Management and Treatment of Sickle Cell Disease - Essay Example

Thesis ment: Better understanding of Sickle Cell Disease has led to improved management and treatment strategies of Sickle Cell Disease. Introduction: Sickle cell disease is an inherited disease that causes the normally round and flexible red blood cells to become stiff and become shaped like a sickle, from whence it derives its name. The stiff sickle shaped blood cells no longer flow through blood vessels easily, causing blockage in the blood supply to the tissues. This lack of blood supply is responsible for the characteristic pain experienced by patients suffering from sickle cell disease, and is also responsible for organ damage in sickle cell disease. These sickle shaped blood cells die much faster than normal red blood cells, and this could result in anemia. (1). The incidence of sickle cell disease in the United States of America is such that almost one in every three hundred and fifty new born African- American infants are afflicted by the disease, and this has led to nearly 72,000 individuals in the United States of America to be afflicted with sickle cell disease. This incidence of sickle cell disease in the United States of America has made it the most prevalent genetic hematologic disorder in the country. (2). The Need for Better Understanding of Sickle Cell Disease: The socio-economic aspects of sickle cell disease led to it being considered for long as an exotic disease, leading to unsatisfactory management and treatment of the disease, as there was no proper understanding of the disease. This was made worse by the disease manifesting itself with different symptoms in different patients. Some patients would report acute pain, while multiple and chronic organ alterations leading to death would be seen in some of the victims of the disease. For more effective management of the disease it became necessary to have a better understanding of the disease, and dissemination of this information to the physician. (3). Political Action for Better Understanding of Sickle Cell Disease: The Congress passed the National Sickle Cell Disease Control Act more than three decades ago in 1972. This landmark legislation was responsible for the creation of the National Sickle Cell Disease Program, which made it mandatory to provide funds for scientific research programs for a better understanding of sickle cell disease, so that the care and quality of life of patients with sickle cell disease could be improved. The National Heart, Lung, and Blood Institute has had the responsibility of organizing and funding research programs on sickle cell disease, since the establishment of the National Sickle Cell Disease Program. (2). The Better Understanding of Sickle Cell Disease: Considerable progress has been achieved over the last few decades in understanding the natural history of sickle cell disease. This has resulted in more efficient management approaches that have targeted the treatment and even the prevention of certain complications that are a consequence of the disease. (4). Pain in sickle cell disease is responsible for debilitating many of the patients. This debilitating pain in sickle cell disease is responsible for 75,000 hospitalizations every year in the United States of America, and costs $475 million in terms of health care expenditure every year. The understanding of the pathophysiology and presentation of pain in sickle cell disease, as an example, provides clarity of the better levels of understanding of sickle cell disease. Occlusion of the vascular beds by distorted erythrocytes causes tissue ischemia, which is responsible for the pain experienced by sickle cell patients. In hypoxic conditions, hemoglobin S (HbS) in the erythrocytes of patients afflicted with sickle cell disease reacts to form polymers, which are rigid. This causes the distortion of the erythrocytes. At the same time polymorphonuclear leukocytes disperse cytokines, which lead to the formation of adhesion molecules on the endothelium of the blood vessels. These receptors capture passing erythrocytes. This abnormal binding of the erythrocytes to the walls of the blood vessels narrows the vascular lumen, and leads to trapping of more cells, thus creating a hypoxic environment encouraging further distortion of the erythrocytes. Added to this, and enhancing the hypoxic situation in the blood vessels is the release of vasoconstrictors, as a result of the interaction between the erythrocytes and the endothelium of the blood of the vessels. This enhances the restriction of regional blood flow. The consequences of this cycle of action and reaction results in the occlusion of vascular beds most often seen in vertebra, femur, hip, and ribs. The regional hypoxia that results from the occlusion of the vascular beds could lead to serious complications like vascular necrosis of the bone marrow or infarct of tissues. The acute inflammatory response to tissue injury is the major cause of pain seen in patients with sickle cell disease. (5). Better Management and Treatment as a Result of Better Understanding: The old management and treatment of sickle cell disease consisted of increasing the affinity of HbS for oxygen, and inhibiting the release of oxygen. The drugs used for this purpose were found to be to be ineffective, and produced serious side effects in the patients. Better understanding of the pathophysiology and presentation of sickle cell disease has enabled development of more efficient management and treatment pathways for sickle cell disease. The first pathway uses the strategy of reducing HbS concentrations through the increase of other hemoglobin chains. The increase of the synthesis of gamma chains and thereby the formation of hemoglobin F is achieved by activating the specific promoters, or through inducing the production of precursors that are capable of synthesizing gamma chains. This pathway uses the administration of butyrate and arginine to achieve the management strategy. This strategy has been most effective in improving the condition of the patients with sickle cell disease. A second approach to the management and treatment of sickle cell disease has been to decrease the adhesive properties of the HbS red blood corpuscles, and thereby reduce their adhesion to the walls of the blood vessels, which results in regional hypoxia. This approach is achieved through the administration of hydroxyurea, which causes reduction in granulocytes. The significant factor of both these present day approaches in the management and treatment of sickle cell disease is that the strategies are based on the efforts that have brought a better understanding of the sickle cell disease. (6). Conclusion: Management and treatment of sickle cell disease suffered from a proper understanding of the disease. The efforts over the last three decades have led to a better understanding. It is this understanding of the disease that has led to improved management and treatment strategies for sickle cell disease. Works Cited 1. “Sickle Cell Disease”. March of Dimes. 2004. 15 Oct. 2006 http://www.marchofdimes.com/professionals/681_1221.asp. 2. Bonds, D.R. “Three decades of innovation in the management of sickle cell disease: the road to understanding the sickle cell disease clinical phenotype”. Blood reviews 19.2 (2005): 99-110. 3. Gulbis, B., et al. “Sickle cell disease: exotic disease or a Belgian public health problem?” Revue medicale de Bruxelles 26.4 (2005): S309-313. 4. Buchanan, G.R., et al. “Sickle cell disease”. Hematology (Am Soc Hematol Educ Program) (2004): 35-47. 5. Marlowe, F., Karen et al. “Treatment of Sickle Cell Pain”. Pharmacotherapy 22.4 (2002): 484-491 6. Armandola, A. E. “Management of Sickle Cell Anemia: New Approaches”. Medscape Today. 15 Oct. 2006 http://www.medscape.com/viewarticle/437504. . Read More