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Auteur Elizabeth BERRY-KRAVIS |
Documents disponibles écrits par cet auteur (28)
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The NIH Toolbox Cognitive Battery for intellectual disabilities: three preliminary studies and future directions / D. HESSL in Journal of Neurodevelopmental Disorders, 8-1 (December 2016)
[article]
Titre : The NIH Toolbox Cognitive Battery for intellectual disabilities: three preliminary studies and future directions Type de document : Texte imprimé et/ou numérique Auteurs : D. HESSL, Auteur ; Stephanie M. SANSONE, Auteur ; Elizabeth BERRY-KRAVIS, Auteur ; K. RILEY, Auteur ; K. F. WIDAMAN, Auteur ; Leonard ABBEDUTO, Auteur ; A. SCHNEIDER, Auteur ; J. COLEMAN, Auteur ; D. OAKLANDER, Auteur ; K. C. RHODES, Auteur ; R. C. GERSHON, Auteur Article en page(s) : p.35 Langues : Anglais (eng) Mots-clés : Assessment Cognition Down syndrome FMR1 gene Fragile X syndrome Outcome measures Index. décimale : PER Périodiques Résumé : BACKGROUND: Recent advances in understanding molecular and synaptic mechanisms of intellectual disabilities (ID) in fragile X syndrome (FXS) and Down syndrome (DS) through animal models have led to targeted controlled trials with pharmacological agents designed to normalize these underlying mechanisms and improve clinical outcomes. However, several human clinical trials have failed to demonstrate efficacy of these targeted treatments to improve surrogate behavioral endpoints. Because the ultimate index of disease modification in these disorders is amelioration of ID, the validation of cognitive measures for tracking treatment response is essential. Here, we present preliminary research to validate the National Institutes of Health Toolbox Cognitive Battery (NIH-TCB) for ID. METHODS: We completed three pilot studies of patients with FXS (total n = 63; mean age 19.3 +/- 8.3 years, mean mental age 5.3 +/- 1.6 years), DS (n = 47; mean age 16.1 +/- 6.2, mean mental age 5.4 +/- 2.0), and idiopathic ID (IID; n = 16; mean age 16.1 +/- 5.0, mean mental age 6.6 +/- 2.3) measuring processing speed, executive function, episodic memory, word/letter reading, receptive vocabulary, and working memory using the web-based NIH-TB-CB, addressing feasibility, test-retest reliability, construct validity, ecological validity, and syndrome differences and profiles. RESULTS: Feasibility was good to excellent (>/=80 % of participants with valid scores) for above mental age 4 years for all tests except list sorting (working memory). Test-retest stability was good to excellent, and convergent validity was similar to or better than results obtained from typically developing children in the normal sample for executive function and language measures. Examination of ecological validity revealed moderate to very strong correlations between the NIH-TCB composite and adaptive behavior and full-scale IQ measures. Syndrome/group comparisons demonstrated significant deficits for the FXS and DS groups relative to IID on attention and inhibitory control, a significant reading weakness for FXS, and a receptive vocabulary weakness for DS. CONCLUSIONS: The NIH-TCB has potential for assessing important dimensions of cognition in persons with ID, and several tests may be useful for tracking response to intervention. However, more extensive psychometric studies, evaluation of the NIH-TCB's sensitivity to change, both developmentally and in the context of treatment, and perhaps establishing links to brain function in these populations, are required to determine the true utility of the battery as a set of outcome measures. En ligne : http://dx.doi.org/10.1186/s11689-016-9167-4 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=349
in Journal of Neurodevelopmental Disorders > 8-1 (December 2016) . - p.35[article] The NIH Toolbox Cognitive Battery for intellectual disabilities: three preliminary studies and future directions [Texte imprimé et/ou numérique] / D. HESSL, Auteur ; Stephanie M. SANSONE, Auteur ; Elizabeth BERRY-KRAVIS, Auteur ; K. RILEY, Auteur ; K. F. WIDAMAN, Auteur ; Leonard ABBEDUTO, Auteur ; A. SCHNEIDER, Auteur ; J. COLEMAN, Auteur ; D. OAKLANDER, Auteur ; K. C. RHODES, Auteur ; R. C. GERSHON, Auteur . - p.35.
