Controlled trial of lovastatin combined with an open-label treatment of a parent-implemented language intervention in youth with fragile X syndrome / Angela John THURMAN in Journal of Neurodevelopmental Disorders, 12 (2020)  

Public ISBD [article]  inJournal of Neurodevelopmental Disorders > 12 (2020) Titre : Controlled trial of lovastatin combined with an open-label treatment of a parent-implemented language intervention in youth with fragile X syndrome Type de document : texte imprimé Auteurs : Angela John THURMAN, Auteur ; Laura A. POTTER, Auteur ; Kyoungmi KIM, Auteur ; Flora TASSONE, Auteur ; Amy BANASIK, Auteur ; Sarah Nelson POTTER, Auteur ; Lauren BULLARD, Auteur ; Vivian NGUYEN, Auteur ; Andrea MCDUFFIE, Auteur ; Randi HAGERMAN, Auteur ; Leonard ABBEDUTO, Auteur Langues : Anglais (eng) Mots-clés : Adolescent  California  Child  Communication  Double-Blind Method  Female  Fragile X Syndrome/therapy  Humans  Language  Language Therapy/methods  Lovastatin/therapeutic use  Male  Mothers/education  Outcome Assessment, Health Care  Telecommunications  Distance teleconferencing  Expressive language sampling  Fragile X syndrome  Lovastatin  Narrative storytelling  Pili  Parent-implemented language intervention  outcome measures from Fulcrum Therapeutics and the Azrieli Foundation. FT has  received funding from the Azrieli Foundation, Zynerba, and Asuragen, Inc., for  studies in FXS. RH has received funding from Zynerba, Ovid, and the Azrieli  Foundation for treatment studies in children and adults with FXS. She has also  consulted with Zynerba and Fulcrum regarding treatment studies in FXS. LA has  received funding for the development and implementation of treatment outcome  measures from the F. Hoffmann-La Roche Ltd., Roche TCRC, Inc., Neuren  Pharmaceuticals Ltd., Fulcrum Therapeutics, Azrieli Foundation, and LuMind IDSC  Foundation. Index. décimale : PER Périodiques Résumé : BACKGROUND: The purpose of this study was to conduct a 20-week controlled trial of lovastatin (10 to 40 mg/day) in youth with fragile X syndrome (FXS) ages 10 to 17 years, combined with an open-label treatment of a parent-implemented language intervention (PILI), delivered via distance video teleconferencing to both treatment groups, lovastatin and placebo. METHOD: A randomized, double-blind trial was conducted at one site in the Sacramento, California, metropolitan area. Fourteen participants were assigned to the lovastatin group; two participants terminated early from the study. Sixteen participants were assigned to the placebo group. Lovastatin or placebo was administered orally in a capsule form, starting at 10 mg and increasing weekly or as tolerated by 10 mg increments, up to a maximum dose of 40 mg daily. A PILI was delivered to both groups for 12 weeks, with 4 activities per week, through video teleconferencing by an American Speech-Language Association-certified Speech-Language Pathologist, in collaboration with a Board-Certified Behavior Analyst. Parents were taught to use a set of language facilitation strategies while interacting with their children during a shared storytelling activity. The main outcome measures included absolute change from baseline to final visit in the means for youth total number of story-related utterances, youth number of different word roots, and parent total number of story-related utterances. RESULTS: Significant increases in all primary outcome measures were observed in both treatment groups. Significant improvements were also observed in parent reports of the severity of spoken language and social impairments in both treatment groups. In all cases, the amount of change observed did not differ across the two treatment groups. Although gains in parental use of the PILI-targeted intervention strategies were observed in both treatment groups, parental use of the PILI strategies was correlated with youth gains in the placebo group and not in the lovastatin group. CONCLUSION: Participants in both groups demonstrated significant changes in the primary outcome measures. The magnitude of change observed across the two groups was comparable, providing additional support for the efficacy of the use of PILI in youth with FXS. TRIAL REGISTRATION: US National Institutes of Health (ClinicalTrials.gov), NCT02642653. Registered 12/30/2015. En ligne : https://dx.doi.org/10.1186/s11689-020-09315-4 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=573 [article] Controlled trial of lovastatin combined with an open-label treatment of a parent-implemented language intervention in youth with fragile X syndrome [texte imprimé] / Angela John THURMAN, Auteur ; Laura A. POTTER, Auteur ; Kyoungmi KIM, Auteur ; Flora TASSONE, Auteur ; Amy BANASIK, Auteur ; Sarah Nelson POTTER, Auteur ; Lauren BULLARD, Auteur ; Vivian NGUYEN, Auteur ; Andrea MCDUFFIE, Auteur ; Randi HAGERMAN, Auteur ; Leonard ABBEDUTO, Auteur. Langues : Anglais (eng) in Journal of Neurodevelopmental Disorders > 12 (2020) Mots-clés : Adolescent  California  Child  Communication  Double-Blind Method  Female  Fragile X Syndrome/therapy  Humans  Language  Language Therapy/methods  Lovastatin/therapeutic use  Male  Mothers/education  Outcome Assessment, Health Care  Telecommunications  Distance teleconferencing  Expressive language sampling  Fragile X syndrome  Lovastatin  Narrative storytelling  Pili  Parent-implemented language intervention  outcome measures from Fulcrum Therapeutics and the Azrieli Foundation. FT has  received funding from the Azrieli Foundation, Zynerba, and Asuragen, Inc., for  studies in FXS. RH has received funding from Zynerba, Ovid, and the Azrieli  Foundation for treatment studies in children and adults with FXS. She has also  consulted with Zynerba and Fulcrum regarding treatment studies in FXS. LA has  received funding for the development and implementation of treatment outcome  measures from the F. Hoffmann-La Roche Ltd., Roche TCRC, Inc., Neuren  Pharmaceuticals Ltd., Fulcrum Therapeutics, Azrieli Foundation, and LuMind IDSC  Foundation. Index. décimale : PER Périodiques Résumé : BACKGROUND: The purpose of this study was to conduct a 20-week controlled trial of lovastatin (10 to 40 mg/day) in youth with fragile X syndrome (FXS) ages 10 to 17 years, combined with an open-label treatment of a parent-implemented language intervention (PILI), delivered via distance video teleconferencing to both treatment groups, lovastatin and placebo. METHOD: A randomized, double-blind trial was conducted at one site in the Sacramento, California, metropolitan area. Fourteen participants were assigned to the lovastatin group; two participants terminated early from the study. Sixteen participants were assigned to the placebo group. Lovastatin or placebo was administered orally in a capsule form, starting at 10 mg and increasing weekly or as tolerated by 10 mg increments, up to a maximum dose of 40 mg daily. A PILI was delivered to both groups for 12 weeks, with 4 activities per week, through video teleconferencing by an American Speech-Language Association-certified Speech-Language Pathologist, in collaboration with a Board-Certified Behavior Analyst. Parents were taught to use a set of language facilitation strategies while interacting with their children during a shared storytelling activity. The main outcome measures included absolute change from baseline to final visit in the means for youth total number of story-related utterances, youth number of different word roots, and parent total number of story-related utterances. RESULTS: Significant increases in all primary outcome measures were observed in both treatment groups. Significant improvements were also observed in parent reports of the severity of spoken language and social impairments in both treatment groups. In all cases, the amount of change observed did not differ across the two treatment groups. Although gains in parental use of the PILI-targeted intervention strategies were observed in both treatment groups, parental use of the PILI strategies was correlated with youth gains in the placebo group and not in the lovastatin group. CONCLUSION: Participants in both groups demonstrated significant changes in the primary outcome measures. The magnitude of change observed across the two groups was comparable, providing additional support for the efficacy of the use of PILI in youth with FXS. TRIAL REGISTRATION: US National Institutes of Health (ClinicalTrials.gov), NCT02642653. Registered 12/30/2015. En ligne : https://dx.doi.org/10.1186/s11689-020-09315-4 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=573

Patterns and predictors of adaptive skills in 2- to 7-year-old children with Down syndrome / Emily K. SCHWORER in Journal of Neurodevelopmental Disorders, 14 (2022)  

Public ISBD [article]  inJournal of Neurodevelopmental Disorders > 14 (2022) Titre : Patterns and predictors of adaptive skills in 2- to 7-year-old children with Down syndrome Type de document : texte imprimé Auteurs : Emily K. SCHWORER, Auteur ; Anna J. ESBENSEN, Auteur ; Vivian NGUYEN, Auteur ; Lauren BULLARD, Auteur ; Deborah J. FIDLER, Auteur ; Lisa A. DAUNHAUER, Auteur ; Carolyn B. MERVIS, Auteur ; Angela M. BECERRA, Auteur ; Leonard ABBEDUTO, Auteur ; Angela John THURMAN, Auteur Langues : Anglais (eng) Mots-clés : Adaptation, Psychological  Autism Spectrum Disorder/psychology  Child  Child, Preschool  Down Syndrome  Humans  Motor Skills  Socialization  ASD symptoms  Adaptive skills  Cognition  Down syndrome  Motor skills  Pharmaceuticals Ltd., Fulcrum Therapeutics, and Lumind to consult on and  implement outcome measures in clinical trials for FXS and DS. 