The Patient-Oriented Eczema Measure in young children:Responsiveness and minimal clinically important difference

BACKGROUND: The Patient‐Oriented Eczema Measure (POEM) has been recommended as the core patient‐reported outcome measure for trials of eczema treatments. Using data from the Choice of Moisturiser for Eczema Treatment randomized feasibility study, we assess the responsiveness to change and determine the minimal clinically important difference (MCID) of the POEM in young children with eczema. METHODS: Responsiveness to change by repeated administrations of the POEM was investigated in relation to change recalled using the Parent Global Assessment (PGA) measure. Five methods of determining the MCID of the POEM were employed; three anchor‐based methods using PGA as the anchor: the within‐patient score change, between‐patient score change and sensitivity and specificity method, and two distribution‐based methods: effect size estimate and the one half standard deviation of the baseline distribution of POEM scores. RESULTS: Successive POEM scores were found to be responsive to change in eczema severity. The MCID of the POEM change score, in relation to a slight improvement in eczema severity as recalled by parents on the PGA, estimated by the within‐patient score change (4.27), the between‐patient score change (2.89) and the sensitivity and specificity method (3.00) was similar to the one half standard deviation of the POEM baseline scores (2.94) and the effect size estimate (2.50). CONCLUSIONS: The Patient‐Oriented Eczema Measure as applied to young children is responsive to change, and the MCID is around 3. This study will encourage the use of POEM and aid in determining sample size for future randomized controlled trials of treatments for eczema in young children

Development and validation of the Multimorbidity Treatment Burden Questionnaire (MTBQ)

OBJECTIVE: To develop and validate a new scale to assess treatment burden (the effort of looking after one's health) for patients with multimorbidity. DESIGN: Mixed-methods. SETTING: UK primary care. PARTICIPANTS: Content of the Multimorbidity Treatment Burden Questionnaire (MTBQ) was based on a literature review and views from a patient and public involvement group. Face validity was assessed through cognitive interviews. The scale was piloted and the final version was tested in 1546 adults with multimorbidity (mean age 71 years) who took part in the 3D Study, a cluster randomised controlled trial. For each question, we examined the proportion of missing data and the distribution of responses. Factor analysis, Cronbach's alpha, Spearman's rank correlations and longitudinal regression assessed dimensional structure, internal consistency reliability, construct validity and responsiveness, respectively. We assessed interpretability by grouping the global MTBQ scores into zero and tertiles (>0) and comparing participant characteristics across these categories. RESULTS: Cognitive interviews found good acceptability and content validity. Factor analysis supported a one-factor solution. Cronbach's alpha was 0.83, indicating internal consistency reliability. The MTBQ score had a positive association with a comparator treatment burden scale (rs 0.58, P<0.0001) and with self-reported disease burden (rs 0.43, P<0.0001), and a negative association with quality of life (rs-0.36, P<0.0001) and self-rated health (rs-0.36, P<0.0001). Female participants, younger participants and participants with mental health conditions were more likely to have high treatment burden scores. Changes in MTBQ score over 9-month follow-up were associated, as expected, with changes in measures of quality of life (EuroQol five dimensions, five level questionnaire) and patient-centred care (Patient Assessment of Chronic Illness Care). CONCLUSION: The MTBQ is a 10-item measure of treatment burden for patients with multimorbidity that has demonstrated good content validity, construct validity, reliability and responsiveness. It is a useful research tool for assessing the impact of interventions on treatment burden. TRIAL REGISTRATION NUMBER: ISRCTN06180958

Who should we ask about mental health symptoms in adolescents with CFS/ME? Parent-child agreement on the Revised Children’s Anxiety and Depression Scale

Background:One in three adolescents with chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME) have mental health problems. Multi-informant perspectives are key to psychological assessment. Understanding parent-child agreement is crucial to accurate diagnosis, particularly where severe fatigue limits self-report.Methods:Agreement on the revised children’s anxiety and depression scale (RCADs) was assessed between parents and children with CFS/ME (n = 93) using Bland-Altman plots, cross tabulations and regression analyses.Results:Diagnostic thresholds were met more frequently based on child-report. Parent- and child-report had similar sensitivity and specificity on RCADS compared to gold-standard diagnostic interviews. Regression analysis found similar accuracy between both reports. For anxiety diagnoses, odds ratio (OR) for child-report was 1.10 (CI = 1.06–1.14), and 1.10 (CI = 1.05–1.14) for parent-report. For depression, OR for child report was 1.26 (CI = 1.11–1.43), while for parent-report is was 1.25 (CI = 1.10–1.41). For total score, OR for child-report was 1.10 (CI = 1.05–1.13) while OR for parent-report was 1.09 (CI = 1.05–1.13).Conclusions:Reasonable agreement was observed between parent- and child-report of mental health symptoms in paediatric CFS/ME. While parent-report can facilitate psychological evaluation in CFS/ME, this is not a substitute for a child’s own report

Who should we ask about mental health symptoms in adolescents with CFS/ME? Parent-child agreement on the Revised Children’s Anxiety and Depression Scale

BACKGROUND: One in three adolescents with chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME) have mental health problems. Multi-informant perspectives are key to psychological assessment. Understanding parent-child agreement is crucial to accurate diagnosis, particularly where severe fatigue limits self-report. METHODS: Agreement on the revised children’s anxiety and depression scale (RCADs) was assessed between parents and children with CFS/ME (n = 93) using Bland-Altman plots, cross tabulations and regression analyses. RESULTS: Diagnostic thresholds were met more frequently based on child-report. Parent- and child-report had similar sensitivity and specificity on RCADS compared to gold-standard diagnostic interviews. Regression analysis found similar accuracy between both reports. For anxiety diagnoses, odds ratio (OR) for child-report was 1.10 (CI = 1.06–1.14), and 1.10 (CI = 1.05–1.14) for parent-report. For depression, OR for child report was 1.26 (CI = 1.11–1.43), while for parent-report is was 1.25 (CI = 1.10–1.41). For total score, OR for child-report was 1.10 (CI = 1.05–1.13) while OR for parent-report was 1.09 (CI = 1.05–1.13). CONCLUSIONS: Reasonable agreement was observed between parent- and child-report of mental health symptoms in paediatric CFS/ME. While parent-report can facilitate psychological evaluation in CFS/ME, this is not a substitute for a child’s own report

Diagnosis, assessment and treatment of childhood eczema in primary care:cross-sectional study

Background: The majority of children with eczema in the UK are looked after in primary care yet we know little about their care in this setting. Aim: To compare the diagnosis, assessment, and treatment of eczema in primary care with published diagnostic criteria and management guidelines. Design & setting: Cross-sectional study using data from a randomised controlled feasibility study. General practices, UK. Method: Baseline data from children aged 1 month to 5 years recruited ‘in-consultation’ for the Choice of Moisturiser in Eczema Treatment (COMET) feasibility study was used. These included clinician diagnosis and global severity assessment; the parent-completed Patient Orientated Eczema Measure (POEM); a questionnaire about eczema treatments, including use of topical corticosteroid (TCS); and, the Eczema Area Severity Index (EASI) carried out by trained researchers. Descriptive analyses were undertaken to compare diagnoses with UK diagnostic criteria, severity assessments, and treatment with the National Institute for Health and Care Excellent (NICE) guidance. Results: Data were available for 90 participants. Only 46% of participants labelled as having eczema met the UK diagnostic criteria. Agreement between the global severity assessment by a healthcare practitioner with the EASI and POEM measures of eczema severity were 44% and 48% respectively. Emollients and TCSs were underused with 44% of participants not using any emollient and 46% using one or more TCSs. The ‘match’ between eczema severity and TCSs potency was poor. Conclusion: Discrepancies were found between the diagnosis, assessment, and treatment of children with eczema in primary care, and UK diagnostic criteria and guidelines. Further investigation to explore the reasons for this discordance, and whether it matters, is needed

Exceeding the recruitment target in a primary care paediatric trial:an evaluation of the Choice of Moisturiser for Eczema Treatment (COMET) feasibility randomised controlled trial

