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. 2018;6(5):329-343.
doi: 10.1080/21678707.2018.1471983. Epub 2018 May 14.

Advances in new drug therapies for the management of sickle cell disease

Kenneth I Ataga  1 , Payal C Desai  1   2
Affiliations

Advances in new drug therapies for the management of sickle cell disease

Kenneth I Ataga et al. Expert Opin Orphan Drugs. 2018.

Abstract

Introduction: Sickle cell disease (SCD) is an orphan disease in the United States, but is highly prevalent worldwide. Only two drugs, hydroxyurea and L-glutamine, are approved for this disease. With an improved understanding of the pathophysiology of SCD as well as the success of several recently approved drugs for other orphan diseases, there is an increased interest in the development of drugs for SCD.

Areas covered: This review summarizes published studies of drug therapies and ongoing trials of novel agents.

Expert opinion: The development of drugs with different mechanisms of action offers opportunities for combination and individualized therapy in SCD. In addition to acute pain crisis, the evaluation of other SCD-related complications, exercise capacity, patient reported outcomes and validated surrogate endpoints are necessary to advance drug development. It is important to involve sites in sub-Saharan Africa and India, which have the highest burden of SCD, in trials of novel therapies.

Keywords: Clinical Trials; Drug Development; Novel Drugs; Sickle cell disease; Treatment.

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Conflict of interest statement

Declaration of Interests KIA has served on clinical advisory boards and/or served as a consultant for Global Blood Therapeutics, Modus Therapeutics, Novartis and Bioverativ. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Figures

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Mechanisms of action of pharmacologic agents that have been evaluated and/or are being tested in sickle cell disease. RBCs - Red Blood cells; HbF – Hemoglobin F; NO – Nitric Oxide; Invariant NKT cells – Invariant Natural Killer T cells. Modified with permission from [173]
See this image and copyright information in PMC

References

    1. Piel FB, Hay SI, Gupta S, et al. Global burden of sickle cell anaemia in children under five, 2010–2050: modelling based on demographics, excess mortality, and interventions. PLoS Med. 2013;10(7):e1001484. doi: 10.1371/journal.pmed.1001484. - DOI - PMC - PubMed
    1. Couque N, Girard D, Ducrocq R, et al. Improvement of medical care in a cohort of newborns with sickle-cell disease in North Paris: impact of national guidelines. Br J Haematol. 2016. June;173(6):927–37. doi: 10.1111/bjh.14015. - DOI - PubMed
    1. Quinn CT, Rogers ZR, McCavit TL, et al. Improved survival of children and adolescents with sickle cell disease. Blood. 2010. April 29;115(17):3447–52. doi: 10.1182/blood-2009-07-233700. - DOI - PMC - PubMed
    1. Telfer P, Coen P, Chakravorty S, et al. Clinical outcomes in children with sickle cell disease living in England: a neonatal cohort in East London. Haematologica. 2007. July;92(7):905–12. - PubMed
    1. Elmariah H, Garrett ME, De Castro LM, et al. Factors associated with survival in a contemporary adult sickle cell disease cohort. Am J Hematol. 2014. May;89(5):530–5. doi: 10.1002/ajh.23683. - DOI - PMC - PubMed

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