Capecchi Marco, Serpenti Fabio, Giannotta Juri, Pettine Loredana, Reda Gianluigi, Martinelli Ida, Artoni Andrea, Barcellini Wilma, Fattizzo Bruno
Department of Biomedical Sciences for Health, Università degli Studi di Milano, Milan, Italy.
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy.
Front Oncol. 2021 Sep 28;11:680411. doi: 10.3389/fonc.2021.680411. eCollection 2021.
Since their license in 2008, studies on thrombopoietin receptor agonists (TPO-RAs) are proceeding at a fast pace. Their favorable efficacy and safety profile makes them good candidates for the management of thrombocytopenia in different settings, even beyond their current indications. In the last 10 years, we faced patients with refractory thrombocytopenia that required treatment with off-label TPO-RA, despite the paucity of data in the literature and the possible risks, particularly that of thrombosis. We hereby report our 10-year real-life single-center experience of TPO-RA used off-label. Fourteen patients were divided into three groups according to the etiology of thrombocytopenia: myelodysplastic syndromes, post-transplantation, and lymphoproliferative diseases. Clinical features and results are reported within each group. Overall, TPO-RA proved effective in all these conditions achieving responses also in heavily pretreated patients. The overall response rate (ORR) was 100% in patients with thrombocytopenia after transplantation and in those with lymphoproliferative diseases and 75% in patients with myelodysplastic syndromes. The median duration of therapy was 285 days (range 93-1,513 days). Four patients (29%) discontinued treatment because of lack of response (n=2) or a sustained response (n=2). No grade 3-4 adverse events occurred, particularly no thrombosis. In our real-life experience, TPO-RAs were effective and safe and proved of value in the challenging management of patients with refractory thrombocytopenia associated with different conditions.
自2008年获得许可以来，关于血小板生成素受体激动剂（TPO-RAs）的研究进展迅速。其良好的疗效和安全性使其成为不同情况下血小板减少症治疗的理想选择，甚至超出了其目前的适应证范围。在过去10年中，我们遇到了难治性血小板减少症患者，尽管文献数据匮乏且存在潜在风险，尤其是血栓形成风险，但仍需要使用TPO-RAs进行非适应证治疗。在此，我们报告我们10年单中心使用TPO-RAs进行非适应证治疗的真实世界经验。根据血小板减少症的病因，将14例患者分为三组：骨髓增生异常综合征、移植后和淋巴增殖性疾病。每组均报告了临床特征和结果。总体而言，TPO-RAs在所有这些情况下均被证明有效，即使是在经过大量预处理的患者中也能取得反应。移植后血小板减少症患者和淋巴增殖性疾病患者的总缓解率（ORR）为100%，骨髓增生异常综合征患者为75%。中位治疗持续时间为285天（范围93 - 1513天）。4例患者（29%）因无反应（n = 2）或持续反应（n = 2）而停药。未发生3 - 4级不良事件，尤其是未发生血栓形成。在我们的真实世界经验中，TPO-RAs有效且安全，在难治性血小板减少症合并不同疾病患者的挑战性管理中被证明具有价值。
