Oral Communication

Validation and calibration of a health-related quality of life questionnaire for patients suffering from slowly progressive neuromuscular disease

Mr Antoine DANYa, Dr Coralie BARBEb, Dr Aurore WOLAK-THIERRYb, Dr Isabella MORRONEb, Dr Moustapha DRAMÉb, Prof Jean-Luc NOVELLAb, Prof Damien JOLLYb, Prof François BOYERb

a Université de Reims Champagne-Ardenne, b CHU de Reims

Introduction: Medical care and treatments of patients suffering from slowly-progressive neuromuscular disease (NMD) mainly aim to improve health-related quality of life (HRQOL). To date there is no French HRQOL-measurement tool specifically designed for these patients. Recent works allowed developing a new questionnaire of 26 items named “Quality of Life in Neuromuscular Diseases” (QoL-NMD). It is structured in 3 domains and was constructed using focus groups: Delphi method and exploratory statistical analysis. Our objectives are to validate QoL-NMD using an independent sample and to calibrate the scoring system.

Methods: The QOL-NMD and a validated questionnaire (WHOQOL-BREF) were administered to 153 patients recruited in 8 tertiary hospitals dedicated to NMD. Confirmatory statistical analysis included methods derived from both Items Response Theory (IRT) and Classical Test Theory.

Results: Data confirmed the results of the exploratory analysis. The 3 domains of QoL-NMD met IRT assumptions (i.e. unidimensionality, parameter invariance and monotonicity) and showed adequate psychometric properties (Cronbach >0.7, H > 0.3, test-retest ICC>0.7). Each domain was calibrated using a parametric IRT model. Comparison to WHOQOL-BREF allowed investigating similarities, discrepancies and putting forward QoL-NMD psychometric superiority.

Conclusion: This study led to the validation and calibration of a new HRQOL questionnaire specifically designed for NMD patients. It can now be used in various clinical settings. QoL-NMD has been translated and culturally adapted to English but still require a psychometric validation abroad.


  1. Vincent KA et al. Construction and validation of a quality of life questionnaire for neuromuscular disease (INQoL). Neurology. 2007;68(13):1051-1057.
  2. Burns TM et al. Quality of life and measures of quality of life in patients with neuromuscular disorders. Muscle Nerve. 2012; 46(1):9-25.
  3. Graham CD et al. A systematic review of quality of life in adults with muscle disease. J Neurol. 2011; 258(9):1581-92.

Keywords : Health-related quality of life, neuromuscular disease, item response theory, validation, psychometrics