Treatment of Sickle Cell Anemia: Pharmacological, Transfusion, and Genetic Strategies

• DOI: https://doi.org/10.22533/at.ed.1595332520116

• Palavras-chave: Sickle Cell Anemia; Fetal Hemoglobin; Hydroxyurea; Gene Therapy; Stem Cell Transplantation; Voxelotor; Crizanlizumab; Transfusion Management.

• Keywords: Sickle Cell Anemia; Fetal Hemoglobin; Hydroxyurea; Gene Therapy; Stem Cell Transplantation; Voxelotor; Crizanlizumab; Transfusion Management.

• Abstract:

  Sickle cell disease (SCD) is a prevalent hereditary hemoglobinopathy characterized by hemoglobin S (HbS) polymerization, resulting in chronic hemolysis, vaso-occlusion, and progressive organ damage. This narrative review analyzes current therapeutic strategies, ranging from disease-modifying pharmacological interventions to curative approaches. Hydroxyurea remains the standard of care for fetal hemoglobin (HbF) induction, but new therapies such as L-glutamine (control of oxidative stress), crizanlizumab (anti-adhesion via P-selectin), and voxelotor (inhibition of HbS polymerization) have expanded the clinical arsenal, allowing for the possibility of combination therapies. Transfusion management remains vital for stroke prevention and treatment of acute complications, although it presents challenges such as alloimmunization and iron overload. In the curative field, allogeneic hematopoietic stem cell transplantation (HSCT) is effective but limited by donor availability. Advances in gene therapy (gene addition and editing) offer the promise of a donor-independent "functional cure," although they still face barriers of cost and accessibility. The study concludes that effective management of FA requires a multidisciplinary approach, integrating biomedical innovation with health policies that ensure equitable access.