Parents, Clinicians Assess Angelman Behaviors Differently, Study Finds
Agreement highest for hyperactivity in children, lowest for self-injury
Behavioral assessments by parents of children with Angelman syndrome and other genetic neurodevelopmental disorders substantially differed from those reported by clinicians, a study revealed.
Appraisal of hyperactivity showed the highest agreement between parents and professionals, whereas self-injury behaviors showed the poorest agreement.
These findings have implications for clinical trials, supporting a multi-informant approach in which the behavioral observations of both parents and clinicians are considered as outcome measures, the researchers noted.
The study, “Agreement between parents’ and clinical researchers’ ratings of behavioral problems in children with fragile X syndrome and chromosome 15 imprinting disorders,” was published in the journal Research in Developmental Disabilities.
Aggressive behaviors reported by 19% of parents, 64% of clinicians
Angelman syndrome is marked by developmental delays and physical and intellectual disabilities, as well as troubling behaviors. Most cases are caused by a deletion of a region of the maternally inherited chromosome 15 that contains the UBE3A gene.
Clinical trials testing interventions aiming to improve behavior in children with genetic neurodevelopmental disorders such as Angelman have shown limited success. It has been suggested that behavioral outcome data from these trials are limited because they often rely solely on parental reports or brief observations by a clinician.
However, research examining the agreement between parents’ and clinicians’ behavioral ratings in children with these conditions is lacking.
To address this gap, a team led by researchers in Australia compared parents’ and clinicians’ reports on 123 children, ages 3–17 years, diagnosed with genetic neurodevelopmental disorders associated with behavioral abnormalities.
Participants (64% were boys), included 15 children with Angelman syndrome, 19 with Prader-Willi syndrome (PWS), 79 with fragile X syndrome, and 10 with chromosome 15q duplication (Dup15q) syndrome.
All Angelman, PWS, and Dup15q patients were recruited in Australia, while children with fragile X were recruited in Australia and Chile.
Patients were assessed at a mean age of 8.29 years (range 3.2–16.9). Intellectual disabilities were identified in 99 (84%) of the 120 children with evaluable data, and about half of the study’s participants were receiving at least one type of psychotropic medication.
The final analysis did not include children with Dup15q due to their small number.
Parents’ ratings were based on the aberrant behavior checklist-community (ABC-C), a rating scale designed to evaluate abnormal behavior in individuals with intellectual disability. It is “considered a reliable and efficient method to measure treatment outcomes in trials involving children with [neurodevelopmental disorders],” the researchers wrote.
Clinicians’ ratings were based on the autism diagnostic observation schedule-second edition (ADOS-2), “a ‘gold standard’ assessment of autism symptoms and other behaviors” that can be used to measure abnormal behavior severity, they added.
Items selected from ABC-C and ADOS-2 for behavioral assessment included mannerisms/repetitive behaviors, self-injury, hyperactivity, and tantrums/aggressive behaviors due to “their high prevalence in the populations under investigation, and due to the existence of similar areas of assessment on both measures,” the team wrote.
Scores for all items on the ABC-C and ADOS-2 scales range from 0 (not observed/not a problem) to 3 (moderate or severe behavior), except for the self-injury item on the ADOS-2, which is scored from 0 to 2.
Differences in the frequency of raw 0 scores between parents and clinicians were the smallest (13%) for the mannerisms/repetitive behaviors domain, with absence of these behaviors being reported by 28% of parents and by 41% of clinicians.
In contrast, this difference was largest (45%) for tantrums/aggressive behaviors, with 19% of parents reporting no such behaviors compared with 64% of clinicians.
Self-injury was the least frequently reported behavior, with their absence being reported by 63% of parents and 91% of clinicians.
Agreement between the raw ABC-C and ADOS-2 scores was then calculated to generate intraclass correlation coefficients (ICC), in which 0 indicates poor agreement, and 1 reflects perfect agreement.
Across all children, the agreement between parents’ and clinicians’ ratings was lowest for self-injury (ICC of 0.12), and fair for mannerisms/repetitive behaviors (ICC of 0.25) and tantrums/aggression behaviors (ICC of 0.24). Hyperactivity behavior showed the greatest agreement (ICC of 0.48; classified as moderate).
When stratified by the children’s diagnosis, ICC ranged from poor (0 for self-injury in both Angelman and PWS patients) to substantial (0.48 for hyperactivity in girls with fragile X).
‘Both perspectives’ needed for better clinical trial measures
Among Angelman children specifically, self-injury was the least frequently reported behavior by parents and clinicians for both boys and girls. Hyperactivity was the most frequent problematic behavior reported by parents (80%), while mannerisms/repetitive behaviors were highly endorsed by clinicians (93%).
Agreement between parents and clinicians’ scores ranged from poor for self-injury (ICC of 0) to fair for tantrums/aggressive behaviors (ICC of 0.21) and mannerisms/repetitive behaviors (ICC of 0.38). A slight agreement was observed for hyperactivity (ICC of 0.15).
“By comparing parent reports and clinical researchers’ direct observations of behavioral problems in children with neurodevelopmental disorders, we demonstrate highly discrepant appraisal of the behaviors investigated,” the researchers wrote.
These findings “have implications for clinical trials in these neurodevelopmental disorders and suggest that both parents’ and researchers’ observations of behavior should be considered as outcome measures and taken into account to develop new sensitive outcome measures that integrate both perspectives,” they added.
This study supports “a multi-informant approach,” whose adoption “in future clinical trials could increase the chance of clinical trial success and translation of effective treatments into clinical practice,” the researchers concluded.
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