From delayed recognition to long-term disease control: Eltrombopag in severe MYH9-related disease. (PubMed, Transfus Apher Sci) - Oct 7, 2026 - "Eltrombopag, a thrombopoietin receptor agonist, was administered off-label, resulting in a clinically significant increase in platelet count, subsequent normalization during follow-up, and improvement in the bleeding profile. This case suggests that eltrombopag may represent a potentially effective long-term therapeutic option for patients with severe symptomatic MYH9-RD."
Journal • Cataract • Chronic Kidney Disease • Genetic Disorders • Hematological Disorders • Nephrology • Ophthalmology • Otorhinolaryngology • Renal Disease • Thrombocytopenia • Transplantation
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Francesco Di Raimondo; Gabriella Santuccio; Gaetano Giuffrida; Miriana Carmela Limoli; Stephanie Grasso; Uros Markovic
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