Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome.

BACKGROUND: In 1998 secretin, a gastrointestinal hormone, was suggested as an effective treatment for autism spectrum disorders (ASD) based on anecdotal evidence. OBJECTIVES: To assess whether intravenous secretin improves the core features of ASD, other aspects of behaviour or function such as self-injurious behaviour, and the quality of life of affected individuals and their carers. We also assessed whether secretin causes harm. This is an updated version of our review of this topic originally published in 2005. SEARCH METHODS: We searched CENTRAL (2010 Issue 1), MEDLINE (1950 to January 2010) , EMBASE (1980 to 2010 Week 2), PsycINFO (1806 to 2010 Week 2), CINAHL (1938 to January 2010), ERIC (1966 to January 2010), Sociological Abstracts (1952 to January 2010). Sociofile and HealthStar were searched in March 2005 when this review was first published, but were not available for this update. Records were limited to studies published since 1998 as this is when secretin was first proposed as a possible treatment for ASD. We searched reference lists of trials and reviews; we also contacted experts and trialists to find unpublished studies. SELECTION CRITERIA: Randomised controlled trials of intravenous secretin compared to a placebo treatment in children or adults diagnosed with ASD, where at least one standardised outcome measure was reported. DATA COLLECTION AND ANALYSIS: Sixteen studies met the inclusion criteria but for two of these, conducted by Repligen, the only available multisite data were reported in press releases. All outcome data from the other 14 trials were continuous. Where trials used cross-over designs, we conducted analysis on results from the first treatment phase. Where mean change from baseline was reported, we used this in preference to post-treatment scores for meta-analyses or forest plots. Meta-analysis was able to be attempted for only one outcome (Childhood Autism Rating Scale). Insufficient data were available to conduct sensitivity or subgroup analyses to assess the impact of study quality, clinical differences in the intervention or clinically relevant differences between groups, such as age or presence of gastrointestinal symptoms. MAIN RESULTS: Over 900 children were recruited for the secretin trials. Twenty-five established standardised outcome measures were reported to assess core features of ASD, communication, behaviour, visuospatial skills, affect and adverse events. One standardised measure of global impression was also used. No more than four studies used any one outcome measure similarly. When duration from the start of the intervention to outcome assessment was known, outcomes were reported at between three and six weeks. Meta-analysis of data was not possible but there is now consistency of findings, with RCTs of the efficacy of secretin in autism not showing improvements for core features of ASD. AUTHORS' CONCLUSIONS: There is no evidence that single or multiple dose intravenous secretin is effective and as such currently it should not be recommended or administered as a treatment for ASD. Further experimental assessment of secretin's effectiveness for ASD can only be justified if there is new high-quality and replicated scientific evidence that either finds that secretin has a role in neurotransmission in a way that could benefit all children with ASD or identifies important subgroups of children with ASD who could benefit from secretin because of a proven link between the action of secretin and the known cause of their ASD, or the type of problems they are experiencing.

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Intravenous secretin for autism spectrum disorders (ASD).

OBJECTIVES: To provide a concise and practical guide to the development, modification, and use of behavioral coding schemes for observational data in pediatric psychology. METHODS: This article provides a review of relevant literature and experience in developing and refining behavioral coding schemes. RESULTS: A step-by-step guide to developing and/or modifying behavioral coding schemes is provided. Major steps include refining a research question, developing or refining the coding manual, piloting and refining the coding manual, and implementing the coding scheme. Major tasks within each step are discussed, and pediatric psychology examples are provided throughout. CONCLUSIONS: Behavioral coding can be a complex and time-intensive process, but the approach is invaluable in allowing researchers to address clinically relevant research questions in ways that would not otherwise be possible. Language: en

Developing and Modifying Behavioral Coding Schemes in Pediatric Psychology: A Practical Guide

Our study examined stability of self-regulation and affective expression in children with autism or Down syndrome over a 2 year period. A behaviorally-anchored rating scale was used to assess a self-regulation factor (attention, adaptability, object orientation, and persistence), negative affect factor (hostility, irritability, and compliance), and positive affect factor (positive affect, affective sharing, and dull affect) from videotapes of play sessions involving each child and his or her mother. The patterns of ratings within each group were similar from time 1 to time 2, with the autism group showing more deviant ratings on measures of self-regulation and affective sharing. From time 1 to time 2, children with autism showed relatively high stability for the self-regulation factor, but less stability than children with Down syndrome for all three factors.

