Related Experiment Videos
Macrophage activation syndrome in patients with systemic juvenile idiopathic arthritis is associated with MUNC13-4
Kejian Zhang1, Jennifer Biroschak, David N Glass
1Children's Hospital Medical Center, Cincinnati, Ohio 45229, USA.
Objective:
Systemic juvenile idiopathic arthritis (JIA) is associated with macrophage activation syndrome. Macrophage activation syndrome bears a close resemblance to familial hemophagocytic lymphohistiocytosis (HLH). The development of familial HLH has been recently associated with mutations in MUNC13-4. The purpose of this study was to assess for possible sequence alterations in MUNC13-4 in patients with systemic JIA/macrophage activation syndrome.
Methods:
The MUNC13-4 sequence was analyzed in 18 unrelated patients with systemic JIA/macrophage activation syndrome, using 32 primer pair sets designed to amplify the 32 exons and at least 100 basepairs of the adjacent intronic regions. DNA samples obtained from 73 unrelated patients with systemic JIA and no history of macrophage activation syndrome and 229 unrelated healthy individuals were used as controls.
Results:
The biallelic sequence variants in MUNC13-4 reported in familial HLH were present in 2 of the 18 patients with JIA/macrophage activation syndrome. Further analysis of the MUNC13-4 sequences revealed an identical combination of 12 single-nucleotide polymorphisms (SNPs) in 9 of the remaining 16 patients with systemic JIA/macrophage activation syndrome (56%). Additional analysis suggested that these 12 SNPs (154[-19] g>a, 261[+26] c>g, 388[+81] g>a, 388[+122] c>t, 570[-60] t>g, 888 G>C, 1389[+36] g>a, 1992[+5] g>a, 2447[+144] c>t, 2599 A>G, 2830[+37] c>g, 3198 A>G) were inherited as an extended haplotype. In several patients, in addition to the described haplotype, there were other SNPs in the second allele of MUNC13-4. Moreover, 1 patient had a complex mutation with 2 changes, 2542 A>C and 2943 G>C, in a cis configuration. The haplotype was present in only 27 (12%) of 229 healthy control subjects (chi(2) = 23.5) and in 6 (8.2%) of 73 patients with systemic JIA and no history of macrophage activation syndrome.
Conclusion:
The data suggest an association between MUNC13-4 polymorphisms and macrophage activation syndrome in patients with systemic JIA.
Insights
Genetic variations in MUNC13-4 are linked to macrophage activation syndrome in systemic juvenile idiopathic arthritis (JIA). This study investigated MUNC13-4 sequence alterations in patients with systemic JIA and macrophage activation syndrome.
Area of Science:
- Immunology
- Genetics
- Pediatric Rheumatology
Background:
- Systemic juvenile idiopathic arthritis (JIA) is frequently complicated by macrophage activation syndrome (MAS).
- Macrophage activation syndrome shares clinical similarities with familial hemophagocytic lymphohistiocytosis (HLH).
- Mutations in the MUNC13-4 gene have been recently identified as a cause of familial HLH.
Purpose of the Study:
- To investigate potential sequence alterations in the MUNC13-4 gene in patients diagnosed with systemic JIA and MAS.
- To explore the association between MUNC13-4 genetic variations and the development of MAS in systemic JIA.
Main Methods:
- MUNC13-4 gene sequence analysis was performed on 18 patients with systemic JIA/MAS.
- DNA from 73 patients with systemic JIA without MAS and 229 healthy individuals served as controls.
- The MUNC13-4 sequence was analyzed using 32 primer pairs targeting 32 exons and adjacent intronic regions.
Main Results:
- Biallelic MUNC13-4 sequence variants, previously linked to familial HLH, were found in 2 of 18 patients.
- An extended haplotype of 12 single-nucleotide polymorphisms (SNPs) in MUNC13-4 was identified in 56% of the remaining patients.
- This haplotype was significantly more prevalent in patients with systemic JIA/MAS (56%) compared to healthy controls (12%) and JIA patients without MAS (8.2%).
Conclusions:
- The findings suggest a significant association between MUNC13-4 polymorphisms and the occurrence of macrophage activation syndrome in systemic JIA.
- These genetic variations in MUNC13-4 may contribute to the pathogenesis of MAS in the context of systemic JIA.
Related Concept Videos
The JAK-STAT Signaling Pathway
Inflammatory Bowel Disease III: Crohn's Disease
T Cell Types and Functions
Th1 cells stimulate dendritic cells to express necessary co-stimulatory molecules on their surfaces for...
Genome-wide Association Studies-GWAS
GWAS does not require the identification of the target gene involved in...