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The pathogen that causes the disease spends part of its cycle in the red blood cells and triggers an abnormal drop in oxygen levels in the cell. In carriers, this drop is sufficient to trigger the full sickle-cell reaction, which leads to infected cells being rapidly removed from circulation and strongly limiting the infection's progress.
This would be similar to the hemoglobin allele which is responsible for sickle-cell disease, but solely in people with two copies; those with just one copy of the allele have a sickle cell trait and gain partial immunity to malaria as a result. This effect is called heterozygote advantage. [27]
Sickle hemoglobin (HbS) is the most common variant of hemoglobin and arises due to an amino acid substitution in the beta-globin subunit at the sixth residue from glutamic acid to valine. There are different forms of sickle cell disease. HB SS which is the most common and severe form of sickle cell.
Indications for red blood cell transfusion. Transfusion therapy for sickle-cell disease entails the use of red blood cell transfusions in the management of acute cases of sickle cell disease and as a prophylaxis to prevent complications by decreasing the number of red blood cells (RBC) that can sickle by adding normal red blood cells.
It has a safe profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity. [9] It was developed by Global Blood Therapeutics, a subsidiary of Pfizer. [10] In November 2019, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease for those twelve years of age and older.
In the UK, exagamglogene autotemcel is indicated for the treatment of transfusion-dependent beta thalassemia and sickle cell disease in patients aged 12 years and older who should be treated with hematopoietic stem cell transplantation but for whom a suitable stem cell donor is not available.
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