Eltrombopag in preparation for surgery in patients with severe MYH9-related thrombocytopenia

Patients with inherited thrombocytopenias often require platelet transfusions to raise platelet count before surgery or other invasive procedures; moreover, subjects presenting clinically significant spontaneous bleeding may benefit from an enduring improvement of thrombocytopenia. The hypothesis that thrombopoietin-mimetics can increase platelet count in inherited thrombocytopenias is appealing, but evidence is scarce. We conducted a prospective, phase 2 clinical trial to investigate the efficacy of the oral thrombopoietin-mimetic eltrombopag in different forms of inherited thrombocytopenia. We enrolled 24 patients affected with MYH9-related disease, ANKRD26-related thrombocytopenia, X-linked thrombocytopenia/Wiskott-Aldrich syndrome, monoallelic Bernard-Soulier syndrome, or ITGB3-related thrombocytopenia. Average pre-treatment platelet count was 40.4 x109/L. Patients received a 3- to 6-week course of eltrombopag in a dose-escalation manner. Of 23 patients evaluable for response, 11 (47.8%) achieved a major response (platelet count >100 x109/L), 10 (43.5%) a minor response (platelet count at least twice than baseline), whereas 2 patients (8.7%) did not respond. Average increase of platelet count compared to baseline was 64.5 x109/L (p