Abstract：
BACKGROUND: Thrombotic microangiopathy (TMA) is associated with carfilzomib, and knowledge of carfilzomib-induced TMA is based mainly on case reports. This study investigated the clinical characteristics of patients with carfilzomib-induced TMA and provided a reference for the rational use of carfilzomib.
METHODS: Reports of carfilzomib-induced TMA were collected for retrospective analysis by searching the Chinese and English databases from inception to January 31, 2024.
RESULTS: Sixty-six patients were included, with a median age of 63 years (range 39, 85). The median time to onset of TMA was 42 days (range 1, 1825) from initial administration, and the median number of cycles was 3 cycles (range 1, 15). Hemolytic anemia was recorded in 64 patients, with a median of 8.3 g/dL (range 4.6, 13). Sixty-three patients had thrombocytopenia with a median of 18 × 109/L (range 1, 139). The median value of increased LDH was 1192 IU/L (range 141, 5378). ADAMTS13 activity was normal in 41 (62.1 %) of the 42 patients. Mutations were found in 9 (13.6 %) of the 15 patients. Fifty-seven patients achieved a clinical response after discontinuing carfilzomib and receiving therapeutic plasma exchange (53.0 %), eculizumab (24.2 %), or hemodialysis (39.4 %).
CONCLUSIONS: Carfilzomib-induced TMA is an important adverse event that should be considered in patients receiving carfilzomib for multiple myeloma with anemia, thrombocytopenia, and acute kidney injury. Withdrawal of carfilzomib and treatment with eculizumab have proven successful in some patients.
METHODS: Reports of carfilzomib-induced TMA were collected for retrospective analysis by searching the Chinese and English databases from inception to January 31, 2024.
RESULTS: Sixty-six patients were included, with a median age of 63 years (range 39, 85). The median time to onset of TMA was 42 days (range 1, 1825) from initial administration, and the median number of cycles was 3 cycles (range 1, 15). Hemolytic anemia was recorded in 64 patients, with a median of 8.3 g/dL (range 4.6, 13). Sixty-three patients had thrombocytopenia with a median of 18 × 109/L (range 1, 139). The median value of increased LDH was 1192 IU/L (range 141, 5378). ADAMTS13 activity was normal in 41 (62.1 %) of the 42 patients. Mutations were found in 9 (13.6 %) of the 15 patients. Fifty-seven patients achieved a clinical response after discontinuing carfilzomib and receiving therapeutic plasma exchange (53.0 %), eculizumab (24.2 %), or hemodialysis (39.4 %).
CONCLUSIONS: Carfilzomib-induced TMA is an important adverse event that should be considered in patients receiving carfilzomib for multiple myeloma with anemia, thrombocytopenia, and acute kidney injury. Withdrawal of carfilzomib and treatment with eculizumab have proven successful in some patients.
摘要：
背景：血栓性微血管病（TMA）与卡非佐米有关，卡非佐米诱导的TMA的知识主要基于病例报告。本研究调查卡非佐米所致TMA患者的临床特点,为合理使用卡非佐米提供参考。
方法：通过检索从开始到2024年1月31日的中文和英文数据库，收集卡非佐米诱导的TMA报告进行回顾性分析。
结果：纳入66例患者，年龄中位数为63岁（范围39、85）。从初次给药开始，TMA的中位发病时间为42天（范围1，1825），中位周期数为3个周期（范围1、15）。64例患者发生溶血性贫血,中位数为8.3g/dL（范围4.6，13）。63例患者出现血小板减少症，中位数为18×109/L（范围1，139）。LDH增加的中位数为1192IU/L（范围141，5378）。42例患者中有41例(62.1%)ADAMTS13活性正常。15例患者中有9例（13.6%）出现突变。57例患者在停用卡非佐米并接受治疗性血浆置换后达到临床反应（53.0%），依库珠单抗（24.2%），或血液透析（39.4%）。
结论：卡非佐米诱导的TMA是一种重要的不良事件，在接受卡非佐米治疗的多发性骨髓瘤伴贫血患者中应考虑。血小板减少症,和急性肾损伤。在一些患者中，卡非佐米的戒断和依库珠单抗的治疗已被证明是成功的。
方法：通过检索从开始到2024年1月31日的中文和英文数据库，收集卡非佐米诱导的TMA报告进行回顾性分析。
结果：纳入66例患者，年龄中位数为63岁（范围39、85）。从初次给药开始，TMA的中位发病时间为42天（范围1，1825），中位周期数为3个周期（范围1、15）。64例患者发生溶血性贫血,中位数为8.3g/dL（范围4.6，13）。63例患者出现血小板减少症，中位数为18×109/L（范围1，139）。LDH增加的中位数为1192IU/L（范围141，5378）。42例患者中有41例(62.1%)ADAMTS13活性正常。15例患者中有9例（13.6%）出现突变。57例患者在停用卡非佐米并接受治疗性血浆置换后达到临床反应（53.0%），依库珠单抗（24.2%），或血液透析（39.4%）。
结论：卡非佐米诱导的TMA是一种重要的不良事件，在接受卡非佐米治疗的多发性骨髓瘤伴贫血患者中应考虑。血小板减少症,和急性肾损伤。在一些患者中，卡非佐米的戒断和依库珠单抗的治疗已被证明是成功的。
