Antoine Dany1,2,3, Coralie Barbe4,5, Amandine Rapin4,5, Christian Réveillère6, Jean-Benoit Hardouin7, Isabella Morrone4,5, Aurore Wolak-Thierry4,5, Moustapha Dramé4,5, Arnaud Calmus4,5, Sabrina Sacconi8, Guillaume Bassez6, Vincent Tiffreau9, Isabelle Richard10, Benjamin Gallais11, Hélène Prigent12, Redha Taiar4, Damien Jolly4,5, Jean-Luc Novella4,5, François Constant Boyer4,5,13. 1. University of Reims Champagne-Ardenne, Reims, France. antoine.dany@univ-reims.fr. 2. Reims University Hospital, Reims, France. antoine.dany@univ-reims.fr. 3. Service de Médecine Physique et Réadaptation, Centre de Référence des Maladies Neuromusculaires, Hôpital Sébastopol, CHU de Reims, 48, rue de Sébastopol, 51092, Reims Cedex, France. antoine.dany@univ-reims.fr. 4. University of Reims Champagne-Ardenne, Reims, France. 5. Reims University Hospital, Reims, France. 6. Henri Mondor Hospital, Créteil, France. 7. University of Nantes, Nantes, France. 8. Nice University Hospital, Nice, France. 9. Swynghedauw Hospital, Lille, France. 10. Angers University Hospital, Angers, France. 11. Myology Institute, Paris, France. 12. Raymond-Poincaré Hospital, Garches, France. 13. Service de Médecine Physique et Réadaptation, Centre de Référence des Maladies Neuromusculaires, Hôpital Sébastopol, CHU de Reims, 48, rue de Sébastopol, 51092, Reims Cedex, France.
Abstract
PURPOSE: To build a questionnaire to assess health-related quality of life (HRQL) in patients suffering from slowly progressive neuromuscular disease (NMD) using item response theory (IRT). METHODS: A pool of 64 items and a validated questionnaire (WHOQOL-BREF) were administered to 159 patients recruited in eight NMD referral centers. Exploratory statistical analysis included methods derived from both IRT and classical test theory. RESULTS: We constructed a questionnaire named QoL-NMD which is composed of two general items and 24 items classified in three domains: (1) "Impact of Physical Symptoms," (2) "Self-perception" and (3) "Activities and Social Participation." Each domain has good psychometric properties (Cronbach's alpha > 0.77, test-retest ICC > 0.81, Loevinger's H > 0.41) and meets IRT assumptions. Comparison with the WHOQOL-BREF enabled assessing similarities and discrepancies with a generic questionnaire. CONCLUSION: This study enabled the development of a new HRQL questionnaire specifically designed for slowly progressive NMD patients. The QoL-NMD is short enough to be used in clinical practice (26 items). The next steps will be to validate QoL-NMD by re-assessing psychometrics in an independent sample of patients and calibrate the IRT scoring system.
PURPOSE: To build a questionnaire to assess health-related quality of life (HRQL) in patients suffering from slowly progressive neuromuscular disease (NMD) using item response theory (IRT). METHODS: A pool of 64 items and a validated questionnaire (WHOQOL-BREF) were administered to 159 patients recruited in eight NMD referral centers. Exploratory statistical analysis included methods derived from both IRT and classical test theory. RESULTS: We constructed a questionnaire named QoL-NMD which is composed of two general items and 24 items classified in three domains: (1) "Impact of Physical Symptoms," (2) "Self-perception" and (3) "Activities and Social Participation." Each domain has good psychometric properties (Cronbach's alpha > 0.77, test-retest ICC > 0.81, Loevinger's H > 0.41) and meets IRT assumptions. Comparison with the WHOQOL-BREF enabled assessing similarities and discrepancies with a generic questionnaire. CONCLUSION: This study enabled the development of a new HRQL questionnaire specifically designed for slowly progressive NMD patients. The QoL-NMD is short enough to be used in clinical practice (26 items). The next steps will be to validate QoL-NMD by re-assessing psychometrics in an independent sample of patients and calibrate the IRT scoring system.
Entities:
Keywords:
Item response theory; Neuromuscular disease; Outcome research; Patient outcome assessment; Quality of life
Authors: David Cella; Susan Yount; Nan Rothrock; Richard Gershon; Karon Cook; Bryce Reeve; Deborah Ader; James F Fries; Bonnie Bruce; Mattias Rose Journal: Med Care Date: 2007-05 Impact factor: 2.983