Skip to main content
Top
Gepubliceerd in: Quality of Life Research 4/2013

01-05-2013 | Original Paper

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

Auteurs: Ingela Wiklund, Mireia Raluy-Callado, Donald E. Stull, Yvonne Jangelind, David A. H. Whiteman, Wen-Hung Chen

Gepubliceerd in: Quality of Life Research | Uitgave 4/2013

Log in om toegang te krijgen
share
DELEN

Deel dit onderdeel of sectie (kopieer de link)

  • Optie A:
    Klik op de rechtermuisknop op de link en selecteer de optie “linkadres kopiëren”
  • Optie B:
    Deel de link per e-mail

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.
Literatuur
1.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
2.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
3.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
4.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
5.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
6.
go back to reference 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. 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 HOSthe hunter outcome survey.
7.
go back to reference Pascolini, D., & Smith, A. (2009). Hearing impairment in 2008: A compilation of available epidemiological studies. International Journal of Audiology, 48(7), 473–485.PubMedCrossRef Pascolini, D., & Smith, A. (2009). Hearing impairment in 2008: A compilation of available epidemiological studies. International Journal of Audiology, 48(7), 473–485.PubMedCrossRef
9.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
10.
go back to reference 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. 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.
go back to reference 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. 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.
go back to reference 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 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
13.
go back to reference Landgraf, J. M., Abetz, L. N., & Ware, J. E. (1999). The CHQ user’s manual. Boston: The Health Institute, New England Medical Center. Landgraf, J. M., Abetz, L. N., & Ware, J. E. (1999). The CHQ user’s manual. Boston: The Health Institute, New England Medical Center.
14.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
15.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
16.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
17.
go back to reference StataCorp. (2009). Stata statistical software: Release 11. College Station, TX: StataCorp LP. StataCorp. (2009). Stata statistical software: Release 11. College Station, TX: StataCorp LP.
18.
go back to reference Cronbach, L. (1951). Coefficient alpha and the internal structure of tests. Psychometrika, 16, 297–334.CrossRef Cronbach, L. (1951). Coefficient alpha and the internal structure of tests. Psychometrika, 16, 297–334.CrossRef
19.
go back to reference 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. 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.
20.
go back to reference Nunnally, J. C., & Bernstein, I. H. (1994). Psychometric theory (3rd ed.). New York: McGraw-Hill. Nunnally, J. C., & Bernstein, I. H. (1994). Psychometric theory (3rd ed.). New York: McGraw-Hill.
21.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
22.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
23.
go back to reference 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.PubMedCrossRef 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.PubMedCrossRef
24.
go back to reference 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 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
25.
go back to reference 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 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
Metagegevens
Titel
The Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) Questionnaire: evaluation of measurement properties
Auteurs
Ingela Wiklund
Mireia Raluy-Callado
Donald E. Stull
Yvonne Jangelind
David A. H. Whiteman
Wen-Hung Chen
Publicatiedatum
01-05-2013
Uitgeverij
Springer Netherlands
Gepubliceerd in
Quality of Life Research / Uitgave 4/2013
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-012-0196-5

Andere artikelen Uitgave 4/2013

Quality of Life Research 4/2013 Naar de uitgave