Successful Eltrombopag Therapy in a Child with MYH9-Related Inherited Thrombocytopenia

Overview

Journal Children (Basel)

Specialty Health Services

Date 2022 Dec 23

PMID 36553283

Authors

Giuseppe Lassandro

Francesco Carriero

Domenico Noviello

Valentina Palladino

Giovanni Carlo Del Vecchio

Maria Felicia Faienza

Paola Giordano

Affiliations

Soon will be listed here.

Abstract

Inherited thrombocytopenias represents a heterogenous group of diseases characterized by a congenital reduction in the platelet count that could lead to a bleeding tendency. MYH9-related disorders are characterized by large platelets and congenital thrombocytopenia. Thrombopoietin-receptor agonists: eltrombopag and romiplostim are currently approved in many countries for the treatment of different forms of acquired thrombocytopenia, such as immune thrombocytopenia. We report, instead, the successful use of eltrombopag to treat inherited thrombocytopenia in a patient with an MHY9-related disease. This is the first report of a chronic use of eltrombopag to elevate platelets in MYH9-related disorders without side effects.

Citing Articles

Successful use of thrombopoietin analogs in thrombocytopenia associated with MYH-9 mutation.

Louzao M, Vargas M, Meseguer E, Pedrote Amador B, Perez Simon J, Mingot-Castellano M Ann Hematol. 2023; 102(11):3283-3284.

PMID: 37610462 DOI: 10.1007/s00277-023-05386-0.

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