Langues : Anglais (eng)
in Journal of Neurodevelopmental Disorders > 8-1 (December 2016) . - p.35
Mots-clés : Assessment Cognition Down syndrome FMR1 gene Fragile X syndrome Outcome measures Index. décimale : PER Périodiques Résumé : BACKGROUND: Recent advances in understanding molecular and synaptic mechanisms of intellectual disabilities (ID) in fragile X syndrome (FXS) and Down syndrome (DS) through animal models have led to targeted controlled trials with pharmacological agents designed to normalize these underlying mechanisms and improve clinical outcomes. However, several human clinical trials have failed to demonstrate efficacy of these targeted treatments to improve surrogate behavioral endpoints. Because the ultimate index of disease modification in these disorders is amelioration of ID, the validation of cognitive measures for tracking treatment response is essential. Here, we present preliminary research to validate the National Institutes of Health Toolbox Cognitive Battery (NIH-TCB) for ID. METHODS: We completed three pilot studies of patients with FXS (total n = 63; mean age 19.3 +/- 8.3 years, mean mental age 5.3 +/- 1.6 years), DS (n = 47; mean age 16.1 +/- 6.2, mean mental age 5.4 +/- 2.0), and idiopathic ID (IID; n = 16; mean age 16.1 +/- 5.0, mean mental age 6.6 +/- 2.3) measuring processing speed, executive function, episodic memory, word/letter reading, receptive vocabulary, and working memory using the web-based NIH-TB-CB, addressing feasibility, test-retest reliability, construct validity, ecological validity, and syndrome differences and profiles. RESULTS: Feasibility was good to excellent (>/=80 % of participants with valid scores) for above mental age 4 years for all tests except list sorting (working memory). Test-retest stability was good to excellent, and convergent validity was similar to or better than results obtained from typically developing children in the normal sample for executive function and language measures. Examination of ecological validity revealed moderate to very strong correlations between the NIH-TCB composite and adaptive behavior and full-scale IQ measures. Syndrome/group comparisons demonstrated significant deficits for the FXS and DS groups relative to IID on attention and inhibitory control, a significant reading weakness for FXS, and a receptive vocabulary weakness for DS. CONCLUSIONS: The NIH-TCB has potential for assessing important dimensions of cognition in persons with ID, and several tests may be useful for tracking response to intervention. However, more extensive psychometric studies, evaluation of the NIH-TCB's sensitivity to change, both developmentally and in the context of treatment, and perhaps establishing links to brain function in these populations, are required to determine the true utility of the battery as a set of outcome measures. En ligne : http://dx.doi.org/10.1186/s11689-016-9167-4 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=349 Updated report on tools to measure outcomes of clinical trials in fragile X syndrome / Dejan B. BUDIMIROVIC in Journal of Neurodevelopmental Disorders, 9-1 (December 2017)
[article]
