Development and validation of a prognostic multivariable model to predict insufficient clinical response to methotrexate in rheumatoid arthritis

PLoS One. 2018 Dec 10;13(12):e0208534. doi: 10.1371/journal.pone.0208534. eCollection 2018.

Abstract

Objective: The objective was to predict insufficient response to 3 months methotrexate (MTX) in DMARD naïve rheumatoid arthritis patients.

Methods: A Multivariable logistic regression model of rheumatoid arthritis patients starting MTX was developed in a derivation cohort with 285 patients starting MTX in a clinical multicentre, stratified single-blinded trial, performed in seven secondary care clinics and a tertiary care clinic. The model was validated in a validation cohort with 102 patients starting MTX at a tertiary care clinic. Outcome was insufficient response (disease activity score (DAS)28 >3.2) after 3 months of MTX treatment. Clinical characteristics, lifestyle variables, genetic and metabolic biomarkers were determined at baseline in both cohorts. These variables were dichotomized and used to construct a multivariable prediction model with backward logistic regression analysis.

Results: The prediction model for insufficient response in the derivation cohort, included: DAS28>5.1, Health Assessment Questionnaire>0.6, current smoking, BMI>25 kg/m2, ABCB1 rs1045642 genotype, ABCC3 rs4793665 genotype, and erythrocyte-folate<750 nmol/L. In the derivation cohort, AUC of ROC curve was 0.80 (95%CI: 0.73-0.86), and 0.80 (95%CI: 0.69-0.91) in the validation cohort. Betas of the prediction model were transformed into total risk score (range 0-8). At cutoff of ≥4, probability for insufficient response was 44%. Sensitivity was 71%, specificity 72%, with positive and negative predictive value of 72% and 71%.

Conclusions: A prognostics prediction model for insufficient response to MTX in 2 prospective RA cohorts by combining genetic, metabolic, clinical and lifestyle variables was developed and validated. This model satisfactorily identified RA patients with high risk of insufficient response to MTX.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • ATP Binding Cassette Transporter, Subfamily B / genetics
  • Adult
  • Antirheumatic Agents / therapeutic use*
  • Area Under Curve
  • Arthritis, Rheumatoid / drug therapy*
  • Arthritis, Rheumatoid / pathology
  • Cohort Studies
  • Female
  • Folic Acid / analysis
  • Folic Acid / blood
  • Humans
  • Logistic Models
  • Male
  • Methotrexate / therapeutic use*
  • Middle Aged
  • Multidrug Resistance-Associated Proteins / genetics
  • Polymorphism, Single Nucleotide
  • Predictive Value of Tests
  • Prognosis
  • ROC Curve
  • Risk Factors
  • Treatment Outcome

Substances

  • ABCB1 protein, human
  • ATP Binding Cassette Transporter, Subfamily B
  • Antirheumatic Agents
  • Multidrug Resistance-Associated Proteins
  • multidrug resistance-associated protein 3
  • Folic Acid
  • Methotrexate

Grants and funding

This work was supported by tREACH: Unrestricted grant from Pfizer (https://www.pfizer.nl) 0881-102217 to JMWH and the Dutch Arthritis Association (https://reumanederland.nl) 06-02-402 and 09-1-402 to RdJ. The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript.