Long-term response of refractory primary cold agglutinin disease to eculizumab therapy
Long-term response of refractory primary cold agglutinin disease to eculizumab therapy
复制标题
难治性原发性冷凝集素病对依库珠单抗治疗的长期反应
DOI：
10.1007/s00277-013-1800-7
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发表时间：
2014
影响因子：
3.5
通讯作者：
E. Wang
中科院分区：
文献类型：
--
作者：
N. Gupta;E. Wang
冷凝集素病（CAD）是一种自身免疫性溶血性贫血，其中冷凝集素（IgM抗体）负责触发溶血。原发性CAD的特征是不存在相关的自身免疫性疾病、基础感染或恶性肿瘤[1]。虽然不需要治疗轻度溶血，但可能发生严重溶血伴临床症状。迄今为止，CAD的治疗方式仅限于利妥昔单抗和烷化剂[2-4]。在此，我们报告了首例使用多克隆抗体的难治性CAD病例，观察到对依库珠单抗的持续长期反应。一位61岁女性于2003年10月因黄疸、呼吸困难和疲乏就诊。实验室检查结果显示：血红蛋白：9.4 g/dL;网织红细胞计数：2.7%;总胆红素：6.6 mg/dL;乳酸脱氢酶：681 IU/L。在室温下鉴定出一种强冷凝集素，加热至37 ℃后分散。抗C3 d抗体的多特异性抗血清直接库姆斯试验呈阳性，表明IgM结合。冷凝集效价为1：5，120。免疫固定显示克隆IgM免疫球蛋白。全面检查其他病毒，自身免疫，
Dear Editor, Cold agglutinin disease (CAD) is an autoimmune hemolytic anemia where cold agglutinins (IgM antibodies) are responsible for triggering hemolysis. Primary CAD is characterized by absence of associated autoimmune disease, underlying infection, or malignancy [1]. Although treatment for low-grade hemolysis is not needed, development of severe hemolysis with clinical symptoms can occur. To date, treatment modalities for CAD have been limited to rituximab and alkylating agents [2–4].Here, we report the first case of refractory CAD with polyclonal antibodies where a sustained long-term response to eculizumab was observed. A 61-year-old woman presented in October 2003 with jaundice, dyspnea, and fatigue. Laboratory results revealed: hemoglobin, 9.4 g/dL; reticulocyte count, 2.7%; total bilirubin, 6.6 mg/dL; and lactate dehydrogenase, 681 IU/L. A strong cold agglutinin was identified at room temperature which dispersed with warming to 37 C. Direct Coombs test was positive for polyspecific anti-serum for anti-C3d antibody indicating IgM binding. The cold agglutination titer was 1: 5,120. Immunofixation demonstrated clonal IgM immunoglobulins. Comprehensive work up for other viral, autoimmune,