Health-Related Quality of Life in Patients with Spinocerebellar Ataxia: a Validation Study of the EQ-5D-3L

  1. Buchholz, Maresa
  2. Weber, Niklas
  3. Rädke, Anika
  4. Faber, Jennifer
  5. Schmitz-Hübsch, Tanja
  6. Jacobi, Heike
  7. Xie, Feng
  8. Klockgether, Thomas
  9. Michalowsky, Bernhard
  10. The EUROSCA study group
  11. du Montcel, Sophie Tezenas
  12. Bauer, Peter
  13. Giunti, Paola
  14. Cook, Arron
  15. Labrum, Robyn
  16. Parkinson, Michael H.
  17. Durr, Alexandra
  18. Brice, Alexis
  19. Charles, Perrine
  20. Marelli, Cecilia
  21. Mariotti, Caterina
  22. Nanetti, Lorenzo
  23. Panzeri, Marta
  24. Rakowicz, Maria
  25. Sulek, Anna
  26. Sobanska, Anna
  27. Schöls, Ludger
  28. Hengel, Holger
  29. Baliko, Laszlo
  30. Melegh, Bela
  31. Filla, Alessandro
  32. Antenora, Antonella
  33. Infante, Jon
  34. Berciano, José
  35. van de Warrenburg, Bart P.
  36. Timmann, Dagmar
  37. Szymanski, Sandra
  38. Boesch, Sylvia
  39. Kang, Jun-Suk
  40. Pandolfo, Massimo
  41. Schulz, Jörg B.
  42. Molho, Sonia
  43. Diallo, Alhassane
  44. The ESMI study group
  45. Hübener-Schmid, Jeanette
  46. Santana, Magda
  47. Grobe-Einsler, Marcus
  48. Koyak, Berkan
  49. Raposo, Mafalda
  50. Lima, Manuela
  51. Garcia-Moreno, Hector
  52. Giunti, Paola
  53. de Almeida, Luís Pereira
  54. van de Warrenburg, Bart
  55. van Gaalen, Judith
  56. Timmann, Dagmar
  57. Thieme, Andreas
  58. Reetz, Kathrin
  59. Dogan, Imis
  60. Wilke, Carlo
  61. Schöls, Ludger
  62. Riess, Olaf
  63. Synofzik, Matthis
  64. de Vries, Jeroen
  65. Infante, Jon
  66. Gulin, Oz
  67. Joers, James
  68. Onyike, Chiadikaobi
  69. Povazan, Michal
  70. Ratai, Eva-Maria
  71. Schmahmann, Jeremy

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1000 Titel
  • Health-Related Quality of Life in Patients with Spinocerebellar Ataxia: a Validation Study of the EQ-5D-3L
1000 Autor/in
  1. Buchholz, Maresa |
  2. Weber, Niklas |
  3. Rädke, Anika |
  4. Faber, Jennifer |
  5. Schmitz-Hübsch, Tanja |
  6. Jacobi, Heike |
  7. Xie, Feng |
  8. Klockgether, Thomas |
  9. Michalowsky, Bernhard |
  10. The EUROSCA study group |
  11. du Montcel, Sophie Tezenas |
  12. Bauer, Peter |
  13. Giunti, Paola |
  14. Cook, Arron |
  15. Labrum, Robyn |
  16. Parkinson, Michael H. |
  17. Durr, Alexandra |
  18. Brice, Alexis |
  19. Charles, Perrine |
  20. Marelli, Cecilia |
  21. Mariotti, Caterina |
  22. Nanetti, Lorenzo |
  23. Panzeri, Marta |
  24. Rakowicz, Maria |
  25. Sulek, Anna |
  26. Sobanska, Anna |
  27. Schöls, Ludger |
  28. Hengel, Holger |
  29. Baliko, Laszlo |
  30. Melegh, Bela |
  31. Filla, Alessandro |
  32. Antenora, Antonella |
  33. Infante, Jon |
  34. Berciano, José |
  35. van de Warrenburg, Bart P. |
  36. Timmann, Dagmar |
  37. Szymanski, Sandra |
  38. Boesch, Sylvia |
  39. Kang, Jun-Suk |
  40. Pandolfo, Massimo |
  41. Schulz, Jörg B. |
  42. Molho, Sonia |
  43. Diallo, Alhassane |
  44. The ESMI study group |
  45. Hübener-Schmid, Jeanette |
  46. Santana, Magda |
  47. Grobe-Einsler, Marcus |
  48. Koyak, Berkan |
  49. Raposo, Mafalda |
  50. Lima, Manuela |
  51. Garcia-Moreno, Hector |
  52. Giunti, Paola |
  53. de Almeida, Luís Pereira |
  54. van de Warrenburg, Bart |
  55. van Gaalen, Judith |
  56. Timmann, Dagmar |
  57. Thieme, Andreas |
  58. Reetz, Kathrin |
  59. Dogan, Imis |
  60. Wilke, Carlo |
  61. Schöls, Ludger |
  62. Riess, Olaf |
  63. Synofzik, Matthis |
  64. de Vries, Jeroen |
  65. Infante, Jon |
  66. Gulin, Oz |
  67. Joers, James |
  68. Onyike, Chiadikaobi |
  69. Povazan, Michal |
