Università degli Studi di Genova, Genoa, Italy.
IRCCS Istituto Giannina Gaslini, Genoa, Italy.
Pediatr Rheumatol Online J. 2020 Jul 11;18(1):56. doi: 10.1186/s12969-020-00450-9.
Hyperferritinemic syndromes are systemic inflammatory disorders characterized by a dysfunctional immune response, which leads to excessive activation of the monocyte-macrophage system with hypercytokinemia and may pursue a rapidly fatal course.
We describe two patients of 11 and 9 years of age with hyperferritinemic syndromes, one with impending macrophage activation syndrome (MAS) and one with overt MAS, who were refractory or intolerant to conventional therapies, but improved dramatically with canakinumab.
Our report indicates that canakinumab may be efficacious in the management of hyperferritinemic syndromes, including MAS.
高血铁清症候群是一种全身性炎症性疾病，其特征为免疫功能紊乱，导致单核细胞-巨噬细胞系统过度激活伴细胞因子血症，并可能迅速发展为致命性疾病。
我们描述了两名年龄分别为 11 岁和 9 岁的高血铁清症候群患者，其中一名患有即将发生的巨噬细胞活化综合征（MAS），另一名患有明显的 MAS，他们对常规治疗无效或不耐受，但使用卡那单抗后病情显著改善。
我们的报告表明，卡那单抗可能对高血铁清症候群，包括 MAS 的治疗有效。
