18 recherche sur le mot-clé 'Outcome measures'

Outcome measures in Angelman syndrome / Doesjka A. HAGENAAR in Journal of Neurodevelopmental Disorders, 16 (2024)  

Public ISBD [article]  inJournal of Neurodevelopmental Disorders > 16 (2024) Titre : Outcome measures in Angelman syndrome Type de document : texte imprimé Auteurs : Doesjka A. HAGENAAR, Auteur ; Karen G.C.B. BINDELS-DE HEUS, Auteur ; Maud M. VAN GILS, Auteur ; Louise VAN DEN BERG, Auteur ; Leontine W. TEN HOOPEN, Auteur ; Philine AFFOURTIT, Auteur ; Johan J.M. PEL, Auteur ; Koen F.M. JOOSTEN, Auteur ; Manon H.J. HILLEGERS, Auteur ; Henriette A. MOLL, Auteur ; Marie-Claire Y. DE WIT, Auteur ; Gwen C. DIELEMAN, Auteur ; Sabine E. MOUS, Auteur Langues : Anglais (eng) Mots-clés : Child  Humans  Angelman Syndrome/complications/diagnosis  Reproducibility of Results  Body Composition  Plethysmography/methods  Electric Impedance  Angelman syndrome  Bod pod  Bio-impedance analysis  Eye-tracking  Functional near-Infrared Spectroscopy  Indirect calorimetry  Outcome measures  hospital received funding for this study. The hospital also received compensation  for advice to Roche and Jazz Pharmaceuticals. The department of Child- and  Adolescent Psychiatry/Psychology (Erasmus MC) is the Dutch distributer of the  Achenbach System of Empirically Based Assessment (ASEBA) measurement instruments,  which include the Child Behaviour Checklist (CBCL). The department receives  financial compensation for selling the measurement instruments. All other authors  have no conflict of interest to declare. Index. décimale : PER Périodiques Résumé : BACKGROUND: Angelman syndrome (AS) is a rare neurodevelopmental disorder characterized by severe intellectual disability, little to no expressive speech, visual and motor problems, emotional/behavioral challenges, and a tendency towards hyperphagia and weight gain. The characteristics of AS make it difficult to measure these children's functioning with standard clinical tests. Feasible outcome measures are needed to measure current functioning and change over time, in clinical practice and clinical trials. AIM: Our first aim is to assess the feasibility of several functional tests. We target domains of neurocognitive functioning and physical growth using the following measurement methods: eye-tracking, functional Near-Infrared Spectroscopy (fNIRS), indirect calorimetry, bio-impedance analysis (BIA), and BOD POD (air-displacement plethysmography). Our second aim is to explore the results of the above measures, in order to better understand the AS phenotype. METHODS: The study sample consisted of 28 children with AS aged 2-18 years. We defined an outcome measure as feasible when (1) at least 70% of participants successfully finished the measurement and (2) at least 60% of those participants had acceptable data quality. Adaptations to the test procedure and reasons for early termination were noted. Parents rated acceptability and importance and were invited to make recommendations to increase feasibility. The results of the measures were explored. RESULTS: Outcome measures obtained with eye-tracking and BOD POD met the definition of feasibility, while fNIRS, indirect calorimetry, and BIA did not. The most important reasons for early termination of measurements were showing signs of protest, inability to sit still and poor/no calibration (eye-tracking specific). Post-calibration was often applied to obtain valid eye-tracking results. Parents rated the BOD POD als most acceptable and fNIRS as least acceptable for their child. All outcome measures were rated to be important. Exploratory results indicated longer reaction times to high salient visual stimuli (eye-tracking) as well as high body fat percentage (BOD POD). CONCLUSIONS: Eye-tracking and BOD POD are feasible measurement methods for children with AS. Eye-tracking was successfully used to assess visual orienting functions in the current study and (with some practical adaptations) can potentially be used to assess other outcomes as well. BOD POD was successfully used to examine body composition. TRIAL REGISTRATION: Registered d.d. 23-04-2020 under number 'NL8550' in the Dutch Trial Register: https://onderzoekmetmensen.nl/en/trial/23075. En ligne : https://dx.doi.org/10.1186/s11689-024-09516-1 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=575 [article] Outcome measures in Angelman syndrome [texte imprimé] / Doesjka A. HAGENAAR, Auteur ; Karen G.C.B. BINDELS-DE HEUS, Auteur ; Maud M. VAN GILS, Auteur ; Louise VAN DEN BERG, Auteur ; Leontine W. TEN HOOPEN, Auteur ; Philine AFFOURTIT, Auteur ; Johan J.M. PEL, Auteur ; Koen F.M. JOOSTEN, Auteur ; Manon H.J. HILLEGERS, Auteur ; Henriette A. MOLL, Auteur ; Marie-Claire Y. DE WIT, Auteur ; Gwen C. DIELEMAN, Auteur ; Sabine E. MOUS, Auteur. Langues : Anglais (eng) in Journal of Neurodevelopmental Disorders > 16 (2024) Mots-clés : Child  Humans  Angelman Syndrome/complications/diagnosis  Reproducibility of Results  Body Composition  Plethysmography/methods  Electric Impedance  Angelman syndrome  Bod pod  Bio-impedance analysis  Eye-tracking  Functional near-Infrared Spectroscopy  Indirect calorimetry  Outcome measures  hospital received funding for this study. The hospital also received compensation  for advice to Roche and Jazz Pharmaceuticals. The department of Child- and  Adolescent Psychiatry/Psychology (Erasmus MC) is the Dutch distributer of the  Achenbach System of Empirically Based Assessment (ASEBA) measurement instruments,  which include the Child Behaviour Checklist (CBCL). The department receives  financial compensation for selling the measurement instruments. All other authors  have no conflict of interest to declare. Index. décimale : PER Périodiques Résumé : BACKGROUND: Angelman syndrome (AS) is a rare neurodevelopmental disorder characterized by severe intellectual disability, little to no expressive speech, visual and motor problems, emotional/behavioral challenges, and a tendency towards hyperphagia and weight gain. The characteristics of AS make it difficult to measure these children's functioning with standard clinical tests. Feasible outcome measures are needed to measure current functioning and change over time, in clinical practice and clinical trials. AIM: Our first aim is to assess the feasibility of several functional tests. We target domains of neurocognitive functioning and physical growth using the following measurement methods: eye-tracking, functional Near-Infrared Spectroscopy (fNIRS), indirect calorimetry, bio-impedance analysis (BIA), and BOD POD (air-displacement plethysmography). Our second aim is to explore the results of the above measures, in order to better understand the AS phenotype. METHODS: The study sample consisted of 28 children with AS aged 2-18 years. We defined an outcome measure as feasible when (1) at least 70% of participants successfully finished the measurement and (2) at least 60% of those participants had acceptable data quality. Adaptations to the test procedure and reasons for early termination were noted. Parents rated acceptability and importance and were invited to make recommendations to increase feasibility. The results of the measures were explored. RESULTS: Outcome measures obtained with eye-tracking and BOD POD met the definition of feasibility, while fNIRS, indirect calorimetry, and BIA did not. The most important reasons for early termination of measurements were showing signs of protest, inability to sit still and poor/no calibration (eye-tracking specific). Post-calibration was often applied to obtain valid eye-tracking results. Parents rated the BOD POD als most acceptable and fNIRS as least acceptable for their child. All outcome measures were rated to be important. Exploratory results indicated longer reaction times to high salient visual stimuli (eye-tracking) as well as high body fat percentage (BOD POD). CONCLUSIONS: Eye-tracking and BOD POD are feasible measurement methods for children with AS. Eye-tracking was successfully used to assess visual orienting functions in the current study and (with some practical adaptations) can potentially be used to assess other outcomes as well. BOD POD was successfully used to examine body composition. TRIAL REGISTRATION: Registered d.d. 23-04-2020 under number 'NL8550' in the Dutch Trial Register: https://onderzoekmetmensen.nl/en/trial/23075. En ligne : https://dx.doi.org/10.1186/s11689-024-09516-1 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=575

