Successful treatment of refractory hyperferritinemic syndromes with canakinumab: A report of two cases

R. Papa, V. Natoli, R. Caorsi, F. Minoia, M. Gattorno, A. Ravelli

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Background: 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. Case presentation: 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. Conclusions: Our report indicates that canakinumab may be efficacious in the management of hyperferritinemic syndromes, including MAS.

Original languageEnglish
JournalPediatric Rheumatology
Issue number1
Publication statusPublished - 2020

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