A Patient Report Outcome Measure of Sensory Difficulties in Autism Spectrum Disorder

Background:

The addition of hyper or hypo reactivity to sensory input as diagnostic criterion in Autism Spectrum Disorder (ASD) calls for a new approach to identify and quantify atypical sensory behavior in daily life. Studies report both over and under responsiveness in children with ASD and similar physiological sensory processing deficits can lead to a variety of behaviors. Currently used instruments cannot capture the wide heterogeneity of these behaviors: the questionnaires are often diagnostic tools, developed from the perspective of the professional and symptom based. Besides, it is acknowledged that current tools and questionnaires for ASD have significant limitations to be used as a patient-oriented outcome measure for intervention research. They have no established cut-off for clinically relevant change, a minimal important difference. Considering the recent focus on and rise in novel interventions aimed at treating aberrant sensory processing, the need arises for relevant outcomes from the perspective of patients and/or caregivers themselves that can be used in both clinical and research settings.

Objectives:

To develop and validate a patient-reported outcome measure (PROM) for problems arising from sensory reactivity difficulties in daily life that additionally serves as (relevant) effect measure in intervention studies.

Methods:

This is a mixed-methods study guided by the iterative approach described in the US Food and Drug Administration Guidance for Industry Patient-reported Outcome Measures. Information from 20 parents of patients with different severity of ASD and intellectual functioning was collected. Qualitative thematic data analysis resulted in main themes, pool of items and response options, providing a pilot version of the PROM. Weighting was added to individualize the PROM. ‘Think aloud’-procedures will be used to evaluate and optimize pilot versions and psychometric testing will be carried out to assess validity, reliability and sensitivity to change.

Results:

First results show major discrepancy between items on conventional questionnaires and our reported main themes. We have identified novel and meaningful problem areas, such as fatigue, anger outbursts and attentional problems, which currently used questionnaires have failed to address. Results of the development of the PROM and preliminary data will be presented.

Conclusions:

By using a novel approach to identify and quantify sensory reactivity deficits in children with ASD in daily life, we will be able to develop a multidimensional and individually tailored PROM. Now we can identify and compare sensory reactivity deficits on both individual and group level for future application as outcome measure for interventions and clinical trials.