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New agents for sickle cell disease: patient perceptions of benefit in the real world
  1. Robert T Means
  1. Internal Medicine and Pathology, James H Quillen College of Medicine, East Tennessee State University, Johnson City, Tennessee, USA
  1. Correspondence to Dr Robert T Means, Internal Medicine, James H Quillen College of Medicine, East Tennessee State University, Johnson City, TN 37614, USA; meansr{at}

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Sickle cell disease (SCD) is one of the most important monogeneic diseases in the world, causing significant morbidity and mortality and consuming substantial healthcare resources.1 SCD results from a single amino acid substitution in the β-globin chain, leading to sickle hemoglobin (HbS). In oxygen-limited settings, HbS forms polymers that lead to the characteristic sickle-shaped erythrocytes and also to changes in the erythrocyte membrane. The pathological features of SCD result from a combination of vaso-occlusive events precipitated both by abnormal erythrocyte morphology and the increased expression of adhesion molecules on its outer membrane; accelerated hemolysis that can deplete nitric oxide and that leads to anemia; and a high level of endogenous inflammatory activation associated …

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  • Contributors RTM is the sole contributor of this work.

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Competing interests RTM is an Associate Editor for the Journal of Investigate Medicine. No other competing interests declared.

  • Provenance and peer review Commissioned; internally peer reviewed.

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