Achieving consensus on assessing health-related quality of life (HRQoL) in people with cognitive impairments (CI)a Delphi study.

BACKGROUND: The prevalence of impairments of cognitive functions is expected to increase. Enhancing the QoL of those affected is important. HRQoL in people with CI can be assessed by self-report, proxy-report or observation but there is uncertainty how to best assess HRQoL in people with CI, and which assessment method is most appropriate. Therefore the aim of our study was to use Delphi methodology in order to achieve consensus on how HRQoL should be assessed in people with CI and which content domains should be assessed. METHODS: The Delphi process consisted of three online survey rounds and a concluding consensus conference. Participants were experts as well as individuals and relatives of people affected by CI. The Delphi survey was developed based on existing literature and included 55 statements at the first round. Consensus was considered to be achieved when a minimum of 75 % of responses fell into the categories 6 (agree) and 7 (strongly agree) (positive consensus) or in categories 1 (strongly disagree) and 2 (disagree) (negative consensus). RESULTS: Consensus was reached for a total of 41 of 56 statements/substatements. In the 1st survey round 102 experts and 11 relatives participated. In the 2nd survey round 68 experts and 11 relatives continued to participate. In the 3rd survey round 41 experts and 9 relatives participated. In the consensus conference 17 experts and 4 relatives of individuals with CI and in the second one-hour online conference session 14 experts and 2 relatives of individuals with CI participated. CONCLUSION: The combination of the three assessment methods self-report, proxy-report and observation across all stages of CI is the preferred method and should be used whenever possible. As domains Physical capacity, Psychological, Level of Independence, Social Relationships, Environment and Spirituality/Relogion/Personal Beliefs should be assessed.

VersKiK: Study protocol of an observational registry-based study on the current state of follow-up care and adherence to follow-up guidelines after cancer in childhood or adolescence.

BACKGROUND: This article describes the study design of the quantitative part of the VersKiK study, The primary objectives of this study are to examine the occurrence of late effects in survivors of childhood or adolescent cancer (module 1), investigate health-related vulnerabilities and medical service utilization within this survivor group (modules 1 and 3), and assess the alignment between documented follow-up care for cardiological and audiological late effects with guideline recommendations, along with evaluating the extent of adherence among paediatric cancer survivors (module 3). METHODS: This is a non-interventional retrospective observational cohort study. It is based on stochastically linked insurance claims data from approximately 150,000 statutory insured persons with information concerning around 25,000-30,000 cancer survivors recorded in the German Childhood Cancer Register (GCCR). To explore adherence to selected follow-up guidelines, intention to treat treatment data from clinical study groups for particular diagnostic entities will be additionally included. DISCUSSION: The growing group of survivors after cancer in childhood and adolescence is representing a special population with an increasing demand for life-long healthcare services through relative high probability of late effects. Currently, there is a limited evidence in Germany on utilization of corresponding medical services and adherence to follow-up guidelines. With this study design, we are aiming to address these gaps and, consequently, suggest improvements to existing follow-up guidelines and follow-up care provision in Germany.

Implementation of the HOME core outcome set for clinical trials of atopic eczema-barriers and opportunities: the HOME IX meeting report.

The Harmonising Outcome Measures for Eczema (HOME) initiative established a core outcome set (COS) for atopic eczema (AE) clinical trials in 2019. This set encompasses four core outcome domains and corresponding measurement instruments: clinical signs (EASI), patient-reported symptoms (POEM and NRS 11 point for worst itch over the last 24 h), quality of life (DLQI/CDLQI/IDQoLI), and long-term control (Recap or ADCT). Following its roadmap, the HOME initiative is now focused on supporting implementation of the COS. To identify barriers and facilitators to implementation of the COS, and to guide the effort to promote COS uptake, a virtual consensus meeting was held over 2 days (September 25-26, 2021) attended by 55 participants (26 healthcare professionals, 16 methodologists, 5 patients, 4 industry representatives, and 4 students). Implementation themes were identified by a pre-meeting survey distributed to HOME members, presentations, and whole-group discussion. Participants were divided into five multi-professional small groups which ranked their top 3 most important themes, followed by whole-group discussion and anonymous consensus voting (consensus criteria: < 30% disagreement). Three most important implementation themes were identified and agreed upon: (1) awareness and stakeholder engagement, (2) universal applicability of the COS, and (3) ensuring minimum administrative burden. Working groups to address these issues are now a priority for the HOME initiative. The results from this meeting will inform the development of a HOME Implementation Roadmap in an effort to support other COS groups planning for effective implementation of their core sets.