Self-regulation and affective expression during play in children with autism or Down Syndrome: a short-term longitudinal study.

BACKGROUND\nSecretin is a gastro-intestinal hormone which has been presented as an effective treatment for autism based on anecdotal evidence.\n\n\nOBJECTIVES\nTo determine if intravenous secretin:1. improves the core features of autism (social interaction, communication and behaviour problems); 2. improves the non-core aspects of behaviour or function such as self injurious behaviour;3. improves the quality of life of affected individuals and their carers; 4. has short term and long term effects on outcome; 5. causes harm.\n\n\nSEARCH STRATEGY\nResults of electronic searches of CENTRAL, MEDLINE, EMBASE, PsycINFO, CINAHL, ERIC, HealthStar and Sociofile (1998 - March 2005) were independently examined by two authors. Reference lists of trials and reviews were searched; experts and trialists were contacted to find unpublished studies.\n\n\nSELECTION CRITERIA\nRandomised controlled trials of intravenous secretin comparing secretin with a placebo treatment in children or adults diagnosed with autism spectrum disorders, where at least one standardised outcome measure was reported.\n\n\nDATA COLLECTION AND ANALYSIS\nFourteen studies met inclusion criteria. All outcome data were continuous. Where trials used cross-over designs, analysis was conducted on results from first treatment phase, allowing combined analysis with parallel design trials. Where standardised assessment tools generated scores as outcome measures, comparisons were made between means of these scores. Where baseline means were reported, differences between treatment and control were determined to assess possible bias. Where mean change from baseline was reported, this was used in preference to post-treatment scores for meta-analyses or forest plots. As meta-analysis was possible for only one outcome (Childhood Autism Rating Scale), it was impossible to use sensitivity or subgroup analyses to assess impact of study quality, clinical differences in the intervention, or clinically relevant differences between groups, such as age or presence of gastrointestinal symptoms.\n\n\nMAIN RESULTS\nTwenty-five established standardised outcome measures were reported to assess core features of autism, communication, behaviour, visio-spatial skills, affect and adverse events within fourteen included studies. No more than four studies used any one outcome measure similarly. Outcomes were reported between three and six weeks. RCTs of efficacy of secretin in autism have not shown improvements for core features of autism.\n\n\nAUTHORS' CONCLUSIONS\nThere is no evidence that single or multiple dose intravenous secretin is effective and as such it should not currently be recommended or administered as a treatment for autism. Further experimental assessment of secretin's effectiveness for autism can only be justified if methodological problems of existing research can be overcome.

Intravenous secretin for autism spectrum disorder (Review)

Assessing the impact of disease and treatment of the emotional state and temperament of preschool children has been limited by the lack of sensitive and objective measurement techniques. To construct such a measure for longitudinal use, a sample of 179 children with febrile seizures and 85 normal children were used to develop the Minnesota Preschool Affect Rating Scales (MN-PARS). Video-taped play sessions were a source of behaviorally anchored ratings on 12 scales. Factor analysis yielded three factors of Negative Affect, Positive Affect, and Self-regulation with additional individual scales of Dependency and Activity Level. These scales and factors yield reliable ratings, as measured by interrater agreement and split-half techniques, as well as initial evidence of concurrent validity. Although they were developed for measuring the behavioral effects of phenobarbital on children with febrile seizures, these scales provide an objective means of measuring emotional expression and self-regulation useful for other studies.

Jeffrey T. Mcphee

Papers

Minnesota Preschool Affect Rating Scales: Development, Reliability, and Validity