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.
Similar content being viewed by others
References
Abresch, R. T., Carter, G. T., Jensen, M. P., & Kilmer, D. D. (2002). Assessment of pain and health-related quality of life in slowly progressive neuromuscular disease. American Journal of Hospice and Palliative Medicine, 19, 39–48.
Carter, G. T., Han, J. J., Abresch, R. T., & Jensen, M. P. (2007). The importance of assessing Quality of Life in patients with neuromuscular disorders. American Journal of Hospice and Palliative Care, 23, 493.
Bottomley, A., Jones, D., & Claassens, L. (2009). Patient-reported outcomes: Assessment and current perspectives of the guidelines of the Food and Drug Administration and the reflection paper of the European Medicines Agency. European Journal of Cancer, 45, 347–353.
Speight, J., & Barendse, S. M. (2010). FDA guidance on patient reported outcomes A prompt for the industry to raise scientific standards. British Medical Journal, 340, c2921.
Boyer, F., Novella, J. L., Bertaud, S., Delmer, F., Vesselle, B., & Etienne, J. C. (2005). Hereditary neuromuscular disease and multicomposite subjective health status: Feasibility, internal consistency and test-retest reliability in the French version of the Nottingham Health Profile, the ISPN. Clinical rehabilitation, 19(6), 644–653.
Boyer, F., Morrone, I., Laffont, I., Dizien, O., Etienne, J. C., & Novella, J. L. (2006). Health related quality of life in people with hereditary neuromuscular diseases: an investigation of test–retest agreement with comparison between two generic questionnaires, the Nottingham health profile and the short form-36 items. Neuromuscular Disorders, 16(2), 99–106.
Working Group on Health Outcomes for Older Persons with Multiple Chronic Conditions. (2012). Universal health outcome measures for older persons with multiple chronic conditions. Journal of the American Geriatrics Society, 60(12), 2333–2341.
Ahlstrom, G. (1994). Consequences of muscular dystrophy: impairment, disability, coping and quality of life. PhD thesis dissertation, Uppsala University.
Natterlund, B., & Ahlstrom, G. (2001). Activities of daily living and quality of life in persons with muscular dystrophy. Journal of Rehabilitation Medicine, 33(5), 206–211.
Vincent, K. A., Carr, A. J., Walburn, J., Scott, D. L., & Rose, M. R. (2007). Construction and validation of a quality of life questionnaire for neuromuscular disease (INQoL). Neurology, 68(13), 1051–1057.
Boyer, F. C., Tiffreau, V., Richard, I., & et al. (2011). Patient reported outcome in neuromuscular diseases: The QoL–NMD. Qualitative and quantitative generation of items. Annals of Physical and Rehabilitation Medicine, 54S, e238.
Willis, G. B., & Lessler, J. T. Question Appraisal System: QAS 99. In National Cancer Institute [online]. http://appliedresearch.cancer.gov/areas/cognitive/qas99.pdf. Accessed November 18, 2014
Kitzinger, J. (1995). Qualitative research: Introducing focus groups. British Medical Journal, 311, 299.
World Health Organization (WHO). (2001). The International Classification of Functioning, Disability and Health. Geneva.
Rayens, M. K., & Hahn, E. J. (2000). Building consensus using the policy Delphi method. Policy, Politics, & Nursing Practice, 1(4), 308–315.
Skevington, S. M., Lotfy, M., & O’Connell, A. K. (2004). The World Health Organization’s WHOQOL-BREF quality of life assessment: Psychometric properties and results of the international field trial. A report from the WHOQOL group. Quality of Life Research, 13(2), 299–310.
Collin, C., Wade, D. T., Davies, S., & Horne, V. (1988). The Barthel ADL Index: A reliability study. International Disability Studies, 10(2), 61–63.
Sijtsma, K., & Molenaar, I. W. (2002). Introduction to nonparametric item response theory. Thousand Oaks, CA: Sage.
Mokken, R. J. (1971). A theory and procedure of Scale Analysis. Berlin: De Gruyter.
Wismeijer, A. A., Sijtsma, K., van Assen, M. A., & Vingerhoets, A. J. (2008). A comparative study of the dimensionality of the Self-Concealment Scale using principal components analysis and Mokken Scale analysis. Journal of Personality Assessment, 90(4), 323–334.
Dinno, A. (2009). Exploring the sensitivity of Horn’s parallel analysis to the distributional form of random data. Multivariate Behavioral Research, 44(3), 362–388.
Cronbach, L. (1951). Coefficient alpha and the internal structure of tests. Psychometrika, 16(3), 297–334.
R Core Team. (2013). R: A language and environment for statistical computing. Vienna, Austria. http://www.R-project.org/. Accessed November 18, 2014
van der Ark, L. A. (2007). Mokken Scale analysis in R. Journal of Statistical Software 20(11), 1–19. http://www.jstatsoft.org/v20/i11/. Accessed November 18, 2014.
Arias, C. A., & Cervantes, V. H. (2013). pcaPA: Parallel analysis for ordinal and numeric data using polychoric and Pearson correlations with S3 classes. R package version 1.2. http://CRAN.R-project.org/package=pcaPA. Accessed November 18, 2014.
Falissard, B. (2012). psy: Various procedures used in psychometry. R package version 1.1. http://CRAN.R-project.org/package=psy. Accessed November 18, 2014.
Masters, G. N. (1982). A Rasch model for partial credit scoring. Psychometrika, 47(2), 149–174.
Samejima, F. (1969). Estimation of latent ability using a response pattern of Graded Scores. Psychometrika Monograph Supplement, 34, 1–94.
Stevens, S. S. (1946). On the theory of Scale of Measurement. Science, 103(2684), 677–680.
Thissen, D. (2000). Reliability and measurement precision. In H. E. Wainer (Ed.), Computerized adaptative testing (2nd ed., pp. 159–184). London: Lawrence Erlbaum.
Bos, I., Stallinga, A. H., Middel, B., Kuks, J. B. M., & Wynia, K. (2013). Validation of the ICF core set for neuromuscular diseases. European Journal of Physical and Rehabilitation Medicine, 49, 179–187.
PROMIS Statistical Center Working Group. (2015). The patient reported outcomes measurement information system (PROMIS R) Perspective on: Universally-relevant vs. Disease-Attributed Scales. National Institute of Health [online]. http://www.nihpromis.org/Documents/Universally-Relevant_vs_Disease-Attributed_2014-2-12_final508.pdf. Accessed April 27, 2015.
Cella, D., Yount, S., Rothrock, N., et al. (2007). The patient-reported outcomes measurement information system (PROMIS): Progress of an NIH roadmap cooperative group during its first two years. Medical Care, 45(5 Suppl 1), S3–S11.
Acknowledgments
The authors disclosed receipt of the following financial support for the research, authorship and publication of this article: This project was supported by the French Muscular Dystrophy Association (Association française contre les myopathies/AFM-téléthon) and the Champagne-Ardenne Region (programme ESSAIMAGE; Principal Investigator: François Constant Boyer).
Conflict of interest
The authors have declared no potential conflicts of interest with respect to the research, authorship and publication of this article.
Author information
Authors and Affiliations
Corresponding author
Electronic supplementary material
Below is the link to the electronic supplementary material.
Rights and permissions
About this article
Cite this article
Dany, A., Barbe, C., Rapin, A. et al. Construction of a Quality of Life Questionnaire for slowly progressive neuromuscular disease. Qual Life Res 24, 2615–2623 (2015). https://doi.org/10.1007/s11136-015-1013-8
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.1007/s11136-015-1013-8