Chronic hand eczema in Europe: Patient experiences and perspectives (CHEPEP) in qualitative interviews.

BACKGROUND: Chronic hand eczema (CHE) is a very common skin disease among the European population. It causes itch and pain and, in more severe cases, seriously impairs hand functioning at work and in private life. OBJECTIVES: To explore perspectives of people with lived experience on CHE-related problems, wishes and goals. METHODS: Following a qualitative approach, we conducted topic-guided interviews in five European countries and applied template analysis to identify recurrent themes among patients with CHE. RESULTS: We interviewed 60 patients in seven outpatient dermatological and occupational medicine clinics in Croatia, Denmark, Germany, the Netherlands and Spain. Five main themes were identified: (1) knowledge about the disease and its course, (2) preventive behaviour, (3) hand eczema therapy, (4) impact on everyday life and (5) attitudes towards CHE and healthcare. Participants did not feel well informed about CHE, especially about causes, triggers and treatment options. Preventive measures were experienced as more or less effective but also cumbersome. Experiences with therapy were diverse. Treatment satisfaction depended on the results and on the perceived support from the treatment teams. Participants found it important to be taken seriously, to receive practical advice, to try out additional treatments or examinations, find new hope and have occupational perspectives. They wished that others could better understand the physical and emotional burden of CHE. Patient support groups were not mentioned. Participants found it important to learn to take care of themselves and accept life with CHE. CONCLUSIONS: Due to its annoying symptoms, high visibility and impaired functioning at work and in private life, CHE has a high emotional and social impact. Some people may require support to learn coping with CHE and its prevention. Patients wish for information about causes and triggers. They value physicians who listen to them and keep looking for solutions.

Long-term care, care needs and wellbeing of individuals after cancer in childhood or adolescence (VersKiK): study protocol of a large scale multi-methods non-interventional study.

BACKGROUND: It has been shown previously that a relevant proportion of childhood cancer survivors suffers from late effects, which are often directly related to the cancer itself or its therapy, resulting in particular follow-up needs, additionally burdening healthcare systems. Being diagnosed with cancer at a vulnerable stage of development, this group of cancer survivors is at comparatively higher risk of relapse or subsequent cancer. Although national and international follow-up guidelines based on treatment modalities have been developed, their implementation seems to leave room for improvement. Additionally, they lack a sufficient consideration of the survivors' psychosocial needs, affecting their adherence to them. The aim of the VersKiK study is to provide representative information on late effects in childhood and adolescence cancer survivors in Germany. The main research objectives are: (1) to describe the state of follow-up care among survivors after a cancer diagnosis in childhood or adolescence; (2) to quantify the occurrence of late effects among this group of survivors; (3) to examine the adherence to selected audiological and cardiological follow-up guidelines and to identify factors affecting it; (4) to explore actual follow-up needs of paediatric cancer survivors; (5) to review selected follow-up guidelines with the aim to improve and expand them. METHODS: VersKiK is designed as a mixed-methods non-interventional study. We will use claims data from statutory health insurance companies in combination with individually linked population-based registry data from the German Childhood Cancer Registry (GCCR). This data base will permit us to quantify diagnoses and procedures in comparison to the general population as well as the adherence to existing follow-up guidelines. Additional information will be obtained through interviews with childhood and adolescence cancer survivors and their informal caregivers, as well as in focus groups with healthcare professionals. DISCUSSION: The present study aims to research the actual needs of individuals after cancer diagnosis and treatment in childhood or adolescence - physical, psychological and organisational - in order to improve existing follow-up guidelines. These improvements might further positively affect not only actual care provided to paediatric cancer survivors, but also benefit healthcare systems in general while decreasing consequent medical visits in this group of patients. TRIAL REGISTRATION: Registered at German Clinical Trial Register (ID: DRKS00025960 and DRKS00026092).

