PDF(Pubmed)
Abstract：
Although rare, C3 glomerulopathy (C3G) is increasingly recognized thanks to the currently available diagnostic skills. C3G is not a single disease but a group of disorders with distinct pathogenesis and progression. Thus, an essential step for its management remains an in-depth characterization of the specific form and the identification of underlying conditions, which may also impact treatment choices as well. Among these entities, an emerging condition is the association of C3G with monoclonal gammopathy, which confers poor outcomes. Overall, diagnosis of C3G remains challenging, and determining the appropriate treatment remains unclear. Conventional immunosuppressive therapy has proven ineffective in such cases, while clone-directed therapies have shown promising results in small interventional studies and case series. Here, we report a case of a patient affected by C3G with monoclonal gammopathy of renal significance who experienced rapid deterioration of kidney function requiring replacement therapy. After the failure of first-line treatment, a switch to the anti-CD38 therapy with daratumumab resulted in the progressive improvement of the patient\'s kidney function, leading to the discontinuation of hemodialysis after approximately 10 months. Serial renal biopsies were also performed to study the disease\'s evolution in response to the treatment. Based on the description of this single case, we have comprehensively reviewed available studies on daratumumab use in patients with C3G associated with monoclonal gammopathy to provide insights for the design of prospective studies which aim to enhance the management of such poor prognosis disease.
摘要：
虽然罕见，由于当前可用的诊断技能，C3肾小球病（C3G）越来越被认可。C3G不是单一疾病，而是一组具有不同发病机制和进展的疾病。因此，管理的一个重要步骤仍然是对具体形式的深入描述和对潜在条件的识别，这也可能影响治疗选择。在这些实体中，一种新出现的情况是C3G与单克隆丙种球蛋白病的关联，这导致了糟糕的结果。总的来说,C3G的诊断仍然具有挑战性，和确定适当的治疗仍不清楚。在这种情况下，传统的免疫抑制疗法已被证明无效，而克隆导向疗法在小型介入研究和病例系列中显示出有希望的结果。这里,我们报告了一例C3G患者,该患者患有有肾脏意义的单克隆丙种球蛋白病,肾功能迅速恶化,需要进行替代治疗.一线治疗失败后，改用达雷妥单抗治疗抗CD38导致患者肾功能的逐步改善，导致大约10个月后停止血液透析。还进行了连续的肾活检，以研究疾病对治疗的反应。根据对这一单一案例的描述，我们全面回顾了现有的关于Daratumumab在与单克隆丙种球蛋白相关的C3G患者中使用的研究,为设计旨在加强这类不良预后疾病的管理的前瞻性研究提供了见解.
