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

Bibliographic Data

ID15717148
AuthorsGiuseppe Lassandro (0000-0001-7858-1632, Ospedale Pediatrico Giovanni XXIII), Francesco Carriero (0000-0002-4678-8855, University of Bari Aldo Moro), Domenico Noviello (University of Bari Aldo Moro), Valentina Palladino (0000-0002-3976-6463, Ospedale Pediatrico Giovanni XXIII), Giancarlo Vecchio (0000-0001-5462-0027, Ospedale Pediatrico Giovanni XXIII), Maria Felicia Faienza (0000-0002-1899-8337, Ospedale Pediatrico Giovanni XXIII), Paola Giordano (0000-0002-7762-3500, Ospedale Pediatrico Giovanni XXIII, corresponding author)
Year2022
Volume9
Issue12
Pages1839-1839
Publication date2022-11-28
Peer ReviewedYes
Open AccessYes
TypeARTICLE
VenueChildren (JOURNAL)
Journal identifiersISSN: 2227-9067 • E-ISSN: 2227-9067
PublisherMultidisciplinary Digital Publishing Institute (PUBLISHER • CH)
DOI10.3390/children9121839
PMID36553283
OpenAlexW4310211207
LanguageEN
Citations received1

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

Biology · Eltrombopag · Immune thrombocytopenia · Platelet · Romiplostim · Thrombopoietin · Thrombopoietin receptor · Blood groups and transfusion · Immunodeficiency and Autoimmune Disorders · Medicine · Platelet Disorders and Treatments · Internal Medicine · Pediatrics

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