Childhood maltreatment is not associated with atopic dermatitis in adults: results from a cross-sectional population-based cohort study.

BACKGROUND: Childhood maltreatment (CM) is related to poor physical and mental health outcomes in adults. Knowledge on the impact of CM on skin diseases is limited, and no study has previously addressed the association of CM with atopic dermatitis (AD) in adult age. OBJECTIVES: To analyse the prevalence of CM in individuals with physician-diagnosed AD, and to examine the relationship between different types of CM with physician-diagnosed AD in a general population sample of German adults. METHODS: Data from 2973 participants from the cross-sectional population-based Study of Health in Pomerania (SHIP) TREND-0 were analysed (aged 20 to 83 years; 51.4% female). We administered the Childhood Trauma Questionnaire (CTQ) assessing emotional, physical and sexual abuse, and emotional and physical neglect. AD was diagnosed by dermatologists in a standardized clinical examination. We conducted logistic regression analyses adjusted for age, sex and school education to investigate the association of CM types with AD. RESULTS: Among all individuals with AD, 20.6% reported to have experienced at least one type of moderate or severe CM. Emotional and physical neglect were the most frequently reported CM types. Overall, the prevalence of CM types among individuals with AD did not differ from those among individuals without AD. We found no association of CM type with AD. CONCLUSIONS: This is the first study investigating the association of CM with AD in adults. CM was common in the present general population sample, emphasizing that CM is an important public health problem. Our findings suggest that CM is not a risk factor for AD. It might be hypothesized that AD severity is a crucial outcome, and that CM history is a factor with impact on disease severity and course rather than a risk factor for the development of AD. Longitudinal studies are required to address this question.

Lifetime prevalence and determinants of hand eczema in an adolescent population in Germany: 15-year follow-up of the LISA cohort study.

BACKGROUND: Hand eczema is a common inflammatory skin disorder in both adolescence and adulthood. OBJECTIVES: We sought to assess the lifetime prevalence of hand eczema and associated exogenous and endogenous risk factors among adolescents in Germany. METHODS: This was a cross-sectional study embedded into a prospective population-based birth cohort in four regions of Germany, which recruited healthy neonates born between November 1997 and January 1999. We included 1736 participants who had completed the 15-year follow-up from birth cohort and 84.6% (1468/1736) had clearly reported whether they have ever had hand eczema. All the data were based on questionnaires and blood tests (immunoglobulin E). Multivariable logistic regression analysis was used to examine endogenous and exogenous factors in relation to the lifetime prevalence of hand eczema among adolescents. RESULTS: One thousand four hundred and sixty-eight adolescents (715 girls, 48.7%) were included in the final analysis. The lifetime prevalence of hand eczema among adolescents at the age of 15 was 10.4% (95% confidence interval [CI]: 8.9%-12.1%), with a significantly higher lifetime prevalence among girls than boys (12.7% vs. 8.2%, P = 0.005). Multivariable logistic regression analysis indicated statistically significant associations between the lifetime prevalence of hand eczema and having ever been diagnosed with atopic dermatitis (aOR = 1.8, 95% CI: 1.1-2.8) or having ever had dry skin (aOR = 1.9, 95% CI: 1.1-3.1), respectively. No statistically significant independent associations were found between asthma, hay fever, allergy-related clinical symptoms, immunoglobulin E positivity and other exogenous factors in relation to hand eczema. CONCLUSION: Our study fills a research gap on the epidemiological burden of hand eczema among adolescents. One out of ten ever suffered from hand eczema until age 15 years indicating that hand eczema constitutes a significant burden in paediatric populations. The role of atopic dermatitis in hand eczema reinforces previous findings. Exogenous risk factors warrant further investigation.

Refinement and validation of the ItchyQoL using classical test theory and item response theory resulted in a reduction of the response categories from a 5-point to a 3-point scale.

BACKGROUND: The ItchyQoL is an itch-specific patient-reported outcome measure used to assess quality of life in patients with chronic pruritus (CP). OBJECTIVES: We aimed to assess and extend the psychometric properties of the ItchyQoL using classical test theory (CTT) and item response theory (IRT). METHODS: Item characteristic curves were analysed to investigate whether the response categories were functioning optimally. Confirmatory factor analyses were carried out on the ItchyQoL prior to and after rescoring of the response categories. We conducted a Rasch analysis for the ItchyQoL with revised response options and assessed the mean fit residuals in addition to the assumptions of unidimensionality and local independence. RESULTS: In total, 551 patients with CP from nine European countries completed the 22 items of the ItchyQoL. IRT analysis supported the revision of response options from five points to three. This revision was supported by excellent structural validity using CTT. The overall fit to the Rasch model was adequate. Unidimensionality was supported by the ItchyQoL overall scale and by the single subscales; however, local independence was violated in eight cases. CONCLUSIONS: We suggest a revision of the response categories of the ItchyQoL from a 5-point to a 3-point scale. When this revision was applied, the ItchyQoL showed excellent structural validity according to CTT and IRT/Rasch. The calculation of an overall ItchyQoL sum score is allowed.

Piloting an ICU follow-up clinic to improve health-related quality of life in ICU survivors after a prolonged intensive care stay (PINA): study protocol for a pilot randomised controlled trial.

BACKGROUND: Intensive care unit (ICU) survivors often suffer from cognitive, physical and mental impairments, known as post-intensive care syndrome (PICS). ICU follow-up clinics may improve aftercare of these patients. There is a lack of evidence whether or which concept of an ICU follow-up clinic is effective. Within the PINA study, a concept for an ICU follow-up clinic was developed and will be tested in a pilot randomised controlled trial (RCT), primarily to evaluate the feasibility and additionally the potential efficacy. METHODS/DESIGN: Design: Pilot RCT with intervention and control (usual care) arms plus mixed-methods process evaluation. PARTICIPANTS: 100 ICU patients (50 per arm) of three ICUs in a university hospital (Regensburg, Germany), ≥ 18 years with an ICU stay of > 5 days, a sequential organ failure assessment (SOFA) score > 5 during the ICU stay and a life expectancy of more than 6 months. INTERVENTION: The intervention will contain three components: information, consultation and networking. Information will be available in form of an intensive care guide for patients and next of kin at the ICU and phone support during follow-up. For consultation, patients will visit the ICU follow-up clinic at least once during the first 6 months after discharge from ICU. During these visits, patients will be screened for symptoms of PICS and, if required, referred to specialists for further treatment. The networking part (e.g. special referral letter from the ICU follow-up clinic) aims to provide a network of outpatient care providers for former ICU patients. Feasibility Outcomes: Qualitative and quantitative evaluation will be used to explore reasons for non-participation and the intervention´s acceptability to patients and caregivers. Efficacy Outcomes: Health-related quality of life (HRQOL) will be assessed as primary outcome by the physical component score (PCS) of the Short-Form 12 Questionnaire (SF-12). Secondary outcomes encompass further patient-reported outcomes. All outcomes are assessed at 6 months after discharge from ICU. DISCUSSION: The PINA study will determine feasibility and potential efficacy of a complex intervention in a pilot RCT to enhance follow-up care of ICU survivors. The pilot study is an important step for further studies in the field of ICU aftercare and especially for the implementation of a pragmatic multi-centre RCT. TRIAL REGISTRATION: ClinicalTrials.gov , NCT04186468 . Submitted 2 December 2019.

Recommended core outcome instruments for health-related quality of life, long-term control and itch intensity in atopic eczema trials: results of the HOME VII consensus meeting.

