Case report: Emapalumab for active disease control prior to hematopoietic stem cell transplantation in refractory systemic juvenile idiopathic arthritis complicated by macrophage activation syndrome.

ABSTRACT:

Introduction

Macrophage activation syndrome (MAS), a secondary form of hemophagocytic lymphohistiocytosis, is a serious life-threatening complication associated with systemic juvenile idiopathic arthritis (sJIA). MAS is characterized by fever, hepatosplenomegaly, liver dysfunction, cytopenias, coagulation abnormalities, and hyperferritinemia and may progress to multiple organ failure and death. Overproduction of interferon-gamma is a major driver of hyperinflammation in murine models of MAS and primary hemophagocytic lymphohistiocytosis. A subset of patients with sJIA may develop progressive interstitial lung disease, which is often difficult to manage. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) can potentially be a curative immunomodulatory strategy for patient

SUBMITTER: Chellapandian D 

PROVIDER: S-EPMC9986425 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature