Advances in transfusion therapy of packed red blood cells in patients with sickle cell disease, complementary pharmacology and emerging treatments
DOI:
https://doi.org/10.56294/saludcyt20251249Keywords:
SICKLE CELL, BLOOD TRANSFUSIONS, HYDROXYUREA, L-GLUTAMINE, CRIZANLIZUMAB, EMERGING THERAPIESAbstract
Introduction: sickle cell disease is a hemoglobinopathy caused by a point mutation in the HBB gene, which generates hemoglobin S (HbS), the polymerization of which induces sickle cell deformation of red blood cells. This causes severe anemia, vaso-occlusive crises, and organ damage. Transfusion therapy with red blood cell concentrates is essential to mitigate these complications and improve tissue oxygenation. Pharmacological treatments, such as hydroxyurea, L-glutamine, voxeletor, and crizanlizumab, increase hemoglobin production, reducing crises. In addition, emerging therapies such as gene editing and hematopoietic stem cell transplants seek a definitive curative solution, marking significant advances in the management of the disease. Methods: bibliographic search of articles in Spanish and English published between 2018-2024 in databases such as Pubmed, SciELO, Scopus, Web of Science. Results: transfusion methods such as erythrocytapheresis, exchange transfusion and simple transfusion were found to allow the management of sickle cell disease, in addition to drugs such as hydroxyurea, L-glutamine, voxelotor, crizanlizumab that help improve the quality of life of the patient, each type of treatment with its advantages and disadvantages. Likewise, a brief description of stem cell therapy and gene therapy was made, which are a potential future cure for sickle cell disease. Conclusions: transfusion therapy remains essential to treat sickle cell disease, while pharmacological advances have demonstrated efficacy in several clinical manifestations of the disease, including vaso-occlusive crises; however, none has managed to completely replace transfusions. Stem cell treatment may be a curative option, its limitations are compatibility and accessibility; likewise, gene therapy is a promise for the future that is still in the research stage
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