Spoken language outcome measures for treatment studies in Down syndrome: feasibility, practice effects, test-retest reliability, and construct validity of variables generated from expressive language sampling / Angela John THURMAN in Journal of Neurodevelopmental Disorders, 13 (2021)  

Public ISBD [article]  inJournal of Neurodevelopmental Disorders > 13 (2021) Titre : Spoken language outcome measures for treatment studies in Down syndrome: feasibility, practice effects, test-retest reliability, and construct validity of variables generated from expressive language sampling Type de document : texte imprimé Auteurs : Angela John THURMAN, Auteur ; Jamie O. EDGIN, Auteur ; Stephanie L SHERMAN, Auteur ; Audra STERLING, Auteur ; Andrea MCDUFFIE, Auteur ; Elizabeth BERRY-KRAVIS, Auteur ; Debra HAMILTON, Auteur ; Leonard ABBEDUTO, Auteur Langues : Anglais (eng) Mots-clés : Adolescent  Adult  Child  Child, Preschool  Down Syndrome  Feasibility Studies  Humans  Language  Outcome Assessment, Health Care  Reproducibility of Results  Young Adult  Clinical trials  Down syndrome  Expressive language  Expressive language sampling  Outcome measures  Psychometrics  Treatment  Pharmaceuticals Ltd., Fulcrum Therapeutics, and Lumind to consult on and  implement outcome measures in clinical trials for FXS and DS. EBK has received  funding from Seaside Therapeutics, Novartis, Roche, Alcobra, Neuren, Cydan,  Fulcrum, GW, Neurotrope, Marinus, Zynerba, BioMarin, Lumos, Ovid, AMO, Yamo,  Ionis, GeneTx, Acadia, Neurogene, Ultragenyx, and Vtesse/Sucampo/Mallinkcrodt  Pharmaceuticals to consult on trial design or development strategies and/or  conduct clinical trials in FXS or other genetic neurodevelopmental or  neurodegenerative disorders, and from Asuragen Inc. to develop testing standards  for FMR1 testing. AJT has received funding from Fulcrum Therapeutics to develop  outcome measures for FXS. The other authors declare that they have no competing  interests. Index. décimale : PER Périodiques Résumé : BACKGROUND: The purpose of this study was to evaluate expressive language sampling (ELS) as a procedure for generating spoken language outcome measures for treatment research in Down syndrome (DS). We addressed (a) feasibility, (b) practice effects across two short-term administrations, (c) test-retest reliability across two short-term administrations, (d) convergent and discriminant construct validity, and (e) considered comparisons across the conversation and narration contexts. METHOD: Participants were 107 individuals with DS between 6 and 23 years of age who presented with intellectual disability (IQ < 70). The utility of ELS procedures designed to collect samples of spoken language in conversation and narration were evaluated separately. Variables of talkativeness, vocabulary, syntax, utterance planning, and articulation quality, derived from transcripts segmented into C-units (i.e., an independent clause and its modifiers), were considered. A 4-week interval was used to assess practice effects and test-retest reliability. Standardized direct assessments and informant report measures were collected to evaluate construct validity of the ELS variables. RESULTS: Low rates of noncompliance were observed; youth who were under 12 years of age, had phrase-level speech or less, and had a 4-year-old developmental level or less were at particular risk for experiencing difficulty completing the ELS procedures. Minimal practice effects and strong test-retest reliability across the 4-week test-retest interval was observed. The vocabulary, syntax, and speech intelligibility variables demonstrated strong convergent and discriminant validity. Although significant correlations were found between the variables derived from both the conversation and narration contexts, some differences were noted. CONCLUSION: The ELS procedures considered were feasible and yielded variables with adequate psychometric properties for most individuals with DS between 6 and 23 years old. That said, studies of outcome measures appropriate for individuals with DS with more limited spoken language skills are needed. Context differences were observed in ELS variables suggest that comprehensive evaluation of expressive language is