TY - JOUR T1 - Hydroxyurea adherence and treatment outcomes in pediatric sickle cell disease: a prospective cohort study A1 - Ali Hassan Najmi A1 - Amira R. Al Darwish A1 - Atheer A. Alshahrani A1 - Tahani Jubran Almalki A1 - Sara Saeed Mahmoud Hassanien A1 - Fawaz Hadi Tawhari A1 - Ayman Elssafy Abualama A1 - Asma M. Ahmed A1 - Sawsan Mohammed Asiri A1 - Amani Saleh Alwadai A1 - Mohammed Ibrahim Hattan A1 - Mohammed Kanan A1 - Abdullah Abdulrahman Najmi A1 - Zohoor Alshahrani A1 - Badriah Gharamah Al Asmari JF - Journal of Advanced Pharmacy Education and Research JO - J Adv Pharm Educ Res SN - 2249-3379 Y1 - 2026 VL - 16 IS - 3 DO - 10.51847/jSTHpjkB4k SP - 82 EP - 91 N2 - Hydroxyurea is the primary treatment for sickle cell anemia. However, its clinical efficacy depends heavily on long-term adherence. This study aimed to evaluate the levels of adherence to hydroxyurea treatment in pediatric sickle cell anemia patients. The study also sought to identify side effects that might necessitate dose reduction or temporary discontinuation of hydroxyurea treatment. A prospective cohort study was conducted over 12 months with 147 children with sickle cell anemia attending Armed Forces hospitals in the Southern Region. Demographic data were obtained from the hospital's information system, along with data on HU dose, treatment duration, laboratory values, and SCD-related complications. Adherence was assessed using the Morisky Modification Adherence Scale (MMAS-8). Statistical analyses were performed using SPSS software. The mean HU dose was 19.8 ± 3.3 mg/kg/day, and 61.2% of patients demonstrated high adherence (MMAS ≥ 8). HbF levels were significantly higher among adherent patients (15.2% vs. 10.7%, p < 0.001). Following initiation of HU therapy, annual transfusions and SCD-related complications decreased significantly (p < 0.001). ROC analysis indicated that HbF (AUC 0.75, 95% CI 0.62–0.87) is a stronger predictor of adherence than MCV (AUC 0.57, 95% CI 0.37–0.77). Adverse effects requiring HU dose adjustment occurred in 12.9% of patients, the most common being neutropenia and elevated transaminase levels. Higher HU adherence is associated with improved hematological parameters, particularly HbF, and reduced morbidity associated with SCD. MCV alone is a suboptimal indicator of adherence compared to HbF. Enhanced adherence monitoring and patient education are essential to improve the therapeutic benefits of HU in pediatric SCD. UR - https://japer.in/article/hydroxyurea-adherence-and-treatment-outcomes-in-pediatric-sickle-cell-disease-a-prospective-cohort-syhpxc0qagngztg ER -