French protocol for the diagnosis and management of familial Mediterranean fever

Georgin-Lavialle, S.; Savey, L.; Cuisset, L.; Boursier, G.; Boffa, J.-J.; Delplanque, M.; Bourguiba, R.; Monfort, J.-B.; Touitou, I.; Grateau, G.; Kone-Paut, I.; Hentgen, V.

doi:10.1016/j.revmed.2023.10.441

Cited by 7 publications

(2 citation statements)

References 41 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…In the past, the development of valid disease activity assessment tools, standardized outcome measures, and HrQoL instruments in pediatric rheumatology have been important milestones for enabling optimal patient care [49]. The disease activity and disease burden are commonly assessed and quantified by the physician (e.g., PGA) and the patient/parent (e.g., PPGA), by HrQoL tools and missed school/working days, measurement of inflammatory markers, assessment of systemic and organ-specific signs of active disease, and evaluation of disease damage [10,18]. Important milestones for disease activity monitoring in IL-1 AID included the validation of the AIDAI and the development of the ADDI [8,9].…”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Optimized Treatment of Interleukin (IL-1)-Mediated Autoinflammatory Diseases: Impact of Disease Activity-Based Treatment Adjustments

Welzel,

Zapf,

Klotsche

et al. 2024

JCM

View full text Add to dashboard Cite

Background: Effective control of disease activity in Interleukin-1 autoinflammatory diseases (IL-1 AID) is crucial to prevent damage. The aim was to longitudinally analyze the impact of protocolized disease activity-based treatment adjustments in a real-life cohort. Methods: A single-center study of consecutive children with IL-1 AID followed between January 2016 and December 2019 was performed. Demographics, phenotypes, genotypes, inflammatory markers, physician (PGA), and patient/parent (PPGA) global assessment were captured. Disease activity and treatment changes were assessed. The impact of distinct parameters on disease activity trajectories was analyzed. Results: A total of 56 children were included, median follow-up was 2.1 years reflecting 361 visits. Familial Mediterranean Fever was the most common IL-1 AID. At the first visit, 68% of the patients had moderate/severe disease activity. Disease activity-based treatment adjustments were required in 28/56 children (50%). At last follow-up, 79% had a well-controlled disease. Both PGA and PPGA decreased significantly over time (p < 0.001; p < 0.017, respectively), however, both differed statistically at last visit (p < 0.001). Only PGA showed a significant estimated mean decrease across all IL-1 AID over time. Conclusions: Disease activity-based treatment adjustments can effectively refine treat-to-target strategies, enable personalized precision health approaches, and improve outcomes in children with IL-1 AID.

show abstract

Section: Discussionmentioning

confidence: 99%

“…Recently, T2T strategies for IL-1 AID were published [16][17][18]. However, longitudinal real-life data of disease activity-based treatment adjustments and their impact on IL-1 AID in childhood is scarce.…”

Section: Introductionmentioning

confidence: 99%