likely best obtained when utilizing both contexts. En ligne : https://dx.doi.org/10.1186/s11689-021-09361-6 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=573 [article] Spoken language outcome measures for treatment studies in Down syndrome: feasibility, practice effects, test-retest reliability, and construct validity of variables generated from expressive language sampling [texte imprimé] / Angela John THURMAN, Auteur ; Jamie O. EDGIN, Auteur ; Stephanie L SHERMAN, Auteur ; Audra STERLING, Auteur ; Andrea MCDUFFIE, Auteur ; Elizabeth BERRY-KRAVIS, Auteur ; Debra HAMILTON, Auteur ; Leonard ABBEDUTO, Auteur. Langues : Anglais (eng) in Journal of Neurodevelopmental Disorders > 13 (2021) Mots-clés : Adolescent  Adult  Child  Child, Preschool  Down Syndrome  Feasibility Studies  Humans  Language  Outcome Assessment, Health Care  Reproducibility of Results  Young Adult  Clinical trials  Down syndrome  Expressive language  Expressive language sampling  Outcome measures  Psychometrics  Treatment  Pharmaceuticals Ltd., Fulcrum Therapeutics, and Lumind to consult on and  implement outcome measures in clinical trials for FXS and DS. EBK has received  funding from Seaside Therapeutics, Novartis, Roche, Alcobra, Neuren, Cydan,  Fulcrum, GW, Neurotrope, Marinus, Zynerba, BioMarin, Lumos, Ovid, AMO, Yamo,  Ionis, GeneTx, Acadia, Neurogene, Ultragenyx, and Vtesse/Sucampo/Mallinkcrodt  Pharmaceuticals to consult on trial design or development strategies and/or  conduct clinical trials in FXS or other genetic neurodevelopmental or  neurodegenerative disorders, and from Asuragen Inc. to develop testing standards  for FMR1 testing. AJT has received funding from Fulcrum Therapeutics to develop  outcome measures for FXS. The other authors declare that they have no competing  interests. Index. décimale : PER Périodiques Résumé : BACKGROUND: The purpose of this study was to evaluate expressive language sampling (ELS) as a procedure for generating spoken language outcome measures for treatment research in Down syndrome (DS). We addressed (a) feasibility, (b) practice effects across two short-term administrations, (c) test-retest reliability across two short-term administrations, (d) convergent and discriminant construct validity, and (e) considered comparisons across the conversation and narration contexts. METHOD: Participants were 107 individuals with DS between 6 and 23 years of age who presented with intellectual disability (IQ < 70). The utility of ELS procedures designed to collect samples of spoken language in conversation and narration were evaluated separately. Variables of talkativeness, vocabulary, syntax, utterance planning, and articulation quality, derived from transcripts segmented into C-units (i.e., an independent clause and its modifiers), were considered. A 4-week interval was used to assess practice effects and test-retest reliability. Standardized direct assessments and informant report measures were collected to evaluate construct validity of the ELS variables. RESULTS: Low rates of noncompliance were observed; youth who were under 12 years of age, had phrase-level speech or less, and had a 4-year-old developmental level or less were at particular risk for experiencing difficulty completing the ELS procedures. Minimal practice effects and strong test-retest reliability across the 4-week test-retest interval was observed. The vocabulary, syntax, and speech intelligibility variables demonstrated strong convergent and discriminant validity. Although significant correlations were found between the variables derived from both the conversation and narration contexts, some differences were noted. CONCLUSION: The ELS procedures considered were feasible and yielded variables with adequate psychometric properties for most individuals with DS between 6 and 23 years old. That said, studies of outcome measures appropriate for individuals with DS with more limited spoken language skills are needed. Context differences were observed in ELS variables suggest that comprehensive evaluation of expressive language is likely best obtained when utilizing both contexts. En ligne : https://dx.doi.org/10.1186/s11689-021-09361-6 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=573

Brief Report: Do Service Dog Providers Placing Dogs with Children with Developmental Disabilities Use Outcome Measures and, If So, What Are they? / Felicity Ruth BUTTERLY in Journal of Autism and Developmental Disorders, 43-11 (November 2013)  

Public ISBD [article]  inJournal of Autism and Developmental Disorders > 43-11 (November 2013) . - p.2720-2725 Titre : Brief Report: Do Service Dog Providers Placing Dogs with Children with Developmental Disabilities Use Outcome Measures and, If So, What Are they? Type de document : texte imprimé Auteurs : Felicity Ruth BUTTERLY, Auteur ; Carol PERCY, Auteur ; Gillian WARD, Auteur Article en page(s) : p.2720-2725 Langues : Anglais (eng) Mots-clés : Assistance Dogs  Service providers  Outcome measures  Developmental disability Index. décimale : PER Périodiques Résumé : The aim of this study was to identify the outcomes expected and assessed by those providing service dogs to children with developmental disabilities. Seventeen registered service dog providers were invited to complete a mixed methods online survey. Five providers, who prepared dogs to work with a wide range of conditions and behaviours, mainly Asperger’s syndrome, autism and communication disorders, completed the survey. All five participants reported that they expected to see positive changes as a consequence of the service dog placement, in both the recipient child and their family, including improvements in attention span and language skills, as well as increased familial cohesion. Survey responses indicated that not all desired outcomes were routinely assessed. The range of assessments used were interviews, intake conversations, pre-placement questionnaires, child social dairies filled in by parents, follow up surveys after placement, and child observation by parents. No specifically named valid and reliable clinical or research measures were referred to, showing an emphasis on assessments from parents and service dog providers. It is not clear whether pre-intervention assessments are repeated systematically at follow-up, which could show robust intervention effects. There is scope for professionals in developmental disability to work with service dog providers to improve the evidence base in this field. En ligne : http://dx.doi.org/10.1007/s10803-013-1803-1 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=217 [article] Brief Report: Do Service Dog Providers Placing Dogs with Children with Developmental Disabilities Use Outcome Measures and, If So, What Are they? [texte imprimé] / Felicity Ruth BUTTERLY, Auteur ; Carol PERCY, Auteur ; Gillian WARD, Auteur . - p.2720-2725. Langues : Anglais (eng) in Journal of Autism and Developmental Disorders > 43-11 (November 2013) . - p.2720-2725 Mots-clés : Assistance Dogs  Service providers  Outcome measures  Developmental disability Index. décimale : PER Périodiques Résumé : The aim of this study was to identify the outcomes expected and assessed by those providing service dogs to children with developmental disabilities. Seventeen registered service dog providers were invited to complete a mixed methods online survey. Five providers, who prepared dogs to work with a wide range of conditions and behaviours, mainly Asperger’s syndrome, autism and communication disorders, completed the survey. All five participants reported that they expected to see positive changes as a consequence of the service dog placement, in both the recipient child and their family, including improvements in attention span and language skills, as well as increased familial cohesion. Survey responses indicated that not all desired outcomes were routinely assessed. The range of assessments used were interviews, intake conversations, pre-placement questionnaires, child social dairies filled in by parents, follow up surveys after placement, and child observation by parents. No specifically named valid and reliable clinical or research measures were referred to, showing an emphasis on assessments from parents and service dog providers. It is not clear whether pre-intervention assessments are repeated systematically at follow-up, which could show robust intervention effects. There is scope for professionals in developmental disability to work with service dog providers to improve the evidence base in this field. En ligne : http://dx.doi.org/10.1007/s10803-013-1803-1 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=217

Expressive language sampling as a source of outcome measures for treatment studies in fragile X syndrome: feasibility, practice effects, test-retest reliability, and construct validity / Leonard ABBEDUTO in Journal of Neurodevelopmental Disorders, 12 (2020)  

