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

Abstract	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.
Authors	Page Clemons Bankston, Rami A Al-Horani
Journal	International journal of molecular sciences (Int J Mol Sci) Vol. 20 Issue 12 (Jun 20 2019) ISSN: 1422-0067 [Electronic] Switzerland
PMID	31226783 (Publication Type: Journal Article, Review)
Chemical References	Aminopyridines Cinnamates Morpholines Oxazines Pyridines Pyrimidines Receptors, Thrombopoietin Small Molecule Libraries Thiazoles Thiophenes avatrombopag lusutrombopag Syk Kinase fostamatinib
Topics	Aminopyridines Animals Cinnamates (chemistry, pharmacology, therapeutic use) Drug Development Humans Morpholines Oxazines (chemistry, pharmacology, therapeutic use) Pyridines (chemistry, pharmacology, therapeutic use) Pyrimidines Receptors, Thrombopoietin (agonists, metabolism) Small Molecule Libraries (chemistry, pharmacology, therapeutic use) Syk Kinase (antagonists & inhibitors, metabolism) Thiazoles (chemistry, pharmacology, therapeutic use) Thiophenes (chemistry, pharmacology, therapeutic use) Thrombocytopenia (drug therapy, metabolism)

Join CureHunter, for free Research Interface BASIC access!

Take advantage of free CureHunter research engine access to explore the best drug and treatment options for any disease. Find out why thousands of doctors, pharma researchers and patient activists around the world use CureHunter every day.

Realize the full power of the drug-disease research graph!