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The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire: evaluation of measurement properties

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Abstract

Purpose

This study was to conduct the psychometric validation of the patient and parent versions of the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS).

Methods

Data collected in a 53-week placebo-controlled multinational trial were used to evaluate item performance and reliability, validity, and ability to detect change of the six HS-FOCUS function domains.

Results

HS-FOCUS was completed by 49 patients above 12 years old and 84 parents. Floor effects and high average inter-item correlations suggested that some items were less informative or redundant. For both patients and parents, the internal consistency and test–retest reliability met the >0.70 criteria for all domains except for the breathing, sleeping, and schooling/work in patients. Construct validity showed moderate to high correlations with CHAQ, CHQ, and HUI3 in activity-related concepts. Significant differences in domain scores were found in most domains among severity in disability measured by CHAQ DIS. Significant differences in HS-FOCUS change scores were found in patients whose CHAQ DIS score also changed.

Conclusions

Psychometric validation of the HS-FOCUS demonstrates it is a reliable, valid, and responsive instrument that can be applied in clinical trials or disease registries. Findings on the individual item performance suggest some items could be removed without compromising its validity.

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Abbreviations

6MWT:

Six-minute walk test

ANCOVA:

Analysis of covariance

ANOVA:

Analysis of variance

BP:

Bodily Pain

CH:

Change in Health

CHAQ:

Childhood Health Assessment Questionnaire

CHQ:

Childhood Health Questionnaire

CHQ-CF:

Child Health Questionnaire-Child Form

CHQ-PF:

Child Health Questionnaire-Parent Form

DIS:

Disability Index Score

FA:

Family Activities

FC:

Family Cohesion

FEV:

Forced expiratory volume

FIM:

Functional Independence Measure

FVC:

Forced vital capacity

GAG:

Glycosaminoglycans

GBE:

Global Behavior

GH:

General Health Perceptions

HRQL:

Health-related quality of life

HS:

Hunter syndrome

HS-FOCUS:

Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale

HUI:

Health Utilities Index

I2S:

Iduronate-2-sulfatase

ICC:

Intraclass correlation coefficient

MH:

Mental Health

MPS II:

Mucopolysaccharidosis type II

PE:

Parental Impact–Emotional

PF:

Physical Functioning

PODCI:

Pediatric Outcomes Data Collection Instrument

PRO:

Patient-reported outcome

PT:

Parental Impact–Time

RB:

Role/Social–Behavioral

RE:

Role/Social–Emotional

REB:

Role/Social–Emotional and the Role/Social–Behavioral

RP:

Role/Social–Physical

SD:

Standard deviation

SE:

Self Esteem

WHO-ICIDH:

World Health Organization International Classification of Impairment, Disability and Handicap

References

  1. Alcalde-Martin, C., Muro-Tudelilla, J. M., Cancho-Candela, R., Gutierrez-Solana, L. G., Pintos-Morell, G., Marti-Herrero, M., et al. (2010). First experience of enzyme replacement therapy with idursulfase in Spanish patients with Hunter syndrome under 5 years of age: Case observations from the Hunter Outcome Survey (HOS). European Journal of Medical Genetics, 53(6), 371–377.

    Article  PubMed  CAS  Google Scholar 

  2. Martin, R., Beck, M., Eng, C., Giugliani, R., Harmatz, P., Munoz, V., et al. (2008). Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome). Pediatrics, 121(2), e377–e386.

    Article  PubMed  Google Scholar 

  3. Muenzer, J., Beck, M., Eng, C. M., Escolar, M. L., Giugliani, R., Guffon, N. H., et al. (2009). Multidisciplinary management of Hunter syndrome. Pediatrics, 124(6), e1228–e1239.

    Article  PubMed  Google Scholar 

  4. Young, I. D., & Harper, P. S. (1982). Mild form of Hunter’s syndrome: Clinical delineation based on 31 cases. Archives of Disease in Childhood, 57(11), 828–836.

    Article  PubMed  CAS  Google Scholar 

  5. Young, I. D., & Harper, P. S. (1983). The natural history of the severe form of Hunter’s syndrome: A study based on 52 cases. Developmental Medicine and Child Neurology, 25(4), 481–489.

    Article  PubMed  CAS  Google Scholar 

  6. Keilmann, A., Nakarat, T., Bruce, I. A., Molter, D., Malm, G., & on behalf of the HOS Investigators. Hearing loss in patients with mucopolysaccharidosis II: Data from HOS—the hunter outcome survey.

  7. Pascolini, D., & Smith, A. (2009). Hearing impairment in 2008: A compilation of available epidemiological studies. International Journal of Audiology, 48(7), 473–485.

    Article  PubMed  Google Scholar 

  8. World Health Organization (WHO). (1999). WHO ear and hearing disorders survey protocol for a population-based survey of prevalence and causes of deafness and hearing impairment and other ear disorders. Retrieved April 14, 2011, from http://whqlibdoc.who.int/hq/1999/WHO_PBD_PDH_99.8(1).pdf.

  9. Singh, G., Athreya, B. H., Fries, J. F., & Goldsmith, D. P. (1994). Measurement of health status in children with juvenile rheumatoid arthritis. Arthritis and Rheumatism, 37(12), 1761–1769.

    Article  PubMed  CAS  Google Scholar 

  10. Tran, K. T., Gold, K. F., Stephens, J. M., Kimura, A., & Muenzer, J. The development and validation of the hunter syndrome—functional outcomes for clinical understanding scale.