Public ISBD [article]  inJournal of Neurodevelopmental Disorders > 12 (2020) Titre : Expressive language sampling as a source of outcome measures for treatment studies in fragile X syndrome: feasibility, practice effects, test-retest reliability, and construct validity Type de document : texte imprimé Auteurs : Leonard ABBEDUTO, Auteur ; Elizabeth BERRY-KRAVIS, Auteur ; Audra STERLING, Auteur ; Stephanie SHERMAN, Auteur ; Jamie O. EDGIN, Auteur ; Andrea MCDUFFIE, Auteur ; Anne HOFFMANN, Auteur ; Debra HAMILTON, Auteur ; Michael NELSON, Auteur ; Jeannie ASCHKENASY, Auteur ; Angela John THURMAN, Auteur Langues : Anglais (eng) Mots-clés : Adolescent  Autistic Disorder/diagnosis  Child  Feasibility Studies  Female  Fragile X Syndrome/therapy  Humans  Language Tests/statistics & numerical data  Male  Outcome Assessment, Health Care  Psychometrics  Reproducibility of Results  Young Adult  Clinical trials  Expressive language  Fragile X syndrome  Outcome measures  treatment  Pharmaceuticals Ltd., Fulcrum Therapeutics, and Lumind to consult on and  implement outcome measures in clinical trials for FXS and Down syndrome. EBK has  received funding from Seaside Therapeutics, Novartis, Roche, Alcobra, Neuren,  Cydan, Fulcrum, GW, Neurotrope, Marinus, Zynerba, BioMarin, Lumos, Ovid, AMO,  Yamo, Ionis, GeneTx, Acadia, Neurogene, Ultragenyx, and  Vtesse/Sucampo/Mallinkcrodt Pharmaceuticals to consult on trial design or  development strategies and/or conduct clinical trials in FXS or other genetic  Neurodevelopmental or neurodegenerative disorders, and from Asuragen Inc. to  develop testing standards for FMR1 testing. AJT has received funding from Fulcrum  Therapeutics to develop outcome measures for FXS. The other authors declare that  they have no competing interests. Index. décimale : PER Périodiques Résumé : BACKGROUND: The evaluation of treatment efficacy for individuals with fragile X syndrome (FXS) or intellectual disability (ID) more generally has been hampered by the lack of adequate outcome measures. We evaluated expressive language sampling (ELS) as a procedure for generating outcome measures for treatment research in FXS. We addressed: (a) feasibility, (b) practice effects over two administrations, (c) test-retest reliability over the repeated administrations, and (d) construct validity. We addressed these issues for the full sample as well as for subgroups defined by age, IQ, and ASD status. METHODS: Participants were 106 individuals with FXS between ages 6 and 23 years who had IQs within the range of intellectual disability (IQ < 70). ELS procedures for collecting samples in conversation and narration were followed and analyzed separately. Five measures were derived from transcripts segmented into C-units (i.e., an independent clause and its modifiers): number of C-units per minute (talkativeness), number of different word roots (vocabulary), C-unit length in morphemes (syntax), percentage of C-units containing dysfluency (utterance planning), and percentage of C-units that were fully or partly unintelligible (articulatory quality). ELS procedures were administered twice at 4-week intervals for each participant. Standardized tests and informant reports were administered and provided measures for evaluating construct validity of ELS measures. RESULTS: We found low rates of noncompliance, suggesting the task can be completed meaningfully by most individuals with FXS, although noncompliance was higher for younger, lower IQ, and more autistic participants. Minimal practice effects and strong test-retest reliability over the 4-week interval were observed for the full sample and across the range of ages, IQs, and autism symptom severity. Evidence of convergent construct validity was observed for the measures of vocabulary, syntax, and unintelligibility for the full sample and across the range of IQ and autism symptom severity, but not for participants under age 12. Conversation and narration yielded largely similar results in all analyses. CONCLUSIONS: The findings suggest that the ELS procedures are feasible and yield measures with adequate psychometric properties for a majority of 6 to 23 years with FXS who have ID. The procedures work equally well regardless of level of ID or degree of ASD severity. The procedures, however, are more challenging and have somewhat less adequate psychometric properties for individuals with FXS under age 12. En ligne : https://dx.doi.org/10.1186/s11689-020-09313-6 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=573 [article] Expressive language sampling as a source of outcome measures for treatment studies in fragile X syndrome: feasibility, practice effects, test-retest reliability, and construct validity [texte imprimé] / Leonard ABBEDUTO, Auteur ; Elizabeth BERRY-KRAVIS, Auteur ; Audra STERLING, Auteur ; Stephanie SHERMAN, Auteur ; Jamie O. EDGIN, Auteur ; Andrea MCDUFFIE, Auteur ; Anne HOFFMANN, Auteur ; Debra HAMILTON, Auteur ; Michael NELSON, Auteur ; Jeannie ASCHKENASY, Auteur ; Angela John THURMAN, Auteur. Langues : Anglais (eng) in Journal of Neurodevelopmental Disorders > 12 (2020) Mots-clés : Adolescent  Autistic Disorder/diagnosis  Child  Feasibility Studies  Female  Fragile X Syndrome/therapy  Humans  Language Tests/statistics & numerical data  Male  Outcome Assessment, Health Care  Psychometrics  Reproducibility of Results  Young Adult  Clinical trials  Expressive language  Fragile X syndrome  Outcome measures  treatment  Pharmaceuticals Ltd., Fulcrum Therapeutics, and Lumind to consult on and  implement outcome measures in clinical trials for FXS and Down syndrome. EBK has  received funding from Seaside Therapeutics, Novartis, Roche, Alcobra, Neuren,  Cydan, Fulcrum, GW, Neurotrope, Marinus, Zynerba, BioMarin, Lumos, Ovid, AMO,  Yamo, Ionis, GeneTx, Acadia, Neurogene, Ultragenyx, and  Vtesse/Sucampo/Mallinkcrodt Pharmaceuticals to consult on trial design or  development strategies and/or conduct clinical trials in FXS or other genetic  Neurodevelopmental or neurodegenerative disorders, and from Asuragen Inc. to  develop testing standards for FMR1 testing. AJT has received funding from Fulcrum  Therapeutics to develop outcome measures for FXS. The other authors declare that  they have no competing interests. Index. décimale : PER Périodiques Résumé : BACKGROUND: The evaluation of treatment efficacy for individuals with fragile X syndrome (FXS) or intellectual disability (ID) more generally has been hampered by the lack of adequate outcome measures. We evaluated expressive language sampling (ELS) as a procedure for generating outcome measures for treatment research in FXS. We addressed: (a) feasibility, (b) practice effects over two administrations, (c) test-retest reliability over the repeated administrations, and (d) construct validity. We addressed these issues for the full sample as well as for subgroups defined by age, IQ, and ASD status. METHODS: Participants were 106 individuals with FXS between ages 6 and 23 years who had IQs within the range of intellectual disability (IQ < 70). ELS procedures for collecting samples in conversation and narration were followed and analyzed separately. Five measures were derived from transcripts segmented into C-units (i.e., an independent clause and its modifiers): number of C-units per minute (talkativeness), number of different word roots (vocabulary), C-unit length in morphemes (syntax), percentage of C-units containing dysfluency (utterance planning), and percentage of C-units that were fully or partly unintelligible (articulatory quality). ELS procedures were administered twice at 4-week intervals for each participant. Standardized tests and informant reports were administered and provided measures for evaluating construct validity of ELS measures. RESULTS: We found low rates of noncompliance, suggesting the task can be completed meaningfully by most individuals with FXS, although noncompliance was higher for younger, lower IQ, and more autistic participants. Minimal practice effects and strong test-retest reliability over the 4-week interval were observed for the full sample and across the range of ages, IQs, and autism symptom severity. Evidence of convergent construct validity was observed for the measures of vocabulary, syntax, and unintelligibility for the full sample and across the range of IQ and autism symptom severity, but not for participants under age 12. Conversation and narration yielded largely similar results in all analyses. CONCLUSIONS: The findings suggest that the ELS procedures are feasible and yield measures with adequate psychometric properties for a majority of 6 to 23 years with FXS who have ID. The procedures work equally well regardless of level of ID or degree of ASD severity. The procedures, however, are more challenging and have somewhat less adequate psychometric properties for individuals with FXS under age 12. En ligne : https://dx.doi.org/10.1186/s11689-020-09313-6 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=573