Titre : Updated report on tools to measure outcomes of clinical trials in fragile X syndrome Type de document : Texte imprimé et/ou numérique Auteurs : Dejan B. BUDIMIROVIC, Auteur ; Elizabeth BERRY-KRAVIS, Auteur ; C. A. ERICKSON, Auteur ; S. S. HALL, Auteur ; D. HESSL, Auteur ; A. L. REISS, Auteur ; M. K. KING, Auteur ; Leonard ABBEDUTO, Auteur ; W. E. KAUFMANN, Auteur Article en page(s) : p.14 Langues : Anglais (eng) Mots-clés : Autism spectrum disorder Clinical trials Fragile X syndrome Intellectual disability Outcome measures Index. décimale : PER Périodiques Résumé : OBJECTIVE: Fragile X syndrome (FXS) has been the neurodevelopmental disorder with the most active translation of preclinical breakthroughs into clinical trials. This process has led to a critical assessment of outcome measures, which resulted in a comprehensive review published in 2013. Nevertheless, the disappointing outcome of several recent phase III drug trials in FXS, and parallel efforts at evaluating behavioral endpoints for trials in autism spectrum disorder (ASD), has emphasized the need for re-assessing outcome measures and revising recommendations for FXS. METHODS: After performing an extensive database search (PubMed, Food and Drug Administration (FDA)/National Institutes of Health (NIH)'s www.ClinicalTrials.gov, etc.) to determine progress since 2013, members of the Working Groups who published the 2013 Report evaluated the available outcome measures for FXS and related neurodevelopmental disorders using the COSMIN grading system of levels of evidence. The latter has also been applied to a British survey of endpoints for ASD. In addition, we also generated an informal classification of outcome measures for use in FXS intervention studies as instruments appropriate to detect shorter- or longer-term changes. RESULTS: To date, a total of 22 double-blind controlled clinical trials in FXS have been identified through www.ClinicalTrials.gov and an extensive literature search. The vast majority of these FDA/NIH-registered clinical trials has been completed between 2008 and 2015 and has targeted the core excitatory/inhibitory imbalance present in FXS and other neurodevelopmental disorders. Limited data exist on reliability and validity for most tools used to measure cognitive, behavioral, and other problems in FXS in these trials and other studies. Overall, evidence for most tools supports a moderate tool quality grading. Data on sensitivity to treatment, currently under evaluation, could improve ratings for some cognitive and behavioral tools. Some progress has also been made at identifying promising biomarkers, mainly on blood-based and neurophysiological measures. CONCLUSION: Despite the tangible progress in implementing clinical trials in FXS, the increasing data on measurement properties of endpoints, and the ongoing process of new tool development, the vast majority of outcome measures are at the moderate quality level with limited information on reliability, validity, and sensitivity to treatment. This situation is not unique to FXS, since reviews of endpoints for ASD have arrived at similar conclusions. These findings, in conjunction with the predominance of parent-based measures particularly in the behavioral domain, indicate that endpoint development in FXS needs to continue with an emphasis on more objective measures (observational, direct testing, biomarkers) that reflect meaningful improvements in quality of life. A major continuous challenge is the development of measurement tools concurrently with testing drug safety and efficacy in clinical trials. En ligne : http://dx.doi.org/10.1186/s11689-017-9193-x Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=350
in Journal of Neurodevelopmental Disorders > 9-1 (December 2017) . - p.14[article] Updated report on tools to measure outcomes of clinical trials in fragile X syndrome [Texte imprimé et/ou numérique] / Dejan B. BUDIMIROVIC, Auteur ; Elizabeth BERRY-KRAVIS, Auteur ; C. A. ERICKSON, Auteur ; S. S. HALL, Auteur ; D. HESSL, Auteur ; A. L. REISS, Auteur ; M. K. KING, Auteur ; Leonard ABBEDUTO, Auteur ; W. E. KAUFMANN, Auteur . - p.14.
