Reid Mark, Fedutes Henderson Bethany A
From The James Cancer Hospital at The Ohio State University, Columbus, Ohio.
Sanofi, Stockdale, Pennsylvania.
J Adv Pract Oncol. 2024 Sep;15(6):389-395. doi: 10.6004/jadpro.2024.15.6.4. Epub 2024 Sep 1.
Cold agglutinin disease (CAD) is a rare type of autoimmune hemolytic anemia (AIHA) distinct from warm antibody AIHA. One of the ways it is distinct is that CAD is usually not responsive to corticosteroids compared with warm antibody AIHA. Historically, CAD therapy has been limited to immunotherapy or chemoimmunotherapy with varying responses. Cold agglutinin disease also poses a risk for thrombosis and mortality. For patients, fatigue tends to be a common symptom of CAD. The hallmark of CAD is complement-mediated hemolysis, which makes complement inhibitors a critical therapeutic option for patients. Previously, eculizumab, a C5 inhibitor, had limited therapeutic effect for CAD. More recently, sutimlimab, a C1s inhibitor, was shown in two phase III studies to be an efficacious treatment for CAD, improving hemoglobin, hemolysis, and fatigue. However, there is a paucity of medical literature on CAD and on sutimlimab in particular that is geared toward advanced practice providers (APPs). This article aims to provide APPs with a background in CAD and a focus on sutimlimab, assisting these providers in caring for patients with CAD receiving this therapy.
冷凝集素病(CAD)是一种罕见的自身免疫性溶血性贫血(AIHA),与温抗体型AIHA不同。其不同之处之一在于,与温抗体型AIHA相比,CAD通常对皮质类固醇无反应。从历史上看,CAD的治疗仅限于免疫疗法或化学免疫疗法,疗效各异。冷凝集素病还会带来血栓形成和死亡风险。对患者而言,疲劳往往是CAD的常见症状。CAD的标志是补体介导的溶血,这使得补体抑制剂成为患者的关键治疗选择。此前,C5抑制剂依库珠单抗对CAD的治疗效果有限。最近,C1s抑制剂苏替利单抗在两项III期研究中显示对CAD有效,可改善血红蛋白、溶血和疲劳症状。然而,针对高级执业提供者(APP)的关于CAD尤其是苏替利单抗的医学文献匮乏。本文旨在为APP提供CAD的背景知识,并聚焦于苏替利单抗,帮助这些提供者护理接受该疗法的CAD患者。