Eltrombopag for the treatment of the inherited thrombocytopenia deriving from MYH9 mutations

Alessandro Pecci, Paolo Gresele, Catherine Klersy, Anna Savoia, Patrizia Noris, Tiziana Fierro, Valeria Bozzi, Anna Maria Mezzasoma, Federica Melazzini, Carlo L. Balduini

Research output: Contribution to journalArticlepeer-review


Platelet transfusion is currently the primary medical treatment for reducing thrombocytopenia in patients with inherited thrombocytopenias. To evaluate whether stimulating megakaryopoiesis could increase platelet count in these conditions, we treated patients with a severe thrombocytopenia induced by MYH9 mutations (MYH9-related disease) with a nonpeptide thrombopoietin receptor agonist, eltrombopag. Twelve adult patients with MYH9-RD and platelet counts of less than 50 × 10 9/L received 50 mg of eltrombopag orally per day for 3 weeks. Patients who achieved a platelet count higher than 150 × 10 9/L stopped therapy, those with 100 to 150 platelets × 10 9/L continued treatment at the same eltrombopag dose for 3 additional weeks, while those with less than 100 platelets × 10 9/L increased the eltrombopag dose to 75 mg for 3 weeks. Major responses (platelet count of at least 100 × 10 9/L or 3 times the baseline value) were obtained in 8 patients, minor responses (platelet counts at least twice the baseline value) in 3. One patient did not respond. Bleeding tendency disappeared in 8 of 10 patients with bleeding symptoms at baseline. Mild adverse events were reported in 2 patients. The availability of thrombopoietin mimetics opened new prospects in the treatment of inherited thrombocytopenias. This study is registered at www.clinicaltrials.gov as NCT01133860 (European Union Drug Regulating Authorities Clinical Trials number

Original languageEnglish
Pages (from-to)5832-5837
Number of pages6
Issue number26
Publication statusPublished - Dec 23 2010

ASJC Scopus subject areas

  • Hematology
  • Biochemistry
  • Cell Biology
  • Immunology


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