Meaningful health outcomes for paediatric neurodisability: stakeholder prioritisation and appropriateness of patient reported outcome measures

BACKGROUND: Health services are increasingly focused on measuring and monitoring outcomes, particularly those that reflect patients' priorities. To be meaningful, outcomes measured should be valued by patients and carers, be consistent with what health professionals seek to achieve, and be robust in terms of measurement properties. The aim of this study was (i) to seek a shared vision between families and clinicians regarding key aspects of health as outcomes, beyond mortality and morbidity, for children with neurodisability, and (ii) to appraise which multidimensional patient reported outcome measures (PROMs) could be used to assess salient health domains. METHODS: Relevant outcomes were identified from (i) qualitative research with children and young people with neurodisability and parent carers, (ii) Delphi survey with health professionals, and (iii) systematic review of PROMs. The International Classification of Functioning Disability and Health provided a common language to code aspects of health. A subset of stakeholders participated in a prioritisation meeting incorporating a Q-sorting task to discuss and rank aspects of health. RESULTS: A total of 33 pertinent aspects of health were identified. Fifteen stakeholders from the qualitative and Delphi studies participated in the prioritisation meeting: 3 young people, 5 parent carers, and 7 health professionals. Aspects of health that emerged as more important for families and targets for health professionals were: communication, emotional wellbeing, pain, sleep, mobility, self-care, independence, mental health, community and social life, behaviour, toileting and safety. Whilst available PROMs measure many aspects of health in the ICF, no single PROM captures all the key domains prioritised as for children and young people with neurodisability. The paucity of scales for assessing communication was notable. CONCLUSIONS: We propose a core suite of key outcome domains for children with neurodisability that could be used in evaluative research, audit and as health service performance indicators. Future work could appraise domain-specific PROMs for these aspects of health; a single measure assessing the key aspects of health that could be applied across paediatric neurodisability remains to be developed

Outcomes and predictors in preschoolers with speech-language and/or developmental mobility impairments

The purpose of this article is to describe communicative-participation outcomes measured by the Focus on the Outcomes of Communication Under Six (FOCUS©; Thomas-Stonell et al., 2013) for interventions provided by speech-language pathologists (SLPs) in different community settings for preschoolers with speech-language impairments (Sp/LI) with and without developmental mobility impairments (MI). The predictive relationships between communicative-participation and (1) functioning-and-disability, and (2) contextual factors, was also investigated. Sixty-one preschoolers with Sp/LI and their parents participated. Twenty-six preschoolers were identified with Sp/LI and received speech-language interventions (Group 1), 20 preschoolers were identified with Sp/LI and MI and received speech-language interventions (Group 2), and 15 preschoolers with Sp/LI awaiting intervention served as waitlist controls (Group 3). Parents completed structured interviews about children’s communicative-participation outcomes using the FOCUS© at three time points (pre-intervention, post-intervention, and 3-months post-intervention) with an SLP. Only Groups 1 and 2 experienced statistically and clinically meaningful communicative-participation outcomes over time as measured by the FOCUS©. Pre- to post-intervention communicative-participation was predicted by functioning-and-disability and contextual factors, initial social skills and intervention status, respectively. Post-intervention to 3-month post-intervention scores were also predicted by functioning-and-disability and contextual factors, risk status (Sp/LI only, Sp/LI+developmental MI) and intervention status, respectively. Significant and clinically meaningful changes in communicative-participation over time are associated with speech-language interventions for preschoolers with Sp/LI. </jats:p

Construct validity of the <scp>FOCUS</scp> ^© ( <scp>F</scp> ocus on the <scp>O</scp> utcomes of <scp>C</scp> ommunication <scp>U</scp> nder <scp>S</scp> ix): a communicative participation outcome measure for preschool children

OBJECTIVE: The aim of this study was to establish the construct validity of the Focus on the Outcomes of Communication Under Six (FOCUS(©)). This measure is reflective of concepts in the International Classification of Functioning Disability and Health – Children and Youth framework. It was developed to capture ‘real-world’ changes (e.g. communicative participation) in preschoolers' communication following speech-language intervention. METHOD: A pre–post design was used. Fifty-two parents of 3- to 6-year-old preschoolers attending speech-language therapy were included as participants. Speech-language therapists provided individual and/or group intervention to preschoolers. Intervention targeted: articulation/phonology, voice/resonance, expressive/receptive language, play, and use of augmentative devices. Construct validity for communicative participation was assessed using pre-intervention and post-intervention parent interviews using the FOCUS(©) and the communication and socialization domains of the Vineland Adaptive Behavior Scales-II (VABS-II). RESULTS: Significant associations were found between the FOCUS(©), measuring communicative participation, and the VABS-II domains for: (i) pre-intervention scores in communication (r = 0.53, P < 0.001; 95% CI 0.30–0.70) and socialization (r = 0.67, P < 0.001; 95% CI 0.48–0.80); (ii) change scores over-time in communication (r = 0.45, P < 0.001; 95% CI 0.201–0.65) and socialization (r = 0.39, P = 0.002; 95% CI 0.13–0.60); and (iii) scores at post-intervention for communication (r = 0.53, P < 0.001; 95% CI 0.30–0.70) and for socialization (r = 0.37, P = 0.003; 95% CI 0.11–0.50). CONCLUSIONS: The study provided evidence on construct validity of the FOCUS(©) for evaluating real-world changes in communication. We believe that the FOCUS(©) is a useful measure of communicative participation

Exploring participation and impairment-based outcomes for Target Word™: A parent-implemented intervention for preschoolers identified as late-to-talk

© The Author(s) 2019. This study explored participation- and impairment-based outcomes for 24 late-to-talk toddlers (M age = 20.46 months, SD = 3.09, 62.5% male) whose parents participated in Target Word™, The Hanen Program® for Parents of Children who are Late Talkers in community clinics across Ontario. Parents completed the MacArthur–Bates Communicative Development Inventories (MBCDI), The Focus on the Outcomes of Communication Under Six (FOCUS-34), and a speech sound checklist: (1) prior to starting the program, (2) at the end of direct intervention, and (3) after a consolidation period. Speech-language pathologists classified children’s communicative function using the Communication Function Classification System (CFCS) at all assessment points and provided FOCUS scores from an initial assessment. No change was recorded in CFCS levels between initial assessment and start of the program, but many children moved to a more advanced level by the end of the program (n = 12) or consolidation period (n = 19). Significant changes in children’s communicative participation skills (FOCUS) were found between initial assessment and the start of the program, and during the 12-week direct intervention (75% made clinically meaningful change). Significant growth in expressive vocabulary and consonant inventory occurred during direct intervention. Correlations between change on impairment- and participation-based measures were not significant. The Target Word program appears to improve communicative function for late-to-talk preschoolers. Children also made gains in communicative participation skills, expressive vocabulary, and consonant inventory during the program, but further research is needed to determine the effectiveness of Target Word in these areas