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Published on: October 3, 2019
From diagnosis to treatment response: predicting FMF50 response using PREDICT-crFMF and TURPAID scores in children
Eda Nur Dizman1, Feray Kaya1, Elif Kucuk1
1Department of Pediatric Rheumatology, Istanbul Medeniyet University, Istanbul, Turkey.
Insights
Higher PREDICT-crFMF and TURPAID scores at diagnosis predict poor response to colchicine treatment in children with Familial Mediterranean Fever (FMF). Early assessment can identify patients at risk for treatment resistance.
Area of Science:
- Pediatric Rheumatology
- Genetics
- Clinical Trials
Background:
- Familial Mediterranean Fever (FMF) is a genetic autoinflammatory disorder.
- Colchicine is the standard treatment, but some patients exhibit resistance.
- Predictive scoring systems are needed to identify non-responders early.
Purpose of the Study:
- To assess the predictive value of TURPAID and PREDICT-crFMF scores for FMF50 response at 6 months in pediatric FMF patients.
- To examine the association of these scores with disease activity and inflammatory markers.
Main Methods:
- Retrospective analysis of 168 children newly diagnosed with FMF treated with colchicine for at least 6 months.
- Evaluation of treatment response using the FMF50 criteria at 6-month follow-up.
- Statistical analysis of TURPAID and PREDICT-crFMF scores, disease activity indices, and acute-phase reactants.
Main Results:
- 64.8% of patients achieved FMF50 response.
- Higher PREDICT-crFMF and TURPAID scores at diagnosis were significant predictors of FMF50 nonresponse (p < 0.001).
- Both scores demonstrated significant discriminative ability (AUC > 0.67) and correlated with inflammatory markers and disease activity.
Conclusions:
- The PREDICT-crFMF and TURPAID scores can aid in the early identification of pediatric FMF patients at risk for colchicine resistance.
- These scores reflect the early inflammatory burden and can guide treatment strategies.
- This study is the first to jointly evaluate these scores for predicting FMF50 response in children.
Objective:
This study assessed the predictive value of TURPAID and PREDICT-crFMF scores at diagnosis for FMF50 response at the sixth month in children with Familial Mediterranean Fever (FMF) and examined their associations with disease-activity indices and acute-phase reactants.
Methods:
Children newly diagnosed with FMF according to the Eurofever/PRINTO criteria and who received colchicine treatment for at least 6 months were included. Clinical and laboratory data were retrospectively obtained from electronic medical records. Treatment response was evaluated at the 6-month follow-up visit.
Results:
Overall, 168 children with FMF (50.6% female) were included. FMF50 response at 6 months was achieved in 64.8% of patients. PREDICT-crFMF and TURPAID scores were significantly higher in non-responders than in responders (p < 0.001 for both). Higher PREDICT-crFMF (aOR 1.240, 95% CI 1.113-1.382, p < 0.001) and TURPAID (aOR 2.009, 95% CI 1.384-2.916, p < 0.001) scores, and M694V homozygosity (aOR 3.390, 95% CI 1.700-6.760, p < 0.001) independently predicted FMF50 nonresponse. Both scores showed significant discrimination (AUC = 0.685 for PREDICT-crFMF; 0.670 for TURPAID; p < 0.001). Optimal cut-offs were ≥ 3 for PREDICT-crFMF (sensitivity 67.8%, specificity 69.7%) and > 1.5 for TURPAID (sensitivity 76.3%, specificity 54.1%). PREDICT-crFMF scores correlated with erythrocyte sedimentation rate (ESR), serum amyloid A, and disease activity indices, but not Mor score; TURPAID scores correlated with ESR and all evaluated disease activity indices.
Conclusion:
Early assessment of PREDICT-crFMF and TURPAID scores at diagnosis may help identify FMF patients at risk for colchicine resistance. Key Points • This is the first study to evaluate PREDICT-crFMF and TURPAID scores together using FMF50 as the treatment-response outcome in children with FMF. • Higher PREDICT-crFMF and higher TURPAID scores at diagnosis independently predicted failure to achieve FMF50 response at 6 months. • The significant associations of these scores with disease activity indices and inflammatory markers support their value in reflecting early inflammatory burden.
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