTD within 1 year. The data presented, specifically the inflection point of reaching MTD at 1 year and the association of young HU start age with reaching MTD within a year, suggest that successful achievement of MTD may be facilitated by starting patients on HU at a young age and that older patients should receive additional intervention to attain MTD within 1 year. Patients who do not achieve MTD within a year may need the most extensive intervention.
First, we would like to acknowledge and thank all the patients for willingly participating in medical research and making advancement in the care of patients with sickle cell disease possible. VAS is supported by an American Society of Hematology Scholar Award, and National Heart, Lung and Blood Institute, 1K08DK110448-01. JCBA has been sponsored by Cienciactiva, an initiative of the Peruvian National Council of Science, Technology and Technological Innovation (CONCYTEC); grant contract number 231-2015-FONDECYT. PEG would also like to thank Dr. Alex George for access to clinical data and help with the study preparation and conceptualization.
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hydroxyurea, hemoglobin, absolute neutrophil count, absolute reticulocyte count, age