Comparing and contrasting different treatment modalities of sickle cell disease

Thesis (M.A.)--Boston UniversityPLEASE NOTE: Boston University Libraries did not receive an Authorization To Manage form for this thesis or dissertation. It is therefore not openly accessible, though it may be available by request. If you are the author or principal advisor of this work and would like to request open access for it, please contact us at open-help@bu.edu. Thank you.Sickle cell disease (SCD) is an autosomal recessive disorder affecting over 70,000 people in the United States. Following deoxygenation, red blood cells become deformed and appear in the characteristic sickle-shape. This change in protein structure leads to vasa-occlusive episodes, which may result in a variety of clinical manifestation including, but not limited to, painful crisis, stroke, acute chest syndrome, and/or splenic infarct. Due to the diversity of symptoms, management of this disease can be complex.In SCD, some of the treatment modalities involve controlling infections, pain management, fetal hemoglobin stimulation, blood transfusion, hematopoietic stem cell transplant and potentially gene therapy. This paper discusses the risk and benefits of these different treatment modalities.2031-01-0