Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II

Abstract Background Mucopolysaccharidosis II (MPS II) is a rare lysosomal storage disease characterized by cognitive impairment in most patients. This post hoc analysis evaluated changes in cognitive function, adaptive behavior and functional outcomes in patients with neuronopathic MPS II over time....

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Autores principales:	Karen S. Yee, Yanyu Wu, Magdalena Harrington, Susan E. Waisbren
Formato:	article
Lenguaje:	EN
Publicado:	BMC 2021
Materias:	Adaptive behavior Cognitive ability DAS-II HS-FOCUS Mucopolysaccharidosis II VABS-II Medicine R
Acceso en línea:	https://doaj.org/article/44de2c37ddfc4aaa9ede06d95e6cbadb
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spelling	oai:doaj.org-article:44de2c37ddfc4aaa9ede06d95e6cbadb2021-12-05T12:17:01ZAnalysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II10.1186/s13023-021-02118-31750-1172https://doaj.org/article/44de2c37ddfc4aaa9ede06d95e6cbadb2021-12-01T00:00:00Zhttps://doi.org/10.1186/s13023-021-02118-3https://doaj.org/toc/1750-1172Abstract Background Mucopolysaccharidosis II (MPS II) is a rare lysosomal storage disease characterized by cognitive impairment in most patients. This post hoc analysis evaluated changes in cognitive function, adaptive behavior and functional outcomes in patients with neuronopathic MPS II over time. Fifty-five children with MPS II were enrolled in a 24-month observational study (NCT01822184). The Differential Ability Scales, second edition (DAS-II; early years battery for ages 2 years 6 months to 6 years 11 months, school age battery for ages 7 years to 17 years 11 months), Vineland Adaptive Behavior Scales, second edition (VABS-II) and the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) were performed at baseline and 3-month intervals over 2 years. A subgroup of 38 children with a DAS-II General Conceptual Ability (GCA) score of 55–85 (below average–very low abilities) at any time during the study were included in this analysis. Results Mean (standard deviation [SD]) early years DAS-II GCA score decreased from 73.4 (15.7, n = 22) at baseline to 62.7 (34.9, n = 6) at month 24. For the six patients with early years GCA assessments at baseline and month 24, mean (SD) GCA scores decreased from 72.3 (21.3) at baseline to 62.7 (34.9) at month 24. School age GCA scores were stable over 2 years: mean (SD) 72.4 (11.8, n = 10) at baseline; 74.3 (12.3, n = 8) at month 24. Mean (SD) VABS-II Adaptive Behavior Composite (ABC) scores were stable throughout the study (baseline, 81.8 [11.8, n = 36]; month 24, 81.0 [10.2, n = 13]). Some associations between items and domains of HS-FOCUS (p < 0.05) and DAS-II GCA and VABS-II ABC scores were shown, but there was no clear pattern of changes in HS-FOCUS over 2 years. Conclusions The DAS-II measured changes in cognitive function over 2 years in younger patients with MPS II, whereas cognitive function in older patients remained stable. Further research is required to confirm the content validity of the DAS-II in different patient populations with MPS II. The VABS-II and HS-FOCUS were not sensitive tools for measuring behavioral and functional changes over 2 years. These findings may inform selection of appropriate cognitive and behavioral assessment tools for future studies.Karen S. YeeYanyu WuMagdalena HarringtonSusan E. WaisbrenBMCarticleAdaptive behaviorCognitive abilityDAS-IIHS-FOCUSMucopolysaccharidosis IIVABS-IIMedicineRENOrphanet Journal of Rare Diseases, Vol 16, Iss 1, Pp 1-12 (2021)
institution	DOAJ
collection	DOAJ
language	EN
topic	Adaptive behavior Cognitive ability DAS-II HS-FOCUS Mucopolysaccharidosis II VABS-II Medicine R
spellingShingle	Adaptive behavior Cognitive ability DAS-II HS-FOCUS Mucopolysaccharidosis II VABS-II Medicine R Karen S. Yee Yanyu Wu Magdalena Harrington Susan E. Waisbren Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II
description	Abstract Background Mucopolysaccharidosis II (MPS II) is a rare lysosomal storage disease characterized by cognitive impairment in most patients. This post hoc analysis evaluated changes in cognitive function, adaptive behavior and functional outcomes in patients with neuronopathic MPS II over time. Fifty-five children with MPS II were enrolled in a 24-month observational study (NCT01822184). The Differential Ability Scales, second edition (DAS-II; early years battery for ages 2 years 6 months to 6 years 11 months, school age battery for ages 7 years to 17 years 11 months), Vineland Adaptive Behavior Scales, second edition (VABS-II) and the Hunter Syndrome-Functional Outcomes for Clinical Understanding Scale (HS-FOCUS) were performed at baseline and 3-month intervals over 2 years. A subgroup of 38 children with a DAS-II General Conceptual Ability (GCA) score of 55–85 (below average–very low abilities) at any time during the study were included in this analysis. Results Mean (standard deviation [SD]) early years DAS-II GCA score decreased from 73.4 (15.7, n = 22) at baseline to 62.7 (34.9, n = 6) at month 24. For the six patients with early years GCA assessments at baseline and month 24, mean (SD) GCA scores decreased from 72.3 (21.3) at baseline to 62.7 (34.9) at month 24. School age GCA scores were stable over 2 years: mean (SD) 72.4 (11.8, n = 10) at baseline; 74.3 (12.3, n = 8) at month 24. Mean (SD) VABS-II Adaptive Behavior Composite (ABC) scores were stable throughout the study (baseline, 81.8 [11.8, n = 36]; month 24, 81.0 [10.2, n = 13]). Some associations between items and domains of HS-FOCUS (p < 0.05) and DAS-II GCA and VABS-II ABC scores were shown, but there was no clear pattern of changes in HS-FOCUS over 2 years. Conclusions The DAS-II measured changes in cognitive function over 2 years in younger patients with MPS II, whereas cognitive function in older patients remained stable. Further research is required to confirm the content validity of the DAS-II in different patient populations with MPS II. The VABS-II and HS-FOCUS were not sensitive tools for measuring behavioral and functional changes over 2 years. These findings may inform selection of appropriate cognitive and behavioral assessment tools for future studies.
format	article
author	Karen S. Yee Yanyu Wu Magdalena Harrington Susan E. Waisbren
author_facet	Karen S. Yee Yanyu Wu Magdalena Harrington Susan E. Waisbren
author_sort	Karen S. Yee
title	Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II
title_short	Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II
title_full	Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II
title_fullStr	Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II
title_full_unstemmed	Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II
title_sort	analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis ii
publisher	BMC
publishDate	2021
url	https://doaj.org/article/44de2c37ddfc4aaa9ede06d95e6cbadb
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Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II

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