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Sickle cell anemia (SCA) is a severe genetic disorder characterized by the production of abnormal hemoglobin S, leading to the formation of sickle-shaped red blood cells that cause chronic anemia, pain, and organ damage. This review explores recent innovative strategies aimed at improving survival rates and quality of life for SCA patients. Genetic therapies, particularly gene editing with CRISPR-Cas9 and gene therapy using lentiviral vectors, have shown significant potential in correcting the genetic defects responsible for SCA. Clinical trials demonstrate that these approaches can reduce sickle cell crises and minimize the need for blood transfusions by enabling the production of healthy red blood cells. Novel pharmacological treatments such as voxelotor, crizanlizumab, and L-glutamine provide additional mechanisms to prevent hemoglobin polymerization, reduce vaso-occlusive episodes, and decrease oxidative stress, respectively. These therapies offer new hope for patients, particularly those who do not respond adequately to existing treatments. Improved blood transfusion protocols, including automated red cell exchange and advanced donor-matching techniques, have enhanced the safety and efficacy of transfusions, reducing complications like alloimmunization. Comprehensive care models, integrating multidisciplinary care teams, patient education, and telemedicine, have further contributed to better disease management. By providing holistic care that addresses both medical and psychosocial needs, these models improve patient adherence to treatment and overall health outcomes. This review highlights the importance of these innovative strategies and calls for continued research and development to sustain and expand these advancements in SCA care.
Obeagu et al. (Wed,) studied this question.