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: There is substantial variability in adaptive skills among individuals with Down syndrome. Few studies, however, have focused on the early developmental period or on the potential sources of variability in adaptive skills. This study characterizes adaptive skills in young children with Down syndrome and investigates child characteristics associated with adaptive skills. METHODS: Participants were 44 children with Down syndrome ranging in age from 2.50 to 7.99 years (M = 4.66 years, SD = 1.46). The Vineland Adaptive Behavior Scales-3 (VABS-3) Comprehensive Interview Form was used to assess adaptive behavior in the three core domains: socialization, daily living, and communication skills. Caregivers also reported on motor skills and autism spectrum disorder symptoms. Child cognitive abilities were assessed. RESULTS: Analyses comparing mean standard score performance across the three VABS-3 core domains demonstrated significant differences between all pairs of domains, resulting in a group-level pattern of socialization > daily living > communication skills. At the individual level, 10 different patterns of relative strength and weakness were identified, with only 18% of participants evidencing significant differences between adaptive skill domain standard scores corresponding to the group-level pattern of significant differences. Child characteristics (cognitive abilities, motor skills, and autism spectrum disorder symptoms) were significantly associated with VABS-3 adaptive domain standard scores. CONCLUSION: These findings underscore the importance of individualizing intervention programs focused on improving the adaptive skills of young children with Down syndrome based on consideration of the child's relative adaptive strengths and weaknesses. En ligne : https://dx.doi.org/10.1186/s11689-022-09430-4 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=574 [article] Patterns and predictors of adaptive skills in 2- to 7-year-old children with Down syndrome [texte imprimé] / Emily K. SCHWORER, Auteur ; Anna J. ESBENSEN, Auteur ; Vivian NGUYEN, Auteur ; Lauren BULLARD, Auteur ; Deborah J. FIDLER, Auteur ; Lisa A. DAUNHAUER, Auteur ; Carolyn B. MERVIS, Auteur ; Angela M. BECERRA, Auteur ; Leonard ABBEDUTO, Auteur ; Angela John THURMAN, Auteur. Langues : Anglais (eng) in Journal of Neurodevelopmental Disorders > 14 (2022) Mots-clés : Adaptation, Psychological  Autism Spectrum Disorder/psychology  Child  Child, Preschool  Down Syndrome  Humans  Motor Skills  Socialization  ASD symptoms  Adaptive skills  Cognition  Down syndrome  Motor skills  Pharmaceuticals Ltd., Fulcrum Therapeutics, and Lumind to consult on and  implement outcome measures in clinical trials for FXS and DS. 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: There is substantial variability in adaptive skills among individuals with Down syndrome. Few studies, however, have focused on the early developmental period or on the potential sources of variability in adaptive skills. This study characterizes adaptive skills in young children with Down syndrome and investigates child characteristics associated with adaptive skills. METHODS: Participants were 44 children with Down syndrome ranging in age from 2.50 to 7.99 years (M = 4.66 years, SD = 1.46). The Vineland Adaptive Behavior Scales-3 (VABS-3) Comprehensive Interview Form was used to assess adaptive behavior in the three core domains: socialization, daily living, and communication skills. Caregivers also reported on motor skills and autism spectrum disorder symptoms. Child cognitive abilities were assessed. RESULTS: Analyses comparing mean standard score performance across the three VABS-3 core domains demonstrated significant differences between all pairs of domains, resulting in a group-level pattern of socialization > daily living > communication skills. At the individual level, 10 different patterns of relative strength and weakness were identified, with only 18% of participants evidencing significant differences between adaptive skill domain standard scores corresponding to the group-level pattern of significant differences. Child characteristics (cognitive abilities, motor skills, and autism spectrum disorder symptoms) were significantly associated with VABS-3 adaptive domain standard scores. CONCLUSION: These findings underscore the importance of individualizing intervention programs focused on improving the adaptive skills of young children with Down syndrome based on consideration of the child's relative adaptive strengths and weaknesses. En ligne : https://dx.doi.org/10.1186/s11689-022-09430-4 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=574

Using weighted communication scoring procedures in naturalistic play samples: Preliminary validation in preschool-aged boys with autism or fragile X syndrome / Angela J. THURMAN in Autism Research, 15-9 (September 2022)  

