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Criteria to define response to therapy in paediatric rheumatic diseases

Nicolino Ruperto1, Angela Pistorio, Angelo Ravelli

  • 1IRCCS G. Gaslini, Pediatria II, Reumatologia, Pædiatric Rheumatology International Trials Organisation (PRINTO), Genoa, Italy. nicolaruperto@ospedale-gaslini.ge.it

European Journal of Clinical Pharmacology
|November 25, 2010
PubMed

Insights

New criteria help evaluate treatment response in juvenile idiopathic arthritis (JIA), juvenile dermatomyositis (JDM), and juvenile systemic lupus erythematosus (JSLE). These validated measures aid clinical trials and practice for paediatric rheumatic diseases (PRD).

Area of Science:

  • Paediatric Rheumatology
  • Clinical Trial Methodology
  • Outcome Measurement

Background:

  • Paediatric rheumatic diseases (PRD) encompass juvenile idiopathic arthritis (JIA), juvenile dermatomyositis (JDM), and juvenile systemic lupus erythematosus (JSLE).
  • Standardized criteria for evaluating treatment response in PRD are crucial for clinical trials and daily practice.
  • International collaboration is key to developing robust and validated outcome measures.

Purpose of the Study:

  • To identify and validate criteria for assessing treatment response in JIA, JDM, and JSLE.
  • To establish core sets of outcome measures for major paediatric rheumatic diseases.
  • To provide definitions of improvement for use in clinical trials and practice.

Main Methods:

  • A three-phase methodological approach was employed.
  • Phase 1 involved literature review and consensus techniques to develop preliminary core sets.
  • Phase 2 included large-scale data collection for validation, followed by the final development of validated criteria.

Main Results:

  • Core sets include common domains (physician/parent global assessment, disability, quality of life) and disease-specific measures.
  • Responder definitions require a minimum improvement (≥30% JIA, ≥20% JDM, ≥50% JSLE) with limited worsening in other variables.
  • Validated criteria provide a composite endpoint for evaluating global treatment response.

Conclusions:

  • The proposed core sets and improvement definitions offer clinically meaningful composite endpoints for PRD.
  • These validated criteria are suitable for use in PRD clinical trials.
  • The definitions aim to assist physicians in determining adequate patient response to therapy.
Abstract

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