Relations between specific and global outcome measures in a social-communication intervention for children with autism spectrum disorder / Anders NORDAHL-HANSEN in Research in Autism Spectrum Disorders, 29-30 (September–October 2016)  

Public ISBD [article]  inResearch in Autism Spectrum Disorders > 29-30 (September–October 2016) . - p.19-29 Titre : Relations between specific and global outcome measures in a social-communication intervention for children with autism spectrum disorder Type de document : texte imprimé Auteurs : Anders NORDAHL-HANSEN, Auteur ; Sue FLETCHER-WATSON, Auteur ; Helen MCCONACHIE, Auteur ; Anett KAALE, Auteur Article en page(s) : p.19-29 Langues : Anglais (eng) Mots-clés : Autism spectrum disorders  Social communication  Treatment  Intervention  Outcome measures  Brief observation of social communication change (BOSCC)  Children  Joint engagement Index. décimale : PER Périodiques Résumé : Abstract Assessment of relevant outcomes is a key challenge in evaluating effects of social-communication interventions. However, few studies have investigated in what ways specific and more global measures may influence reported results of social-communication interventions for children with autism spectrum disorder (ASD). In this study both a specific and a global, more global autism symptom measure were used to assess effects of a brief social-communication intervention. Fifty-nine children (2–4 years) diagnosed with autistic disorder were assessed with the Joint Engagement (JE) states coding procedure and a preliminary version of the Brief Observation of Social Communication Change (BOSCC). A statistically significant difference was found between intervention and control groups from baseline to intervention endpoint on JE but not on BOSCC. Degree of change on the measures was moderately related, and both were independent of language level and non-verbal mental age. This study adds to the knowledge of what may be expected of different outcome measures and provides suggestions to how measures may be deployed to investigate underlying mechanisms and developmental pathways. En ligne : http://dx.doi.org/10.1016/j.rasd.2016.05.005 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=292 [article] Relations between specific and global outcome measures in a social-communication intervention for children with autism spectrum disorder [texte imprimé] / Anders NORDAHL-HANSEN, Auteur ; Sue FLETCHER-WATSON, Auteur ; Helen MCCONACHIE, Auteur ; Anett KAALE, Auteur . - p.19-29. Langues : Anglais (eng) in Research in Autism Spectrum Disorders > 29-30 (September–October 2016) . - p.19-29 Mots-clés : Autism spectrum disorders  Social communication  Treatment  Intervention  Outcome measures  Brief observation of social communication change (BOSCC)  Children  Joint engagement Index. décimale : PER Périodiques Résumé : Abstract Assessment of relevant outcomes is a key challenge in evaluating effects of social-communication interventions. However, few studies have investigated in what ways specific and more global measures may influence reported results of social-communication interventions for children with autism spectrum disorder (ASD). In this study both a specific and a global, more global autism symptom measure were used to assess effects of a brief social-communication intervention. Fifty-nine children (2–4 years) diagnosed with autistic disorder were assessed with the Joint Engagement (JE) states coding procedure and a preliminary version of the Brief Observation of Social Communication Change (BOSCC). A statistically significant difference was found between intervention and control groups from baseline to intervention endpoint on JE but not on BOSCC. Degree of change on the measures was moderately related, and both were independent of language level and non-verbal mental age. This study adds to the knowledge of what may be expected of different outcome measures and provides suggestions to how measures may be deployed to investigate underlying mechanisms and developmental pathways. En ligne : http://dx.doi.org/10.1016/j.rasd.2016.05.005 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=292

Commentary: Measuring Language Change Through Natural Language Samples / Mihaela D. BAROKOVA in Journal of Autism and Developmental Disorders, 50-7 (July 2020)  

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Measuring social communication behaviors as a treatment endpoint in individuals with autism spectrum disorder / Evdokia ANAGNOSTOU in Autism, 19-5 (July 2015)  

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Occupational therapy in Autism Spectrum Disorder: A scoping umbrella review of randomized controlled trials / Marta MARCILLA-JORDÁ in Research in Autism, 132 (April 2026)  

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Research Review: Measuring life impact of youth mental health difficulties: scoping umbrella review of 80 instruments / Karolin R. KRAUSE in Journal of Child Psychology and Psychiatry, 67-8 (August 2026)  

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The NIH Toolbox Cognitive Battery for intellectual disabilities: three preliminary studies and future directions / David HESSL in Journal of Neurodevelopmental Disorders, 8-1 (December 2016)  

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