NextGen 2024: Unmet Needs & Clinical Trial Challenges Session Part 1

NextGen 2024: Unmet Needs & Clinical Trial Challenges Session Part 1

@SJIA_Foundation
@SJIA_Foundation
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2 weeks ago 72

NextGen 2024: Unmet Needs & Clinical Trial Challenges Session Part 1

@SJIA_Foundation2 weeks ago

SJIA with relapsing MAS
Alexei Grom, MD
Cincinnati Children's Hospital Medical Center
NextGen Th…
1/11
SJIA with refractory course 
• Despite recently approved IL-1 and IL-6 blocking agents, about 20% …
2/11
sJIA sJIA
MAS
sJIA sJIA
MAS
sJIA
MAS MAS MAS
Classic 
MAS
MAS 
at sJIA onset
“relapsing”
…
3/11
SJIA patients with relapsing MAS
• These patients appear at risk for sJIA-associated lung disease
…
4/11
SJIA with relapsing MAS with 
Predominantly Liver Involvement
• Persistently elevated liver enzym…
5/11
CD68+
macrophage
s
TNFα
IL-6
IFNγ
Liver Biopsy in MAS/sJIA
CD8+ 
T lymphocytes
Republished…
6/11
CD68+
macrophages
Liver Biopsy in MAS/sJIA
CD8+ T cells
TNFα IL-6
IFNγ
Republished with permi…
7/11
Liver biopsy: CD163 staining in SJIA
Elevated Ast/Alt, + lab features of subclinical 
MAS
Elevat…
8/11
MAS with Predominantly Liver Involvement
• Similar histologic pattern observed by Prencipe et al. …
9/11
MAS with Predominantly Liver Involvement
•JAKi?
• CyA?
• MMF?
• Emapalumab?
10/11
Challenges with clinical trials in ‘smoldering MAS”
• Do not fully meet criteria for MAS
• Not el…
11/11


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