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Open AccessReview

New Small Molecule Drugs for Thrombocytopenia: Chemical, Pharmacological, and Therapeutic Use Considerations

Division of Basic Pharmaceutical Sciences, College of Pharmacy, Xavier University of Louisiana, New Orleans, LA 70125, USA
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Int. J. Mol. Sci. 2019, 20(12), 3013; https://doi.org/10.3390/ijms20123013
Received: 28 May 2019 / Revised: 16 June 2019 / Accepted: 18 June 2019 / Published: 20 June 2019
(This article belongs to the Section Molecular Pharmacology)
This review provides details about three small molecules that were recently approved by the FDA for the treatment of thrombocytopenia. The new treatments include lusutrombopag, avatrombopag, and fostamatinib. The first two drugs are orally active thrombopoietin receptor (TPO-R) agonists which are FDA-approved for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure. Fostamatinib is orally active prodrug that, after activation, becomes spleen tyrosine kinase (SYK) inhibitor. Fostamatinib is currently used to treat chronic and refractory immune thrombocytopenia in patients who have had insufficient response to previous treatment. Chemical structures, available dosage forms, recommended dosing, pharmacokinetics, results of toxicity studies in animals, most frequent adverse effects, significant outcomes of the corresponding clinical trials, and their use in specific patient populations are thoroughly described. Described also is a comparative summary of the different aspects of five currently available therapies targeting TPO-R or SYK for the treatment of thrombocytopenia. View Full-Text
Keywords: lusutrombopag; avatrombopag; fostamatinib lusutrombopag; avatrombopag; fostamatinib
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MDPI and ACS Style

Clemons Bankston, P.; Al-Horani, R.A. New Small Molecule Drugs for Thrombocytopenia: Chemical, Pharmacological, and Therapeutic Use Considerations. Int. J. Mol. Sci. 2019, 20, 3013.

AMA Style

Clemons Bankston P, Al-Horani RA. New Small Molecule Drugs for Thrombocytopenia: Chemical, Pharmacological, and Therapeutic Use Considerations. International Journal of Molecular Sciences. 2019; 20(12):3013.

Chicago/Turabian Style

Clemons Bankston, Page; Al-Horani, Rami A. 2019. "New Small Molecule Drugs for Thrombocytopenia: Chemical, Pharmacological, and Therapeutic Use Considerations" Int. J. Mol. Sci. 20, no. 12: 3013.

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