Related Experiment Video
Updated: Aug 13, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Impact of Hydroxyurea Starting Dose on Pain Outcomes in Patients with Sickle Cell Disease
Abstract:
In patients with sickle cell disease, hydroxyurea decreases the number of pain crises experienced. This study aimed to evaluate the difference in pain outcomes between patients started on a guideline concordant, weight-based starting dose of at least 15 mg/kg/day of hydroxyurea and those not. The first prescription of hydroxyurea was the baseline date, follow-up was a visit 60-120 days after baseline. The primary outcome was the change in opioid prescribing between baseline and follow-up. 138 patients met inclusion criteria; of these, 55 were started on a guideline concordant dose of hydroxyurea. Greater white blood cell count (9.5 vs 12.0; p < 0.01) was statistically associated with subtherapeutic dosing. Greater actual body weight (68.0 vs 72.1 kg; p = 0.16) also appeared higher in the non-guideline concordant group. No statistically significant difference in opioid prescribing was observed between those started on a guideline concordant dose of hydroxyurea and those who were not. In the guideline concordant starting dose group, 42% had a reduction in pain scores at first follow up, compared to 35% with a non-guideline recommended starting dose. (p = 0.41). While this difference is in the direction that would be expected based on the guidelines, the difference does not appear to be clinically meaningful.
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