Berentsen Sigbjørn
Department of Research and Innovation, Haugesund Hospital, Helse Fonna, Haugesund, Norway.
Hematology Am Soc Hematol Educ Program. 2016 Dec 2;2016(1):226-231. doi: 10.1182/asheducation-2016.1.226.
Primary chronic cold agglutinin disease (CAD) is a well-defined clinicopathologic entity in which a specific, clonal lymphoproliferative B-cell bone marrow disorder results in autoimmune hemolytic anemia. The immune hemolysis is entirely complement-dependent, predominantly mediated by activation of the classical pathway and phagocytosis of erythrocytes opsonized with complement protein C3b. Typical clinical features in CAD have diagnostic and therapeutic implications. Pharmacologic treatment should be offered to patients with symptom-producing anemia or disabling circulatory symptoms. CAD should not be treated with corticosteroids. Based on an individualized approach, rituximab monotherapy or rituximab-fludarabine in combination is recommended as first-line therapy. Rituximab-bendamustine is still an investigational therapy. Although complement-modulating agents are still to be considered experimental in CAD, therapy with the anti-C1s monoclonal antibody TNT009 seems promising.
原发性慢性冷凝集素病（CAD）是一种明确的临床病理实体，其中特定的克隆性淋巴细胞增殖性B细胞骨髓疾病导致自身免疫性溶血性贫血。免疫溶血完全依赖补体，主要由经典途径的激活和被补体蛋白C3b调理的红细胞的吞噬作用介导。CAD的典型临床特征具有诊断和治疗意义。对于出现贫血症状或导致循环系统功能障碍症状的患者应给予药物治疗。CAD不应使用皮质类固醇治疗。基于个体化方法，推荐利妥昔单抗单药治疗或利妥昔单抗与氟达拉滨联合作为一线治疗。利妥昔单抗与苯达莫司汀联合治疗仍处于研究阶段。尽管补体调节药物在CAD中仍被视为实验性治疗，但抗C1s单克隆抗体TNT009治疗似乎很有前景。
