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Journal of Thrombosis and Haemostasis
Article . 2018 . Peer-reviewed
License: Elsevier Non-Commercial
Data sources: Crossref
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Thrombopoietin receptor agonists in hereditary thrombocytopenias

Authors: Rodeghiero, F.; Pecci, A.; Balduini, C. L.;

Thrombopoietin receptor agonists in hereditary thrombocytopenias

Abstract

Hereditary thrombocytopenias (HTPs) constitute a heterogeneous group of diseases characterized by a reduction in platelet count and a potential bleeding risk. As a result of advances in diagnostic methods, HTPs are increasingly being identified, and appear to be less rare than previously thought. Most HTPs do not have effective treatments, except for platelet transfusion when bleeding occurs and in preparation for procedures associated with a risk of bleeding. Preliminary clinical evidence suggests that thrombopoietin receptor agonists (TPO-RAs) with an established use in the treatment of certain acquired thrombocytopenias are well tolerated and provide clinical benefits in patients with some forms of HTP. These drugs may therefore be considered for the treatment of HTPs in clinical practice. However, caution and close monitoring are recommended, owing to the absence of long-term safety data and the potential risks posed by prolonged bone marrow stimulation in certain HTPs. In this review, we summarize the available clinical data on TPO-RAs in the treatment of HTPs, and discuss their use in patients with these disorders. We believe that TPO-RAs will play a major role in the treatment of HTPs, particularly myosin heavy chain 9-related disease, Wiskott-Aldrich syndrome, X-linked thrombocytopenia, and thrombocytopenia caused by THPO mutations.

Country
Italy
Keywords

Risk, Recombinant Fusion Proteins, 610, thrombocytopenia, Receptors, Fc, Bernard–Soulier syndrome, MYH9-related disease, Benzoates, Thrombopoiesis, Genetic Heterogeneity, Clinical Trials, Phase II as Topic, 616, Humans, Thrombophilia, Genetic Predisposition to Disease, Genetic Association Studies, Purpura, Thrombocytopenic, Idiopathic, Clinical Trials, Phase I as Topic, romiplostim, Hematology, Thrombocytopenia, Hydrazines, Thrombopoietin, Primary Myelofibrosis, Hematologic Neoplasms, Pyrazoles, eltrombopag, Receptors, Thrombopoietin

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    popularity
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    influence
    This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
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selected citations
These citations are derived from selected sources.
This is an alternative to the "Influence" indicator, which also reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Citations provided by BIP!
popularity
This indicator reflects the "current" impact/attention (the "hype") of an article in the research community at large, based on the underlying citation network.
BIP!Popularity provided by BIP!
influence
This indicator reflects the overall/total impact of an article in the research community at large, based on the underlying citation network (diachronically).
BIP!Influence provided by BIP!
impulse
This indicator reflects the initial momentum of an article directly after its publication, based on the underlying citation network.
BIP!Impulse provided by BIP!
38
Top 10%
Top 10%
Top 10%
bronze