Lusutrombopag在慢性肝病和血小板减少症中的应用研究

Introduction to Lusutrombopag and Previous Research Evidence Lusutrombopag is a second-generation, oral TPO receptor agonist. It binds to the transmembrane region of the TPO receptor (c-Mpl), activating the JAK-STAT and MAPK pathways, which promote the differentiation of hematopoietic stem cells into mature megakaryocytes and the proliferation and differentiation of megakaryocytes, thereby increasing platelet counts. Lusutrombopag can be taken orally without dietary restrictions [17], has minimal drug interactions [18], and exhibits low hepatotoxicity and nephrotoxicity, making it safe and well-tolerated even in patients with chronic liver disease classified as Child-Pugh C [19]. In two randomized controlled trials (RCTs), Lusutrombopag significantly increased platelet counts in patients with chronic liver disease and thrombocytopenia, with a range of platelet response rates summarized from three studies, reducing the need for transfusions [20, 21]. Additionally, a meta-analysis found that patients with thrombocytopenia treated with Lusutrombopag had a lower risk of bleeding compared to those treated with other TPO receptor agonists [22]. However, there is currently no clinical research evidence for the use of Lusutrombopag in persistent or chronic primary immune thrombocytopenia. 2. Therefore, a single-center, single-arm exploratory study was conducted to investigate the efficacy and safety of Lusutrombopag in Chinese patients with persistent or chronic primary immune thrombocytopenia who have undergone at least one line of treatment. The study also aimed to adjust the treatment dose through dose titration to achieve the optimal therapeutic effect.