  11. Abt. Associates. (2004). Clinical trials. Confirmatory validation study of the CHAQ. Video documentation of functional tasks, and development of a new supplement Questionnaire to assess functional status in hunter syndrome. Final Report.

  12. Giannini, E. H., Ruperto, N., Ravelli, A., Lovell, D. J., Felson, D. T., & Martini, A. (1997). Preliminary definition of improvement in juvenile arthritis. Arthritis and Rheumatism, 40(7), 1202–1209.

    PubMed  CAS  Google Scholar 

  13. Landgraf, J. M., Abetz, L. N., & Ware, J. E. (1999). The CHQ user’s manual. Boston: The Health Institute, New England Medical Center.

    Google Scholar 

  14. Valayannopoulos, V., de Blic, J., Mahlaoui, N., Stos, B., Jaubert, F., Bonnet, D., et al. (2010). Laronidase for cardiopulmonary disease in Hurler syndrome 12 years after bone marrow transplantation. Pediatrics, 126(5), e1242–e1247.

    Article  PubMed  Google Scholar 

  15. McGovern, M. M., Wasserstein, M. P., Giugliani, R., Bembi, B., Vanier, M. T., Mengel, E., et al. (2008). A prospective, cross-sectional survey study of the natural history of Niemann-Pick disease type B. Pediatrics, 122(2), e341–e349.

    Article  PubMed  Google Scholar 

  16. Horsman, J., Furlong, W., Feeny, D., & Torrance, G. (2003). The health utilities index (HUI): Concepts, measurement properties and applications. Health and Quality of Life Outcomes, 1, 54.

    Article  PubMed  Google Scholar 

  17. StataCorp. (2009). Stata statistical software: Release 11. College Station, TX: StataCorp LP.

    Google Scholar 

  18. Cronbach, L. (1951). Coefficient alpha and the internal structure of tests. Psychometrika, 16, 297–334.

    Article  Google Scholar 

  19. Hays, R. D., & Revicki, D. A. (2005). Assessing reliability and validity of measurement in clinical trials. In P. Fayers & R. D. Hays (Eds.), Quality of life assessment in clinical trials: Methods and practice (2nd ed.). New York: Oxford University Press.

    Google Scholar 

  20. Nunnally, J. C., & Bernstein, I. H. (1994). Psychometric theory (3rd ed.). New York: McGraw-Hill.

    Google Scholar 

  21. Dempster, H., Porepa, M., Young, N., & Feldman, B. M. (2001). The clinical meaning of functional outcome scores in children with juvenile arthritis. Arthritis and Rheumatism, 44(8), 1768–1774.

    Article  PubMed  CAS  Google Scholar 

  22. Leidy, N. K., Revicki, D. A., & Geneste, B. (1999). Recommendations for evaluating the validity of quality of life claims for labeling and promotion. Value Health, 2(2), 113–127.

    Article  PubMed  CAS  Google Scholar 

  23. Rutten-van Molken, M., Roos, B., & Van Noord, J. A. (1999). An empirical comparison of the St George’s Respiratory Questionnaire (SGRQ) and the Chronic Respiratory Disease Questionnaire (CRQ) in a clinical trial setting. Thorax, 54(11), 995–1003.

    Article  PubMed  CAS  Google Scholar 

  24. Daltroy, L. H., Liang, M. H., Fossel, A. H., & Goldberg, M. J. (1998). The POSNA pediatric musculoskeletal functional health questionnaire: Report on reliability, validity, and sensitivity to change. Pediatric Outcomes Instrument Development Group. Pediatric Orthopaedic Society of North America. Journal of Pediatric Orthopedics, 18(5), 561–571.

    PubMed  CAS  Google Scholar 

  25. Keith, R. A., Granger, C. V., Hamilton, B. B., & Sherwin, F. S. (1987). The functional independence measure: A new tool for rehabilitation. Advances in Clinical Rehabilitation, 1, 6–18.

    PubMed  CAS  Google Scholar 

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Author information

Authors and Affiliations

  1. United BioSource Corporation, 26-28 Hammersmith Grove, London, W6 7HA, UK

    Ingela Wiklund & Mireia Raluy-Callado

  2. RTI Health Solutions, The Pavilion, Towers Business Park, Wilmslow Road, Didsbury, Manchester, M20 2LS, UK

    Donald E. Stull

  3. Stockholm, Sweden

    Yvonne Jangelind

  4. Shire AG, Business Park Terre-Bonne, Route de Crassier 7, 1262, Eysins, Switzerland

    David A. H. Whiteman

  5. United BioSource Corporation, 7101 Wisconsin Ave. Suite 600, Bethesda, MD, 20814, USA

    Wen-Hung Chen

Authors
  1. Ingela Wiklund
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  2. Mireia Raluy-Callado
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  3. Donald E. Stull
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  4. Yvonne Jangelind
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  5. David A. H. Whiteman
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  6. Wen-Hung Chen
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Corresponding author

Correspondence to Ingela Wiklund.

Additional information

Donald E. Stull was a United BioSource Corporation employee when this work was conducted.

Yvonne Jangelind was a Shire employee when this work was conducted.

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Wiklund, I., Raluy-Callado, M., Stull, D.E. et al. The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire: evaluation of measurement properties. Qual Life Res 22, 875–884 (2013). https://doi.org/10.1007/s11136-012-0196-5

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  • DOI: https://doi.org/10.1007/s11136-012-0196-5

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