THERAPEUTIC APPROACHES IN SICKLE CELL ANEMIA: MANAGEMENT STRATEGIES AND CLINICAL AND SOCIAL CHALLENGES

Abstract

Sickle cell anemia is a hereditary hemoglobinopathy with high prevalence in Brazil, especially among Black and mixed-race populations, characterized by a mutation in the β-globin gene that results in the formation of abnormal hemoglobin (HbS), leading to red blood cell deformation, vaso-occlusive crises, chronic hemolytic anemia, and multiple organ complications. This study aims to analyze the management strategies and the clinical, social, and structural challenges faced by patients with sickle cell anemia, with an emphasis on the available therapeutic options, access to hematopoietic stem cell transplantation (HSCT), and the distribution of medications such as hydroxyurea, both within the Unified Health System (SUS) and the private healthcare network. This is a literature review study of a qualitative nature, based on scientific articles, guidelines from the Ministry of Health, books, and academic publications. The results indicate that, although HSCT is the only therapeutic alternative with curative potential, access to it is still limited due to the scarcity of specialized centers, difficulties in finding compatible donors, and high costs. Moreover, regional and socioeconomic disparities persist in the provision of medications and the quality of care provided to patients. It is concluded that overcoming these challenges requires investment in public policies, decentralization of services, strengthening of the care network, and promotion of equity in access to therapies.