Kuwana Masataka, Tomiyama Yoshiaki
Department of Allergy and Rheumatology, Nippon Medical School Graduate School of Medicine, 1-1-5 Sendagi, Bunkyo-ku, Tokyo, Japan.
Department of Blood Transfusion, Osaka University Hospital, Osaka, Japan.
Int J Hematol. 2025 Mar;121(3):356-362. doi: 10.1007/s12185-025-03924-2. Epub 2025 Jan 28.
Fostamatinib had superior efficacy to a placebo and acceptable safety profiles for at least 1 year in a phase 3 study of Japanese patients with primary immune thrombocytopenia. Here, we report the 3-year safety and efficacy of fostamatinib in that study. Data from 33 patients who received at least one dose of fostamatinib were analyzed. A platelet response > 50,000/µL (at two consecutive visits at least 28 days apart while receiving fostamatinib) was achieved in 16 patients (48%). The median total duration of a platelet response > 50,000/µL was 589 (range: 106-1003) days. Gastrointestinal disorders, such as diarrhea, hypertension, and hepatic enzyme elevation, were the most common fostamatinib-related adverse events. Most events occurred within 12 weeks of treatment. No thromboembolisms, treatment-related infections, or moderate or severe treatment-related bleeding events were observed. In summary, this extension study of a clinical trial found a sustained platelet response without new safety signals during 3-year treatment with fostamatinib.
在一项针对日本原发性免疫性血小板减少症患者的3期研究中， fostamatinib在至少1年的时间里疗效优于安慰剂，且安全性良好。在此，我们报告该研究中fostamatinib的3年安全性和疗效。分析了33例接受至少一剂fostamatinib治疗患者的数据。16例患者（48%）实现了血小板反应>50,000/µL（在接受fostamatinib治疗期间，至少间隔28天的两次连续访视时）。血小板反应>50,000/µL的中位总持续时间为589天（范围：106 - 1003天）。胃肠道疾病，如腹泻、高血压和肝酶升高，是最常见的与fostamatinib相关的不良事件。大多数事件发生在治疗的12周内。未观察到血栓栓塞、治疗相关感染或中度或重度治疗相关出血事件。总之，这项临床试验的扩展研究发现，在fostamatinib 3年治疗期间，血小板反应持续存在，且无新的安全信号。