BACKGROUND: The Harmonising Outcome Measures for Eczema (HOME) initiative has established a core outcome set of domains for atopic eczema (AE) clinical trials. Previous consensus meetings have agreed on preferred instruments for clinician-reported signs (Eczema Area and Severity Index, EASI) and patient-reported symptoms (Patient-Oriented Eczema Measure, POEM). This paper reports consensus decisions from the HOME VII meeting. OBJECTIVES: To complete the core outcome set for AE by agreeing on core outcome instruments for the domains of quality of life (QoL), long-term control and itch intensity. METHODS: A face-to-face consensus meeting was held in Tokyo, Japan (8-10 April 2019) including 75 participants (49 healthcare professionals/methodologists, 14 patients, 12 industry representatives) from 16 countries. Consensus decisions were made by presentations of evidence, followed by whole and small group discussions and anonymous voting using predefined consensus rules. RESULTS: It was agreed by consensus that QoL should be measured using the Dermatology Life Quality Index (DLQI) for adults, the Children's Dermatology Life Quality Index (CDLQI) for children and the Infant's Dermatology Quality of Life Index (IDQoL) for infants. For long-term control, the Recap of Atopic Eczema (RECAP) instrument or the Atopic Dermatitis Control Test (ADCT) should be used. Consensus was not reached over the frequency of data collection for long-term control. The peak itch numerical rating scale (NRS)-11 past 24 h was recommended as an additional instrument for the symptom domain in trials of older children and adults. Agreement was reached that all core outcome instruments should be captured at baseline and at the time of primary outcome assessment as a minimum. CONCLUSIONS: For now, the core outcome set for clinical trials in AE is complete. The specified domains and instruments should be used in all new clinical trials and systematic reviews of eczema treatments.

Hyperhidrosis Quality of Life Index (HidroQoL©): further validation and clinical application in patients with axillary hyperhidrosis using data from a phase III randomized controlled trial.

BACKGROUND: The Hyperhidrosis Quality of Life Index (HidroQoL©) is a validated patient-reported outcome measure capturing the quality of life of people affected by hyperhidrosis. OBJECTIVES: We aimed to extend the validity evidence to physician-confirmed diagnosis of primary axillary hyperhidrosis. METHODS: Data from a phase III randomized placebo-controlled clinical trial were used (n = 171). Confirmatory factor analysis was carried out to confirm the a priori two-factor structure of the HidroQoL. Internal consistency was assessed using Cronbach's α. Intraclass correlation coefficients (ICCs) were calculated to evaluate test-retest reliability after days -7 to -4. Convergent validity was assessed using correlations with the Dermatology Life Quality Index (DLQI), the Hyperhidrosis Disease Severity Scale (HDSS) and gravimetric sweat production. Known groups were analysed to evaluate discriminative validity. Responsiveness after 29 days was assessed and minimal important difference (MID) values were calculated using both anchor- and distribution-based approaches. All analyses were carried out for total HidroQoL and its two domains. RESULTS: The two-factor structure of the HidroQoL was confirmed. Internal consistency and test-retest reliability were strong (Cronbach's α 0·81-0·90; ICCs 0·89-0·93). Correlations with other outcome measures were in line with a priori hypotheses. The HidroQoL discriminated between different severity groups (P ≤ 0·001) and showed sensitivity to change towards improvement (P < 0·001). An MID value of 4 is proposed for the total scale. CONCLUSIONS: This study supports excellent measurement properties including clinical applicability of the HidroQoL in primary axillary hyperhidrosis and suggests a MID of 4 be applied to clinical trial data.

Outcome assessment in dermatology clinical trials and cochrane reviews: call for a dermatology-specific outcome taxonomy.

