Merli Pietro, Strocchio Luisa, Vinti Luciana, Palumbo Giuseppe, Locatelli Franco
a 1 Department of Pediatric Hematology-Oncology, IRCCS, Bambino Gesù Children's Hospital , Rome, Italy +39 06 68 59 26 78 ; +39 06 68 59 22 92 ;
b 2 University of Pavia, Department of Pediatrics , Pavia, Italy.
Expert Opin Pharmacother. 2015;16(14):2243-56. doi: 10.1517/14656566.2015.1085512.
Eltrombopag is an orally bioavailable, non-peptide thrombopoietin receptor agonist capable of stimulating platelet production through the differentiation of CD34+ hematopoietic progenitor cells into committed CD41+ megakaryocyte precursors and proliferation of megakaryocyte progenitor cells.
This drug has been tested in several clinical trials in adult patients with chronic immune thrombocytopenia (ITP), demonstrating the ability of the drug to reduce the burden of thrombocytopenia and its associated side effects. Two multicenter trials on eltrombopag in chronic ITP of childhood have been recently completed, showing that the drug is effective also in pediatric patients. Recent studies have suggested a potential role of eltrombopag in the treatment of thrombocytopenia associated with hepatitis-C virus infection. These studies have documented that adjunct treatment with eltrombopag can help avoid either dose reductions or withdrawal of pegylated interferon due to development of thrombocytopenia. Eltrombopag has shown efficacy also in patients with acquired severe aplastic anemia and myelodysplastic syndromes.
Eltrombopag plays an important therapeutic role in many different conditions characterized by persistent thrombocytopenia. A more comprehensive definition of both long-term safety and benefits deriving from the use of eltrombopag will be obtained through prolonged observation of patients already enrolled in the different studies conducted so far and from future prospective controlled trials.
艾曲泊帕是一种口服生物可利用的非肽类血小板生成素受体激动剂,能够通过将CD34+造血祖细胞分化为定向的CD41+巨核细胞前体以及使巨核细胞祖细胞增殖来刺激血小板生成。
该药物已在成年慢性免疫性血小板减少症(ITP)患者的多项临床试验中进行了测试,证明该药物有能力减轻血小板减少症的负担及其相关副作用。最近完成了两项关于艾曲泊帕治疗儿童慢性ITP的多中心试验,表明该药物在儿科患者中也有效。最近的研究表明艾曲泊帕在治疗丙型肝炎病毒感染相关的血小板减少症方面具有潜在作用。这些研究记录了使用艾曲泊帕进行辅助治疗有助于避免因血小板减少症的发生而减少聚乙二醇化干扰素的剂量或停药。艾曲泊帕在获得性严重再生障碍性贫血和骨髓增生异常综合征患者中也显示出疗效。
艾曲泊帕在许多以持续性血小板减少为特征的不同病症中发挥着重要的治疗作用。通过对已参与目前进行的不同研究的患者进行长期观察以及未来的前瞻性对照试验,将能更全面地明确使用艾曲泊帕的长期安全性和益处。