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一名多发性骨髓瘤患者的卡非佐米相关血栓性微血管病:补体基因改变的影响及依库珠单抗治疗

Carfilzomib-Associated Thrombotic Microangiopathy in a Multiple Myeloma Patient: Complement Genetic Alterations Impact and Eculizumab Therapy.

作者信息

Lopes Joana, Fernandes Vítor, Paulo Nuria, Lisboa Gonçalves Pedro, Figueiredo Rafael, Mendonça Luis, Faria Bernardo, Neto Ricardo, Bergantim Rui, Frazão João

机构信息

Nephrology Department, Unidade Local de Saúde São João, Porto, PRT.

Hematology Department, Unidade Local de Saúde São João, Porto, PRT.

出版信息

Cureus. 2025 Aug 1;17(8):e89189. doi: 10.7759/cureus.89189. eCollection 2025 Aug.

Abstract

Carfilzomib is a second-generation proteasome inhibitor used in relapsed or refractory multiple myeloma (MM). Although effective, it can be associated with rare but life-threatening complications. We describe a 73-year-old woman with relapsed IgG-kappa MM who developed thrombotic microangiopathy (TMA) and acute kidney injury (AKI) requiring hemodialysis, three weeks after initiating a carfilzomib-based regimen. Genetic testing showed a homozygous CFHR3-CFHR1 deletion and variants of uncertain significance in the CFI and ADAMTS13 genes. The patient underwent plasmapheresis followed by treatment with eculizumab, resulting in full renal recovery. This case highlights a rare but potentially reversible complication of carfilzomib therapy, likely driven by complement activation and endothelial injury. It also underscores the role of genetic predisposition and the therapeutic benefit of complement inhibition with eculizumab. Early recognition, drug discontinuation, and consideration of both genetic screening and complement inhibition may improve outcomes in at-risk patients.

摘要

卡非佐米是一种用于复发或难治性多发性骨髓瘤(MM)的第二代蛋白酶体抑制剂。尽管有效,但它可能与罕见但危及生命的并发症有关。我们描述了一名73岁复发IgG-κ型MM的女性,在开始基于卡非佐米的治疗方案三周后,出现血栓性微血管病(TMA)和急性肾损伤(AKI),需要进行血液透析。基因检测显示CFHR3-CFHR1纯合缺失以及CFI和ADAMTS13基因中意义不确定的变异。该患者接受了血浆置换,随后用依库珠单抗治疗,肾功能完全恢复。该病例突出了卡非佐米治疗一种罕见但可能可逆的并发症,可能由补体激活和内皮损伤驱动。它还强调了遗传易感性的作用以及依库珠单抗抑制补体的治疗益处。早期识别、停药以及考虑基因筛查和补体抑制可能改善高危患者的预后。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/8e65/12398684/79da2282c610/cureus-0017-00000089189-i01.jpg

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