WHO世界卫生组织：2026年羟基脲用于儿童镰状细胞病管理的配方产品概况报告（英文版）

This target product profile (TPP) was developed by the World Health Organization to guide the development, manufacturing and procurement of age-appropriate hydroxyurea formulations for the management of sickle cell disease (SCD) in children and adolescents. SCD is one of the most common inherited disorders worldwide and is associated with substantial morbidity and mortality, particularly in low- and middle-income countries where access to diagnosis, treatment and comprehensive care remains li