摘要
阵发性睡眠性血红蛋白尿(paroxysmal nocturnal hemoglobinuria,PNH)是一种罕见的获得性造血干细胞基因突变引起的克隆性疾病。肾脏损伤是较常见也易被忽视的并发症。依库珠单抗通过抑制末端补体活化显著提高了PNH的治疗疗效。该文报道1例反复上呼吸道感染后发作性肉眼血尿的PNH患者,经肾活检确诊含铁血黄素肾小管病,给予标准剂量依库珠单抗治疗后,血管内溶血和肾脏损伤均得到明显改善,治疗和随访过程中无感染事件发生,病情稳定。该病例凸显了提高对PNH相关肾损害认知的重要性,并表明依库珠单抗对于该疾病具有良好的疗效与可靠的安全性。
Paroxysmal nocturnal hemoglobinuria(PNH)is a rare acquired clonal disease caused by genetic mutations in hematopoietic stem cells.Kidney injury is a common and easily ignored complication.Eculizumab significantly improves the therapeutic efficacy of PNH by inhibiting terminal complement activation.The paper reports a PNH patient with episodic gross hematuria after repeated upper respiratory tract infections,diagnosed with hemosiderin-containing renal tubular disease through renal biopsy.After treatment with standard dose of eculizumab,intravascular hemolysis and kidney damage were significantly improved.There were no infection events during treatment and follow-up,and the clinical condition remained stable.This case emphasizes the importance of improved recognition of PNH-associated kidney damage.Furthermore,it indicates that eculizumab therapy is both effective and safe for treating this complication.
作者
汪丽
李思倩
边琪
Wang Li;Li Siqian;Bian Qi(Department of Nephrology,the First Affiliated Hospital of Naval Medical University,Shanghai 200438,China)
出处
《中华肾脏病杂志》
北大核心
2025年第12期1011-1015,共5页
Chinese Journal of Nephrology