  70. Ratai, Eva-Maria |
  71. Schmahmann, Jeremy |
1000 Verlag Springer US
1000 Erscheinungsjahr 2023
1000 Publikationstyp
  1. Artikel |
1000 Online veröffentlicht
  • 2023-09-15
1000 Erschienen in
1000 Quellenangabe
  • 23(3):1020-1030
1000 Copyrightjahr
  • 2023
1000 Lizenz
1000 Verlagsversion
  • https://doi.org/10.1007/s12311-023-01597-3 |
  • https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11102408/ |
1000 Publikationsstatus
1000 Begutachtungsstatus
1000 Sprache der Publikation
1000 Abstract/Summary
  • <jats:title>Abstract</jats:title><jats:p>Although health-related quality of life (HRQoL) has developed into a crucial outcome parameter in clinical research, evidence of the EQ-5D-3L validation performance is lacking in patients with spinocerebellar ataxia (SCA) types 1, 2, 3, and 6. The objective of this study is to assess the acceptability, validity, reliability, and responsiveness of the EQ-5D-3L. For <jats:italic>n</jats:italic> = 842 predominantly European SCA patients of two longitudinal cohort studies, the EQ-5D-3L, PHQ-9 (Patient Health Questionnaire), and ataxia-specific clinical assessments (SARA: Scale for Assessment and Rating of Ataxia; ADL: activities of daily living as part of Friedreich’s Ataxia Rating Scale; INAS: Inventory of Non-Ataxia Signs) were assessed at baseline and multiple annual follow-ups. The EQ-5D-3L was evaluated regarding acceptability, distribution properties, convergent and known-groups validity, test-retest reliability, and effect size measures to analyze health changes. The non-item response was low (EQ-5D-3L index: 0.8%; EQ-VAS: 3.4%). Ceiling effects occurred in 9.9% (EQ-5D-3L) and 3.0% (EQ-VAS) with a mean EQ-5D-3L index of 0.65 ± 0.21. In total, convergent validity showed moderate to strong Spearman’s rho (<jats:italic>r</jats:italic><jats:sub>s</jats:sub> &gt; 0.3) coefficients comparing EQ-5D-3L and EQ-VAS with PHQ-9, SARA, ADL, and INAS. EQ-5D-3L could discriminate between groups of age, SARA, ADL, and INAS. Intra-class correlation coefficients (EQ-5D-3L<jats:sub>ICC</jats:sub>: 0.95/EQ-VAS<jats:sub>ICC</jats:sub>: 0.88) and Kappa statistics (range 0.44 to 0.93 for EQ-5D-3L items) indicated tolerable reliability. EQ-5D-3L shows small (effect size &lt; 0.3) to moderate (effect size 0.3–0.59) health changes regarding ataxia severity. The analysis confirms an acceptable, reliable, valid, and responsive recommended EQ-5D-3L in SCA patients, measuring the HRQoL adequately, besides well-established clinical instruments.</jats:p>
1000 Sacherschließung
lokal Female [MeSH]
lokal Health-related quality of life
lokal Surveys and Questionnaires/standards [MeSH]
lokal Aged [MeSH]
lokal EQ-5D-3L
lokal Adult [MeSH]
lokal Humans [MeSH]
lokal Longitudinal Studies [MeSH]
lokal Middle Aged [MeSH]
lokal Spinocerebellar ataxia
lokal Cohort Studies [MeSH]
lokal Male [MeSH]
lokal Spinocerebellar Ataxias/psychology [MeSH]
lokal Reproducibility of Results [MeSH]
lokal Spinocerebellar Ataxias/diagnosis [MeSH]
lokal Psychometrics/standards [MeSH]
lokal Validation
lokal Quality of Life [MeSH]
lokal Research
lokal Activities of Daily Living [MeSH]
lokal Psychometrics/methods [MeSH]
1000 Fächerklassifikation (DDC)
1000 Liste der Beteiligten
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  1. Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. (DZNE) in der Helmholtz-Gemeinschaft |
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    1000 Förderer Deutsches Zentrum für Neurodegenerative Erkrankungen e.V. (DZNE) in der Helmholtz-Gemeinschaft |
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