Public ISBD [article]  inAutism Research > 15-9 (September 2022) . - p.1755-1767 Titre : Using weighted communication scoring procedures in naturalistic play samples: Preliminary validation in preschool-aged boys with autism or fragile X syndrome Type de document : texte imprimé Auteurs : Angela J. THURMAN, Auteur ; Cesar HOYOS ALVAREZ, Auteur ; Vivian NGUYEN, Auteur Article en page(s) : p.1755-1767 Langues : Anglais (eng) Mots-clés : Autism Spectrum Disorder  Autistic Disorder  Child  Child, Preschool  Communication  Fragile X Syndrome  Humans  Male  Reproducibility of Results  autism  fragile X syndrome  naturalistic  outcome measure  weighting communication scores Index. décimale : PER Périodiques Résumé : In the present study, we provide a preliminary evaluation of the validity and reliability of using weighting procedures to measure communication in play samples for preschool-aged boys with autism or fragile X syndrome (FXS). Because weighting procedure communication scores (WPCSs) reflect growth in both communicative frequency and complexity, establishing the psychometrics of the component scores, in addition to the overall metric, affords investigators the opportunity to describe growth within and across skills. Results, for both groups, provide support regarding the psychometric appropriateness (i.e., convergent validity, divergent validity, and internal consistency) for all WPCSs. That said, a trend was observed for reliability scores to be slightly lower or more variable in boys with autism than in boys with FXS. Finally, although significant associations were observed in the associations between WPCSs across play contexts, contexts effects were observed for all three WPCSs. Together, results from this study provide promising preliminary data indicating the utility of using WPCSs in children with neurodevelopmental disabilities. Lay Summary: Language supports long-term positive outcomes; it is important to identify accurate and flexible ways of measuring language in children over time. We considered the effectiveness of using a procedure that considers changes in the number of communication acts and the types of acts produced during a play session by preschool-aged boys with autism or fragile X syndrome. These procedures were found to be valid and reliable. En ligne : http://dx.doi.org/10.1002/aur.2724 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=483 [article] Using weighted communication scoring procedures in naturalistic play samples: Preliminary validation in preschool-aged boys with autism or fragile X syndrome [texte imprimé] / Angela J. THURMAN, Auteur ; Cesar HOYOS ALVAREZ, Auteur ; Vivian NGUYEN, Auteur . - p.1755-1767. Langues : Anglais (eng) in Autism Research > 15-9 (September 2022) . - p.1755-1767 Mots-clés : Autism Spectrum Disorder  Autistic Disorder  Child  Child, Preschool  Communication  Fragile X Syndrome  Humans  Male  Reproducibility of Results  autism  fragile X syndrome  naturalistic  outcome measure  weighting communication scores Index. décimale : PER Périodiques Résumé : In the present study, we provide a preliminary evaluation of the validity and reliability of using weighting procedures to measure communication in play samples for preschool-aged boys with autism or fragile X syndrome (FXS). Because weighting procedure communication scores (WPCSs) reflect growth in both communicative frequency and complexity, establishing the psychometrics of the component scores, in addition to the overall metric, affords investigators the opportunity to describe growth within and across skills. Results, for both groups, provide support regarding the psychometric appropriateness (i.e., convergent validity, divergent validity, and internal consistency) for all WPCSs. That said, a trend was observed for reliability scores to be slightly lower or more variable in boys with autism than in boys with FXS. Finally, although significant associations were observed in the associations between WPCSs across play contexts, contexts effects were observed for all three WPCSs. Together, results from this study provide promising preliminary data indicating the utility of using WPCSs in children with neurodevelopmental disabilities. Lay Summary: Language supports long-term positive outcomes; it is important to identify accurate and flexible ways of measuring language in children over time. We considered the effectiveness of using a procedure that considers changes in the number of communication acts and the types of acts produced during a play session by preschool-aged boys with autism or fragile X syndrome. These procedures were found to be valid and reliable. En ligne : http://dx.doi.org/10.1002/aur.2724 Permalink : https://www.cra-rhone-alpes.org/cid/opac_css/index.php?lvl=notice_display&id=483

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