DeRemer David L, Katsanevas Katerina, Bradley Amber, Awan Farrukh T
University of Georgia College of Pharmacy, Georgia Health Sciences University Cancer Center, GA, USA.
J Oncol Pharm Pract. 2013 Dec;19(4):369-72. doi: 10.1177/1078155212469640. Epub 2013 Jan 4.
Romiplostim, a thrombopoietin mimetic, is FDA approved for the treatment of thrombocytopenia in patients with refractory immune thrombocytopenia. Given the success of thrombopoietin agonists in the treatment of immune thrombocytopenia, data in other clinical settings are emerging. In the bone marrow transplant setting, secondary failure of platelet recovery and persistent thrombocytopenia are associated with increased mortality. In this case, romiplostim was attempted in a chronic lymphocytic leukemia patient who underwent a matched, unrelated donor, non-myeloablative stem cell transplant. This patient experienced profound thrombocytopenia despite dose escalation. Anti-thrombopoietin or anti-romiplostim antibodies were not detected by specific assays. Furthermore, the patient remains in disease remission with full chimerism. This case demonstrates that heavily pre-treated patients with limited bone marrow reserve may not respond to thrombopoietin agonists.
罗米司亭是一种血小板生成素模拟物，已获美国食品药品监督管理局（FDA）批准用于治疗难治性免疫性血小板减少症患者的血小板减少症。鉴于血小板生成素激动剂在治疗免疫性血小板减少症方面取得的成功，其他临床环境中的数据也不断涌现。在骨髓移植环境中，血小板恢复的继发性失败和持续性血小板减少与死亡率增加相关。在这种情况下，对一名接受了匹配的无关供体非清髓性干细胞移植的慢性淋巴细胞白血病患者尝试使用了罗米司亭。尽管剂量增加，该患者仍出现严重的血小板减少症。通过特定检测未检测到抗血小板生成素或抗罗米司亭抗体。此外，该患者仍处于疾病缓解状态，且具有完全嵌合现象。该病例表明，经过大量预处理且骨髓储备有限的患者可能对血小板生成素激动剂无反应。