Langues : Anglais (eng)
in Journal of Neurodevelopmental Disorders > 9-1 (December 2017) . - p.14
Mots-clés : Autism spectrum disorder Clinical trials Fragile X syndrome Intellectual disability Outcome measures Index. décimale : PER Périodiques Résumé : OBJECTIVE: Fragile X syndrome (FXS) has been the neurodevelopmental disorder with the most active translation of preclinical breakthroughs into clinical trials. This process has led to a critical assessment of outcome measures, which resulted in a comprehensive review published in 2013. Nevertheless, the disappointing outcome of several recent phase III drug trials in FXS, and parallel efforts at evaluating behavioral endpoints for trials in autism spectrum disorder (ASD), has emphasized the need for re-assessing outcome measures and revising recommendations for FXS. METHODS: After performing an extensive database search (PubMed, Food and Drug Administration (FDA)/National Institutes of Health (NIH)'s www.ClinicalTrials.gov, etc.) to determine progress since 2013, members of the Working Groups who published the 2013 Report evaluated the available outcome measures for FXS and related neurodevelopmental disorders using the COSMIN grading system of levels of evidence. The latter has also been applied to a British survey of endpoints for ASD. In addition, we also generated an informal classification of outcome measures for use in FXS intervention studies as instruments appropriate to detect shorter- or longer-term changes. RESULTS: To date, a total of 22 double-blind controlled clinical trials in FXS have been identified through www.ClinicalTrials.gov and an extensive literature search. The vast majority of these FDA/NIH-registered clinical trials has been completed between 2008 and 2015 and has targeted the core excitatory/inhibitory imbalance present in FXS and other neurodevelopmental disorders. Limited data exist on reliability and validity for most tools used to measure cognitive, behavioral, and other problems in FXS in these trials and other studies. Overall, evidence for most tools supports a moderate tool quality grading. Data on sensitivity to treatment, currently under evaluation, could improve ratings for some cognitive and behavioral tools. Some progress has also been made at identifying promising biomarkers, mainly on blood-based and neurophysiological measures. CONCLUSION: Despite the tangible progress in implementing clinical trials in FXS, the increasing data on measurement properties of endpoints, and the ongoing process of new tool development, the vast majority of outcome measures are at the moderate quality level with limited information on reliability, validity, and sensitivity to treatment. This situation is not unique to FXS, since reviews of endpoints for ASD have arrived at similar conclusions. These findings, in conjunction with the predominance of parent-based measures particularly in the behavioral domain, indicate that endpoint development in FXS needs to continue with an emphasis on more objective measures (observational, direct testing, biomarkers) that reflect meaningful improvements in quality of life. A major continuous challenge is the development of measurement tools concurrently with testing drug safety and efficacy in clinical trials. En ligne : http://dx.doi.org/10.1186/s11689-017-9193-x Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=350 Vocabulary comprehension in adults with fragile X syndrome (FXS) / A. HOFFMANN in Journal of Neurodevelopmental Disorders, 11-1 (December 2019)
[article]
Titre : Vocabulary comprehension in adults with fragile X syndrome (FXS) Type de document : Texte imprimé et/ou numérique Auteurs : A. HOFFMANN, Auteur ; S. E. KRAUSE, Auteur ; J. WUU, Auteur ; S. LEURGANS, Auteur ; S. J. GUTER, Auteur ; S. S. BLOCK, Auteur ; J. SALT, Auteur ; E. COOK, Auteur ; D. M. MAINO, Auteur ; Elizabeth BERRY-KRAVIS, Auteur Article en page(s) : 25 p. Langues : Anglais (eng) Mots-clés : Adults Cognition Comprehension Fragile X syndrome Language Vocabulary Index. décimale : PER Périodiques Résumé : BACKGROUND: Receptive and expressive vocabulary in adult and adolescent males with fragile X syndrome (FXS) have been shown as significantly lower than their chronological age; however, receptive vocabulary has been considered a strength relative to mental age. This has not been formally examined, however, and data are needed to compare receptive vocabulary with other language skills and with mental age in individuals with FXS. This is especially important as vocabulary measures are sometimes used as a proxy to estimate language ability. METHODS: This preliminary study examined receptive vocabulary, global language, and cognitive skills in 42 adults (33 males and 