Morales Luis F, Miyara Santiago J, Guevara Sara, Metz Christine N, Shoaib Muhammad, Watt Stacey, Zafeiropoulos Stefanos, McCann-Molmenti Alexia, Hayashida Kei, Takegawa Ryosuke, Shinozaki Koichiro, Choudhary Rishabh C, Brindley Elena C, Nishikimi Mitsuaki, Kressel Adam M, Alsalmay Yaser M, Mazzotta Elvio A, Cho Young Min, Aranalde Gabriel I, Grande Daniel A, Zanos Stavros, Shore-Lesserson Linda, Becker Lance B, Molmenti Ernesto P
Department of Surgery, North Shore University Hospital, Manhasset, New York.
Elmezzi Graduate School of Molecular Medicine, Manhasset, New York.
Int J Angiol. 2021 Sep 3;33(4):318-321. doi: 10.1055/s-0041-1726366. eCollection 2024 Dec.
We describe the clinical course of a 65-year-old male patient who suffered from hydrocarbon-induced myelodysplasia and was successfully treated with the thrombopoietin receptor agonist (TPO-RA), romiplostim. Myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis, cytopenias, and increased risk of leukemic transformation. Here, we present a clinical vignette of MDS-associated thrombocytopenia refractory to first-line drugs as well as the TPO-RA, eltrombopag. To date, romiplostim is an U.S. Food and Drug Administration (FDA)-approved drug for idiopathic thrombocytopenic purpura and thrombocytopenia secondary to liver disease. Of note, currently the FDA advises against its use in MDS based on previous long-term safety concerns. Since the therapeutic options for thrombocytopenia in MDS patients are sparse, repurposing and reassessing romiplostim in this setting have been the focus of recent studies. At the time of writing, no published double-blind randomized clinical trials have conducted a head-to-head comparison between romiplostim and eltrombopag in thrombocytopenic MDS patients. To the best of our knowledge, for a thrombocytopenic patient in the setting of MDS, this is the first documented report of refractory clinical response after a 2-year use of eltrombopag in which replacement of treatment with romiplostim resulted in sustained physiological counts of thrombocytes within four weeks.
我们描述了一名65岁男性患者的临床病程，该患者患有烃诱导的骨髓增生异常综合征，并成功接受了血小板生成素受体激动剂（TPO-RA）罗米司亭的治疗。骨髓增生异常综合征（MDS）的特征是造血无效、血细胞减少以及白血病转化风险增加。在此，我们展示了一例对一线药物以及TPO-RA艾曲泊帕难治的MDS相关性血小板减少症的临床病例。迄今为止，罗米司亭是一种经美国食品药品监督管理局（FDA）批准用于特发性血小板减少性紫癜和肝病继发血小板减少症的药物。值得注意的是，基于先前的长期安全性担忧，目前FDA建议不要在MDS中使用该药。由于MDS患者血小板减少症的治疗选择有限，在此背景下重新利用和重新评估罗米司亭一直是近期研究的重点。在撰写本文时，尚无已发表的双盲随机临床试验对罗米司亭和艾曲泊帕在血小板减少性MDS患者中进行直接比较。据我们所知，对于一名处于MDS背景下的血小板减少症患者，这是第一份有记录的报告，该患者在使用艾曲泊帕2年后出现难治性临床反应，改用罗米司亭治疗后在四周内血小板计数维持在生理水平。