BACKGROUND: Recruiting to target in randomised controlled trials of investigational medicinal products (CTIMPs) in primary care and paediatric populations is notoriously difficult. More evidence is needed for effective recruitment strategies in these settings. We report on the impact of different recruitment strategies used in the Choice of Moisturiser in Eczema Treatment (COMET) study – a feasibility trial comparing the effectiveness of four emollients for the treatment of childhood eczema – recruiting via general practitioner (GP) surgeries. METHODS: Initially, 16 GP practices invited potentially eligible children to take part in the trial by sending an invitation letter (self-referral pathway) or by consenting and randomising them into the study during a visit to the practice (in-consultation referral). Measures implemented during the study to maximise accrual included signing up six additional GP practices, increasing the upper age limit eligibility criterion from 3 to 5 years, and permitting healthcare professionals other than doctors to confirm participant eligibility. We used descriptive statistics and univariate linear regression models to explore associations with practice recruitment rates. RESULTS: A total of 197 participants were recruited, exceeding the target of 160. Of these, 107 children entered via self-referral and 90 by in-consultation pathways. Of the recruited population, 12.6 % were aged between 3 and 5 years (the raised upper age limit). The six additional practices contributed 37.4 % (40 of 107) of participants recruited by self-referral. Only almost one-third (18 of 56 [32.1 %]) of potential recruiting clinicians recruited one or more participants in-consultation, which was a more problematic pathway because of data verification issues. Three research nurses and a pharmacist from four practices recruited 48.9 % (44 of 90) of participants via this pathway. Univariate linear regression models showed no evidence of association between the number of children recruited via the self-referral pathway by practice and practice list size (p = 0.092) or practice deprivation decile (p = 0.270), but practice deprivation was associated with a higher number of children recruited in-consultation (p = 0.020) by practice. CONCLUSIONS: Self-referral and in-consultation recruitment yielded similar numbers, but the in-consultation pathway was more problematic. Future trials of this type should consider the condition, normal care pathway and number of potentially eligible children and be prepared to use multiple recruitment strategies to achieve recruitment targets. TRIAL REGISTRATION: ISRCTN21828118. Registered on 1 May 2014. EudraCT2013-003001-26. Registered on 23 Dec 2013

Informed consent in randomised controlled trials:further development and evaluation of the participatory and informed consent (PIC) measure

Background: Informed consent is an accepted ethical and legal prerequisite for trial participation, yet there is no standardised method of assessing patient understanding for informed consent. The participatory and informed consent (PIC) measure was developed for application to recruitment discussions to evaluate recruiter information provision and evidence of patient understanding. Preliminary evaluation of the PIC indicated the need to improve inter-rater and intra-rater reliability ratings and conduct further psychometric evaluation. This paper describes the assessment, revision and evaluation of the PIC within the context of OPTiMISE, a pragmatic primary care-based trial. Methods: This study used multiple methods across two phases. In phase one, one researcher applied the existing PIC measure to 18 audio-recorded recruitment discussions from the OPTiMISE study and made detailed observational notes about any uncertainties in application. Appointments were sampled to be maximally diverse for patient gender, study centre, recruiter and before and after an intervention to optimise information provision. Application uncertainties were reviewed by the study team, revisions made and a coding manual developed and agreed. In phase two, the coding manual was used to develop tailored guidelines for applying the PIC to appointments within the OPTiMISE trial. Two researchers then assessed 27 further appointments, purposively sampled as above, to evaluate inter-rater and intra-rater reliability, content validity and feasibility. Results: Application of the PIC to 18 audio-recorded OPTiMISE recruitment discussions resulted in harmonisation of the scales rating recruiter information provision and evidence of patient understanding, minor amendments to clarify wording and the development of detailed generic coding guidelines for applying the measure within any trial. Application of the revised measure using these guidelines to 27 further recruitment discussions showed good feasibility (time to complete), content validity (completion rate) and reliability (inter- and intra-rater) of the measure. Conclusion: The PIC provides a means to evaluate the content of information provided by recruiters, patient participation in recruitment discussions and, to some extent, evidence of patient understanding. Future work will use the measure to evaluate recruiter information provision and evidence of patient understanding both across and within trials