关键词: font-size:medium, ">罕见病；Castleman病；特发性多中心型Castleman病；司妥昔单抗；白细胞介素-6

Abstract: ]Idiopathic multicentric Castleman disease(iMCD) is a rare and life-threatening blood disease involving systemic inflammatory symptoms， polyclonal lymphoproliferation， cytopenias， and multiple organ system dysfunction caused by a cytokine storm often including interleukin-6.Siltuximab for injection(trade name：Sylvant) is a human-mouse chimeric monoclonal antibody， which can block the binding of human IL-6 and IL-6 receptor， and produces an inhibitory effect to IL-6， which in turn inhibits cell growth.As a rare disease drug urgently needed in clinical practice，National Medical Products Administration(NMPA) has approved siltuximab for injection through the priority review approval process in November 30, 2021 for the treatment drug of human immunodeficiency virus(HIV)-negative and human herpes virus-8(HHV-8)-negative adult patients with MCD.Siltuximab is the only drug that has been evaluated by randomized controlled clinical trial for treating iMCD， which demonstrate a favorable efficacy and safety profile. Meanwhile the extension analysis of two trials also confirmed the safety of long-term administration.Siltuximab has been approved for iMCD by more than 50 countries and regions in the world. Many clinical consensus and guidelines recommended siltuximab as the first-line treatment drug for iMCD， including the consensus on diagnosis and treatment of Castleman disease（CD） in China， CSCO guideline， CDCN consensus， NCCN guideline， BSH guideline， etc.It is also recommended for treatment of other types of CD. The launch of siltuximab for injection provides a treatment option for patients with CD. In view of this， this study based on the collection of domestic and foreign scientific literature， clinical trial data， guidelines and consensus and other information， summarize the mechanism of action， research and development process， clinical research results of siltuximab， in order to promote the scientific publicity and scientific application of the drug， and to provide reference for the clinical treatment and medication of rare disease patients in China.

Key words: Rare disease;Castleman disease;Idiopathic multicentric Castleman disease, Siltuximab； Interleukin-6