9 females) with FXS as a portion of the baseline evaluation prior to randomization in a clinical trial of ampakine CX516. The battery of standardized tests addressed receptive vocabulary with the Peabody Picture Vocabulary Test, Third Edition (PPVT-III), receptive and expressive language (termed henceforth as global language) via the Preschool Language Scale, Fourth Edition or the Clinical Evaluation of Language Fundamentals, Third Edition, and non-verbal cognition via the Stanford-Binet Intelligence Scales, Fourth Edition (SB-IV). RESULTS: Results showed (1) significantly higher receptive vocabulary than global language, (2) significantly better receptive vocabulary than non-verbal cognition, (3) equivalent non-verbal cognition and global language, and (4) severity of autism symptomatology was not correlated to receptive vocabulary or global language once non-verbal cognition was removed as factor. The scores from the PPVT-III did not represent the global language skills in our sample of adults with FXS. CONCLUSIONS: Findings from this investigation strongly suggest that the PPVT-III should not be used as a screening tool for language levels or cognitive function in clinical studies since the scores from the PPVT-III were not representative of global language or non-verbal cognitive skills in adults with intellectual disabilities. This finding is critical in order to understand how to evaluate, as well as to treat, language in individuals with FXS. Development of efficient and appropriate tools to measure language, cognition, and behavior in individuals with FXS is essential. En ligne : https://dx.doi.org/10.1186/s11689-019-9285-x Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=409
in Journal of Neurodevelopmental Disorders > 11-1 (December 2019) . - 25 p.[article] Vocabulary comprehension in adults with fragile X syndrome (FXS) [Texte imprimé et/ou numérique] / A. HOFFMANN, Auteur ; S. E. KRAUSE, Auteur ; J. WUU, Auteur ; S. LEURGANS, Auteur ; S. J. GUTER, Auteur ; S. S. BLOCK, Auteur ; J. SALT, Auteur ; E. COOK, Auteur ; D. M. MAINO, Auteur ; Elizabeth BERRY-KRAVIS, Auteur . - 25 p.
Langues : Anglais (eng)
in Journal of Neurodevelopmental Disorders > 11-1 (December 2019) . - 25 p.
Mots-clés : Adults Cognition Comprehension Fragile X syndrome Language Vocabulary Index. décimale : PER Périodiques Résumé : BACKGROUND: Receptive and expressive vocabulary in adult and adolescent males with fragile X syndrome (FXS) have been shown as significantly lower than their chronological age; however, receptive vocabulary has been considered a strength relative to mental age. This has not been formally examined, however, and data are needed to compare receptive vocabulary with other language skills and with mental age in individuals with FXS. This is especially important as vocabulary measures are sometimes used as a proxy to estimate language ability. METHODS: This preliminary study examined receptive vocabulary, global language, and cognitive skills in 42 adults (33 males and 9 females) with FXS as a portion of the baseline evaluation prior to randomization in a clinical trial of ampakine CX516. The battery of standardized tests addressed receptive vocabulary with the Peabody Picture Vocabulary Test, Third Edition (PPVT-III), receptive and expressive language (termed henceforth as global language) via the Preschool Language Scale, Fourth Edition or the Clinical Evaluation of Language Fundamentals, Third Edition, and non-verbal cognition via the Stanford-Binet Intelligence Scales, Fourth Edition (SB-IV). RESULTS: Results showed (1) significantly higher receptive vocabulary than global language, (2) significantly better receptive vocabulary than non-verbal cognition, (3) equivalent non-verbal cognition and global language, and (4) severity of autism symptomatology was not correlated to receptive vocabulary or global language once non-verbal cognition was removed as factor. The scores from the PPVT-III did not represent the global language skills in our sample of adults with FXS. CONCLUSIONS: Findings from this investigation strongly suggest that the PPVT-III should not be used as a screening tool for language levels or cognitive function in clinical studies since the scores from the PPVT-III were not representative of global language or non-verbal cognitive skills in adults with intellectual disabilities. This finding is critical in order to understand how to evaluate, as well as to treat, language in individuals with FXS. Development of efficient and appropriate tools to measure language, cognition, and behavior in individuals with FXS is essential. En ligne : https://dx.doi.org/10.1186/s11689-019-9285-x Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=409