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Sickle Cell Disease Treatments And Education Term Paper

Sickle Cell Anemia: Ethical Considerations The only known cure for sickle cell disease is hematopoietic stem cell transplant (HSCT). Survival rates after HSCT are high, almost 100%, with cure rates of more than 90% (Nickel, Hendrickson & Haight, 2014; Nickel & Kamani, 2017). To receive HSCT most successfully, the donor is ideally a HLA-identical sibling. This raises several ethical concerns. The most pressing of all ethical concerns related to HSCT is whether the procedure should be offered to children with “less severe” cases of sickle cell disease (Nickel, Hendrickson & Haight, 2014; Nickel & Kamani, 2017). Less severe cases have been defined as those that do not have overt complications. However, Nickel, Hendrickson & Haight, (2014) point out that many “less severe” cases may become more severe over time, compelling healthcare workers to offer HSCT to all children with sickle cell disease. The same issue of access to HSCT is related to adult cases of sickle cell, cases from low income countries, or cases where the potential recipient has “social problems” that might significantly impact health outcomes (Nickel & Kamani, 2017). Another potential ethical issue has to do with the deliberate use of reproductive technologies to create the HLA-identical donor (Nickel & Kamani, 2017). Healthcare workers should take care to preserve...

Resources are scarce with HSCT, until new research in cryobiology and cryopreservation reduces the ethical and financial burden, as well as the physical and psychological burden of donor transplants.
Genetics for Improving Health Outcomes

Sickle cell disease is “characterized by a single point mutation in the seventh codon of the ?-globin gene,” (Hoban, Cost, Mendel, et al, 2015, p. 2597). As a genetic condition, sickle cell disease is responsive to genetic interventions. Few have been widely tested. One study shows the potential efficacy for zinc finger nucleases (ZFNs) to “precisely cleave and disrupt the erythroid enhancer of the BCL11A gene,” (Holmes, Reik, Rebar, et al, 2017, p. 2066). However, few studies offer promising solutions for reducing overall costs of treatment, with HSCT remaining the only viable intervention. The cultivation of specialized stem cells for the treatment of sickle cell disease may be on the horizon, but in the meantime, cryopreservation of patients’ oocytes is a means of at least preserving the patient’s fertility after treatment for the disease (Lavery, Islam, Hunt, et al, 2016). Genetic testing may also help reduce…

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References

Hoban, M.D., Cost, G. J., Mendel, M, C., et al (2015). Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells. Blood 2015(125): 2597-2604.

Holmes, M.C., Reik, A., Rebar, E.J., et al (2017). A potential therapy for beta-thalassemia (ST-400) and sickle cell disease. Blood 2017(130): 2066.

Lavery, S.A., Islam, R., Hunt, J., et al (2016). The medical and ethical challenges of fertility preservation in teenage girls. Human Reproduction 31(7): 1501-1507.

Nickel, R.S., Hendrickson, J.E. & Haight, A.E. (2014). The ethics of a proposed study of hematopoietic stem cell transplant for children with “less severe” sickle cell disease. Blood 2014(124): 861-866.

Nickel, R.S. & Kamani, N. (2017). The ethics of hematopoietic stem cell transplantation for sickle cell disease. In Meier E., Abraham A., Fasano R. (eds) Sickle Cell Disease and Hematopoietic Stem Cell Transplantation, Springer, pp. 199-219.


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