Blood Cancer Journal ( IF 12.9 ) Pub Date : 2024-11-07 , DOI: 10.1038/s41408-024-01182-9 Can Chen, Yiwei Li, Pengfei Shi, Shenxian Qian
Proteasome inhibitors (PIs) are crucial in treating multiple myeloma but carry a risk of thrombotic microangiopathy (TMA), especially with carfilzomib use. This systematic review includes 44 studies with 115 cases of PI-induced TMA, where carfilzomib was implicated in 101 cases. Treatment approaches varied: 28 patients received supportive care, 43 underwent therapeutic plasma exchange (TPE), 9 were treated exclusively with eculizumab (ECU), and 13 received both TPE and ECU. Notably, eculizumab significantly improved outcomes for patients unresponsive to initial TPE, achieving complete remission in seven cases. The need for dialysis emerged as a significant predictor of outcomes, often indicating a poor prognosis. For patients suspected of having PI-TMA, it is advisable to discontinue the offending medication promptly, even without definitive laboratory confirmation. In cases where diagnosis is challenging, kidney biopsy may assist if conditions permit. Comprehensive evaluation of the complement system, including genetic mutations, function, and associated complement inhibitory factor antibodies, should be included in the assessment of PI-TMA. Early administration of eculizumab may be beneficial in cases of suspected complement abnormalities or suboptimal response to initial treatments.
中文翻译:
蛋白酶体抑制剂相关血栓性微血管病:系统全面的综述
蛋白酶体抑制剂 (PIs) 在治疗多发性骨髓瘤中至关重要,但存在血栓性微血管病 (TMA) 的风险,尤其是在使用卡非佐米时。本系统评价包括 44 项研究,涉及 115 例 PI 诱导的 TMA,其中 101 例与卡非佐米有关。治疗方法多种多样: 28 例患者接受支持性治疗,43 例接受治疗性血浆置换 (TPE),9 例仅接受依库珠单抗 (ECU) 治疗,13 例同时接受 TPE 和 ECU。值得注意的是,依库珠单抗显著改善了对初始 TPE 无反应的患者的预后,7 例患者达到完全缓解。透析需求成为结果的重要预测指标,通常表明预后不良。对于疑似患有 PI-TMA 的患者,即使没有明确的实验室确认,也建议立即停用致病药物。在诊断困难的情况下,如果条件允许,肾活检可能会有所帮助。PI-TMA 的评估应包括对补体系统的综合评估,包括基因突变、功能和相关的补体抑制因子抗体。在疑似补体异常或对初始治疗反应不佳的情况下,早期给予依库珠单抗可能是有益的。