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Eltrombopag for the treatment of inherited thrombocytopenias: a phase II clinical trial

Haematologica (Roma), 2020-03, Vol.105 (3), p.820-828 [Peer Reviewed Journal]

Copyright© 2020 Ferrata Storti Foundation. ;Copyright© 2020 Ferrata Storti Foundation 2020 ;ISSN: 0390-6078 ;EISSN: 1592-8721 ;DOI: 10.3324/haematol.2019.223966 ;PMID: 31273088

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  • Title:
    Eltrombopag for the treatment of inherited thrombocytopenias: a phase II clinical trial
  • Author: Zaninetti, Carlo ; Gresele, Paolo ; Bertomoro, Antonella ; Klersy, Catherine ; De Candia, Erica ; Veneri, Dino ; Barozzi, Serena ; Fierro, Tiziana ; Alberelli, Maria Adele ; Musella, Valeria ; Noris, Patrizia ; Fabris, Fabrizio ; Balduini, Carlo L ; Pecci, Alessandro
  • Subjects: Benzoates - adverse effects ; Humans ; Hydrazines - adverse effects ; Prospective Studies ; Pyrazoles ; Thrombocytopenia - drug therapy
  • Is Part Of: Haematologica (Roma), 2020-03, Vol.105 (3), p.820-828
  • Description: Patients with inherited thrombocytopenias often require platelet transfusions to raise their platelet count before surgery or other invasive procedures; moreover, subjects with 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 II clinical trial to investigate the efficacy of the oral thrombopoietin-mimetic eltrombopag in different forms of inherited thrombocytopenia. We enrolled 24 patients affected by -related disease, -related thrombocytopenia, X-linked thrombocytopenia/ Wiskott-Aldrich syndrome, monoallelic Bernard-Soulier syndrome, or -related thrombocytopenia. The average pre-treatment platelet count was 40.4 ×10 /L. Patients received a 3- to 6-week course of eltrombopag in a dose-escalated manner. Of 23 patients evaluable for response, 11 (47.8%) achieved a major response (platelet count >100 ×10 /L), ten (43.5%) had a minor response (platelet count at least twice the baseline value), and two patients (8.7%) did not respond. The average increase of platelet count compared to baseline was 64.5 ×10 /L ( <0.001). Four patients with clinically significant spontaneous bleeding entered a program of long-term eltrombopag administration (16 additional weeks): all of them obtained remission of mucosal hemorrhages, with the remission persisting throughout the treatment period. Treatment was globally well tolerated: five patients reported mild adverse events and one patient a moderate adverse event. In conclusion, eltrombopag was safe and effective in increasing platelet count and reducing bleeding symptoms in different forms of inherited thrombocytopenia. Despite these encouraging results, caution is recommended when using thrombopoietinmimetics in inherited thrombocytopenias predisposing to leukemia. ClinicalTrials.gov identifier: NCT02422394.
  • Publisher: Italy: Ferrata Storti Foundation
  • Language: English
  • Identifier: ISSN: 0390-6078
    EISSN: 1592-8721
    DOI: 10.3324/haematol.2019.223966
    PMID: 31273088
  • Source: DOAJ Directory of Open Access Journals
    MEDLINE
    PubMed Central
    Alma/SFX Local Collection
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