Sickle Cell Anemia
Sickle cell anemia is a genetic disorder of the red blood cells. It is a distressing situation that is triggered by an autosomal recessive genetic hemoglobinopathy, which results in the hallmark clinical sequelae of hemolysis and vascoocclusive phenomena (Al-Salem, 2015). Red blood cells are typically shaped like discs to give them flexibility when traveling through even the smallest of the blood vessels. However, with sickle cell anemia, the red blood cells have an abnormal shape, which is crescent. As a result, they are prone to be trapped in small vessels. They also become rigid and sticky. Consequently, they block blood from reaching the various body parts. This may cause tissue damage and pain.
Hydroxyurea is a drug which represents a significant breakthrough in dealing with sickle cell anemia. Besides, this drug is the only one approved by the US FDA, dubbed the Food and Drug Administration, in treating adults with this sickle cell disease. Also, this drug represents the only available agent who can modify the pathogenesis of the disease and has significantly transformed its treatment. The drug increases HbF in the sickle cell anemia (Steinberg, 2016). The cytotoxic effects of hydroxyurea cause erythroid regeneration. Also, the drug causes myelosuppression, leading to a decrease in the leukocyte counts as well as less inflammation. This will likely lead to a reduction of both vasoocclusion and hemolysis.
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Although the FDA has approved hydroxyurea, there are patients with intolerance and nonresponders. The likely reason for this may be associated with the patient’s genetic makeup. One of the evidence for this is the hereditary component in the HbF induction with hydroxyurea treatment. One study found out two SNPs, dubbed as single-nucleotide polymorphisms that were confirmed to correlate with the hydroxyurea response (Costa & Fertrin, 2016). Some of the most common side effects include hyperpigmentation, cytopenias, and weight gain, among others. To control these side effects, it is essential for the patients' genetic makeup to be ascertained before prescribing the drugs.
References
Al-Salem, A. (2015). Hematopoietic Stem Cell Transplantation for Patients with Sickle Cell Anemia. Medical and Surgical Complications of Sickle Cell Anemia , 339-349. doi:10.1007/978-3-319-24762-5_22
Costa, F. F., & Fertrin, K. Y. (2016). Clinical Manifestations and Treatment of Adult Sickle Cell Disease. Sickle Cell Anemia , 285-318. doi:10.1007/978-3-319-06713-1_12
Steinberg, M. H. (2016). Overview of Sickle Cell Anemia Pathophysiology. Sickle Cell Anemia , 49-73. doi:10.1007/978-3-319-06713-1_3
