title: Construction of a Quality of Life Questionnaire for slowly progressive neuromuscular disease.
abstract: OBJECTIVE
  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.
  
  
  CONCLUSIONS
  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.
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France. antoine.dany@univ-reims.fr.|Reims University Hospital, Reims, France. antoine.dany@univ-reims.fr.|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.
authors.forename: Antoine
authors.initials: A
authors.lastname: Dany
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France.|Reims University Hospital, Reims, France.
authors.forename: Coralie
authors.initials: C
authors.lastname: Barbe
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France.|Reims University Hospital, Reims, France.
authors.forename: Amandine
authors.initials: A
authors.lastname: Rapin
authors.affiliation: Henri Mondor Hospital, Créteil, France.
authors.forename: Christian
authors.initials: C
authors.lastname: Réveillère
authors.affiliation: University of Nantes, Nantes, France.
authors.forename: Jean-Benoit
authors.initials: JB
authors.lastname: Hardouin
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France.|Reims University Hospital, Reims, France.
authors.forename: Isabella
authors.initials: I
authors.lastname: Morrone
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France.|Reims University Hospital, Reims, France.
authors.forename: Aurore
authors.initials: A
authors.lastname: Wolak-Thierry
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France.|Reims University Hospital, Reims, France.
authors.forename: Moustapha
authors.initials: M
authors.lastname: Dramé
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France.|Reims University Hospital, Reims, France.
authors.forename: Arnaud
authors.initials: A
authors.lastname: Calmus
authors.affiliation: Nice University Hospital, Nice, France.
authors.forename: Sabrina
authors.initials: S
authors.lastname: Sacconi
authors.affiliation: Henri Mondor Hospital, Créteil, France.
authors.forename: Guillaume
authors.initials: G
authors.lastname: Bassez
authors.affiliation: Swynghedauw Hospital, Lille, France.
authors.forename: Vincent
authors.initials: V
authors.lastname: Tiffreau
authors.affiliation: Angers University Hospital, Angers, France.
authors.forename: Isabelle
authors.initials: I
authors.lastname: Richard
authors.affiliation: Myology Institute, Paris, France.
authors.forename: Benjamin
authors.initials: B
authors.lastname: Gallais
authors.affiliation: Raymond-Poincaré Hospital, Garches, France.
authors.forename: Hélène
authors.initials: H
authors.lastname: Prigent
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France.
authors.forename: Redha
authors.initials: R
authors.lastname: Taiar
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France.|Reims University Hospital, Reims, France.
authors.forename: Damien
authors.initials: D
authors.lastname: Jolly
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France.|Reims University Hospital, Reims, France.
authors.forename: Jean-Luc
authors.initials: JL
authors.lastname: Novella
authors.affiliation: University of Reims Champagne-Ardenne, Reims, France.|Reims University Hospital, Reims, France.|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.
authors.forename: François Constant
authors.initials: FC
authors.lastname: Boyer
mesh_terms: D000328:Adult; D000368:Aged; D018450:Disease Progression; D005260:Female; D006304:Health Status; D006801:Humans; D008297:Male; D008875:Middle Aged; D009468:Neuromuscular Diseases / Q000523:psychology*; D011594:Psychometrics; D011788:Quality of Life / Q000523:psychology*; D012649:Self Concept; D011795:Surveys and Questionnaires
keywords: Item response theory; Neuromuscular disease; Outcome research; Patient outcome assessment; Quality of life