BACKGROUND: Standardized outcome reporting is crucial for trial evidence synthesis and translation of findings into clinical decision-making. The OMERACT 2.0 Filter and COMET outcome domain taxonomy propose frameworks for consistent reporting of outcomes. There is an absence of a uniform dermatology-specific reporting strategy that uses precise and consistent outcome definitions. OBJECTIVES: Our aim was to map efficacy/effectiveness outcomes assessed in dermatological trials to the OMERACT 2.0 Filter as a starting point for developing an outcome taxonomy in dermatology. METHODS: We critically appraised 10 Cochrane Skin Reviews randomly selected from all 69 Cochrane Skin Reviews published until 01/2015 and the 220 trials included covering a broad spectrum of dermatological conditions and interventions. Efficacy/effectiveness outcomes were mapped to core areas and domains according to the OMERACT 2.0 Filter. The extracted trial outcomes were used for critical appraisal of outcome reporting in dermatology trials and for the preliminary development of a dermatology-specific outcome taxonomy. RESULTS: The allocation of 1086 extracted efficacy/effectiveness outcomes to the OMERACT 2.0 Filter resulted in a hierarchically structured dermatology-specific outcome classification. In 506 outcomes (47%), the outcome concept to be measured was insufficiently described, hindering meaningful evidence synthesis. Although the core areas assessed in different dermatology trials of the same condition overlap considerably, quantitative evidence synthesis usually failed due to imprecise outcome definitions, non-comparable outcome measurement instruments, metrics and reporting. CONCLUSIONS: We present an efficacy/effectiveness outcome classification as a starting point for a dermatology-specific taxonomy to provide trialists and reviewers with the opportunity to better synthesize and compare evidence.

IDQoL, CDLQI and the 45-item CADIS received a sufficient content validity rating during the HOME VII meeting in Japan: a group discussion study.

BACKGROUND: The Harmonising Outcome Measures for Eczema (HOME) initiative has agreed that quality of life should be measured in all atopic eczema clinical trials. Various candidate instruments exist for this domain but their content validity in atopic eczema is largely unclear. OBJECTIVE: To assess the content validity of quality-of-life candidate instruments for atopic eczema in infants, children and adults in order to aid the decision on what instrument to include in the core outcome set for the quality-of-life domain. METHODS: Six group discussions were conducted at the HOME VII Meeting in Tokyo. Each group was composed of 8-12 patients or parents of patients, clinicians, methodologists and pharmaceutical industry delegates and discussed one or two candidate instruments. The COSMIN criteria on relevance, comprehensiveness and comprehensibility were used to determine the overall content validity rating per instrument. RESULTS: Content validity of the Infant's Dermatitis Quality of Life Index, Children's Dermatology Life Quality Index and the Childhood Atopic Dermatitis Impact Scale (CADIS) long-form was rated as sufficient (+). Results for the CADIS short-form, DLQI and Skindex were inconsistent (±). DISABKIDS, Infants and Toddlers Dermatology Quality of Life and ABS-A were classified as having insufficient content validity. CONCLUSIONS: The content validity rating allowed for a comparison of all candidate instruments and informed the consensus-seeking process regarding the core instrument for the quality-of-life domain.

Recommended core outcome instruments for health-related quality of life, long-term control and itch intensity in atopic eczema trials: results of the HOME VII consensus meeting.

BACKGROUND: The Harmonising Outcome Measures for Eczema (HOME) initiative has established a core outcome set of domains for atopic eczema clinical trials. Previous consensus meetings have agreed upon preferred instruments for clinician-reported signs (Eczema Area and Severity Index - EASI) and patient-reported symptoms (Patient-Oriented Eczema Measure - POEM). This paper reports consensus decisions from the HOME VII meeting. OBJECTIVE: To complete the core outcome set for atopic eczema by agreeing upon core outcome instruments for the domains of quality of life, long-term control and itch intensity. METHODS: Face-to-face consensus meeting held in Tokyo, Japan (8th to 10th April, 2019) including 74 participants (47 healthcare professionals/methodologists, 14 patients, 13 industry representatives), from 16 countries. Consensus decisions were made by presentations of evidence, followed by whole and small group discussions and anonymous voting using pre-defined consensus rules. RESULTS: It was agreed by consensus that quality of life should be measured using the Dermatology Life Quality Index (DLQI) for adults, the Children's Dermatology Life Quality Index (CDLQI) for children, and the Infant's Dermatology Quality of Life Index (IDQoL) for infants. For long-term control, the Recap of Atopic Eczema (RECAP) instrument or the Atopic Dermatitis Control Test (ADCT) should be used. Consensus was not reached over the frequency of data collection for long-term control. The peak itch numerical rating scale(NRS)-11 past 24 hours was recommended as an additional instrument for the symptom domain in trials of older children and adults. Agreement was reached that all core outcome instruments should be captured at baseline and at the time of primary outcome assessment as a minimum. CONCLUSIONS: For now, the core outcome set for clinical trials in atopic eczema is complete. The specified domains and instruments should be used in all new clinical trials and systematic reviews of eczema treatments.

