Understanding The Treatment Of Sickle Cell Disease With Hydroxyurea

719 Words Aug 25th, 2016 3 Pages
Understanding the treatment of sickle cell disease with hydroxyurea (HU) is an ever evolving topic as new mechanisms that are affected by HU have and continue to be discovered since it became the only FDA-approved drug for this disease. However, there are many physicians around the world that underutilize this drug. While it may be due to the potential adverse effects that are associated with the use of HU, education on all the affected mechanisms may sway the physicians thinking if they can be controlled.
Since the “sickle” shape of the cells is due to a point mutation and the genome of the infected cells can’t be changed we must look into the biochemical pathways that HU affects. Throughout the literature the main theme has been discussing the ways in which HU upregulates the production of fetal hemoglobin (HbF) by maintaining a high level γ-globin. This increased production of HbF diminishes the polymerization of sickle globin in vitro which in turn will reduce the clinical morbidities. Along the lines of making HU “safer” to use, researchers are delving deeper into the mechanisms of action to see if they can be replicated in drugs that do not carry the same side effect burden. This does, however, bring to fruition a deep dive into the biochemical pathways that HU effects. Pule et al. expanded on the knowledge base surrounding how HU induces the γ-globin expression in cord blood cells. While this does not combat the β6 point mutation, the γ-globin upregulates the…

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