Helen McConachie

ArticlesParent-mediated communication-focused treatment in children with autism (PACT): a randomised controlled trial

Summary Background Results of small trials suggest that early interventions for social communication are effective for the treatment of autism in children. We therefore investigated the efficacy of such an intervention in a larger trial. Methods Children with core autism (aged 2 years to 4 years and 11 months) were randomly assigned in a one-to-one ratio to a parent-mediated communication-focused (Preschool Autism Communication Trial [PACT]) intervention or treatment as usual at three specialist centres in the UK. Those assigned to PACT were also given treatment as usual. Randomisation was by use of minimisation of probability in the marginal distribution of treatment centre, age (≤42 months or >42 months), and autism severity (Autism Diagnostic Observation Schedule-Generic [ADOS-G] algorithm score 12–17 or 18–24). Primary outcome was severity of autism symptoms (a total score of social communication algorithm items from ADOS-G, higher score indicating greater severity) at 13 months. Complementary secondary outcomes were measures of parent-child interaction, child language, and adaptive functioning in school. Analysis was by intention to treat. This study is registered as an International Standard Randomised Controlled Trial, number ISRCTN58133827. Results 152 children were recruited. 77 were assigned to PACT (London [n=26], Manchester [n=26], and Newcastle [n=25]); and 75 to treatment as usual (London [n=26], Manchester [n=26], and Newcastle [n=23]). At the 13-month endpoint, the severity of symptoms was reduced by 3·9 points (SD 4·7) on the ADOS-G algorithm in the group assigned to PACT, and 2·9 (3·9) in the group assigned to treatment as usual, representing a between-group effect size of −0·24 (95% CI −0·59 to 0·11), after adjustment for centre, sex, socioeconomic status, age, and verbal and non-verbal abilities. Treatment effect was positive for parental synchronous response to child (1·22, 0·85 to 1·59), child initiations with parent (0·41, 0·08 to 0·74), and for parent-child shared attention (0·33, −0·02 to 0·68). Effects on directly assessed language and adaptive functioning in school were small. Interpretation On the basis of our findings, we cannot recommend the addition of the PACT intervention to treatment as usual for the reduction of autism symptoms; however, a clear benefit was noted for parent-child dyadic social communication. Funding UK Medical Research Council, and UK Department for Children, Schools and Families.

Daslne: the challenge of developing a regional database for autism spectrum disorder

The setting up of a database of children with autism spectrum disorder (ASD) in the north east of England is described. Best practice has been followed and included involving parents in planning and implementation at all stages, oversight by a multi-agency group, management by a multidisciplinary steering group, and independent administration of the database. From a potential listing of 986 children with ASD aged 3–12 years, the parents of 511 have so far responded (51.8%), although response rate varies considerably by local authority. Data checking has shown the information to be valid and case ascertainment broadly representative. The uses to which the data are being put and the continuing challenges are outlined.

Predictors of reliable symptom change: Secondary analysis of the Preschool Autism Communication Trial:

Background and aims Despite recent gains in the amount and quality of early autism intervention research, identifying what works for whom remains an ongoing challenge. Exploiting data from the Preschool Autism Communication Trial (PACT), we undertook secondary analysis to explore prognostic indicators and predictors of response to one year of PACT therapy versus treatment as usual within this large and rigorously characterised cohort recruited across three UK trial sites. Methods In this secondary analysis of variability in child gains on the primary trial outcome measure – social-communication symptom severity – we used a pragmatic and data-driven approach to identify a subgroup of children who showed reliable improvement and a subgroup showing clear lack thereof. We then tested which among several baseline child and family factors – including measures routinely collected in research trials and clinical practice – varied as a function of child outcome status and treatment group. Results Greater baseline child non-verbal ability was a significant prognostic indicator of symptom reduction over time (i.e. irrespective of treatment group). By contrast, parent synchrony presented as marginal predictor, and trial recruitment site as a significant predictor, of differential outcome by treatment group. Specifically, lower parent synchrony showed some association with poorer outcomes for children from families assigned to treatment as usual (but with no such effect for those assigned to PACT). Similarly, children at one recruitment site were more likely to have poorer outcomes if assigned to treatment as usual, compared to children at the same site assigned to PACT. Conclusions The current data contribute to an evidence base indicting that early non-verbal ability is a robust indicator of generally better prognosis for young children with autism. Lower parent synchrony and a broadly more deprived socio-geographical context may inform the appropriate targeting of PACT. That is, given that the former factors predicted poorer outcome in children from families assigned to treatment as usual, the receipt of a relatively low-dose, parent-mediated and communication-focused therapy might be developmentally protective for young children with autism. Nevertheless, results from this study also highlight the paucity of meaningful predictors of outcome among routine clinical characterisation measures such as those investigated here. Implications Understanding the factors associated with differential treatment outcomes is critical if we are to individualise treatment decisions for children with autism. Inherently tied to this objective is a need to delineate those factors which specifically predict positive response (or lack of response) to one or other treatment option, versus those that indicate generally better (or poorer) prognosis, irrespective of treatment.