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Abstract

Sickle cell disease is a severe genetic blood disorder marked by the production of abnormal hemoglobin (HbS), leading to sickle-shaped red blood cells that obstruct blood flow and cause various problems, such as the increased risk of infection, persistent anemia, acute pain episodes, and organ damage. Roughly 100,000 Americans suffer from SCD, with approximately 40,000 of them being children. Black people have the highest frequency of the disease. There are six Food and Drug Administration (FDA)-approved drugs, hydroxyurea, L-glutamine, crizanlizumab-TMCA, voxelotor, Casgevy, and Lyfgenia, that are used for the prophylaxis and treatment of serious complications of sickle cell disease. Current treatment approaches focus on symptom management, including pain control, hydroxyurea to reduce pain crises, and transfusions for severe anemia. Based on the clinical trial results, L-glutamine and crizanlizumab-TMCA prevent cell damage and hemoglobin sickling by reducing the sickle cell crisis episodes. At the same time, voxelotor improves hemoglobin oxygen-binding capacity in patients with SCD. Novel therapies, such as gene therapy and clustered regularly interspaced short palindromic repeats/CRISPR-associated protein 9 (CRISPR-Cas9) technology, aim to correct the genetic defect. At the same time, stem cell and bone marrow transplants offer potential cures but are limited by the availability of donors and side effects. Ongoing research seeks to enhance treatment options and develop potential cures for SCD. This review attempts to present a comprehensive overview of the current therapeutic approaches and newly developed innovative medicines to combat and potentially eradicate SCD with an emphasis on their mechanisms, efficacy, and clinical implications.

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2025-07-14
2025-09-26

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/content/journals/chddt/10.2174/011871529X364941250627090838
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Updated Review of Current Therapeutic Approaches for the Management of Sickle Cell Disease
Bentham Science Publishers ; https://doi.org/10.2174/011871529X364941250627090838
/content/journals/chddt/10.2174/011871529X364941250627090838
/content/journals/chddt/10.2174/011871529X364941250627090838
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  • Article Type:     Review Article
Keywords: hydroxyurea ; gene therapy ; vaso-occlusion crisis ; blood transfusion ; hemoglobin ; stem cell transplantation ; Sickle cell disease

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