Protocol for the development of a core domain set for hand eczema trials.

BACKGROUND: Clinical hand eczema trials measure a variety of outcome domains to determine the success of interventions. This considerably limits the comparability and overall confidence in the study results, and thereby the strength of recommendations for clinical practice. OBJECTIVES: The Hand Eczema Core Outcome Set (HECOS) initiative aims to develop a core outcome set (COS) for the standardized evaluation of interventions in future hand eczema trials and reviews. This COS will define the minimum that should be measured and reported in controlled and randomized-controlled trials of therapeutic hand eczema interventions. The objective of this protocol is to specify the methods to develop a core domain set. METHODS: In Phase 1, a list of candidate domains will be derived from a systematic literature review concerning previously measured outcomes in hand eczema trials, from qualitative patient interviews and from expert interviews. In Phase 2, a consensus study about core domains will be conducted by an online 3-round Delphi survey and a face-to-face meeting, applying predefined consensus criteria. HECOS involves hand eczema and methods experts as well as patients and further stakeholders with an interest in the initiative. OUTLOOK: When a set of core domains has been defined, HECOS is going to identify appropriate outcome measurement instruments in a development process that will be detailed in another protocol. The COS will considerably enhance the methodological quality, comparability and usefulness of hand eczema trials for clinical decision-making and the development of new therapeutic options for hand eczema, and also reduce the effort of planning, conducting, and reporting individual hand eczema studies, reviews and meta-analyses.

Development of a validated short-form of the Childhood Atopic Dermatitis Impact Scale, the CADIS-SF15.

BACKGROUND: The Childhood Atopic Dermatitis Impact Scale (CADIS) with 45 items may be burdensome to complete. We therefore aimed to develop a CADIS short-form. METHODS: Parents of 300 children completed the prototype CADIS. Exploratory factor analysis was conducted on the 45-item CADIS version. The most representative items were chosen. Confirmatory factor analysis was used to confirm the a priori factor structure. Content validity was assessed in a focus group of patients, parents, clinicians, methodologists and industry delegates. Internal consistency, 48-h test-retest reliability, construct validity and responsiveness of the newly developed short-form were assessed. RESULTS: A total of 270 families provided data at baseline, 34 after 48 h and 228 after 4 weeks. Fourteen items of three different factors fulfilled the proposed eligibility criteria and were included in the draft short-form. After the content validity rating, one item relating to the child's sleep was added to further improve content validity. The confirmatory factor analyses showed good model fit, and a 15-item short-form was initiated, the CADIS-SF15. The total scale and the three domains showed good internal consistency and test-retest reliability. The correlation between SCORAD and other subjective measures was consistent with our hypotheses. Differences in scores between mild, moderate and severe AD patients were significant, and the CADIS-SF15 was able to detect changes in 'improving' patients over time. CONCLUSION: The CADIS-SF15 with 15 items in three domains is an internally consistent, reliable, valid, responsive and brief measure of QoL in children affected with AD and their parents. Further evaluation of clinical applicability is required.

Evaluation of responsiveness and estimation of smallest detectable change and minimal important change scores for the Childhood Atopic Dermatitis Impact Scale.

BACKGROUND: The Childhood Atopic Dermatitis Impact Scale (CADIS) is an instrument to measure quality of life in young children affected by atopic dermatitis, and their parents. OBJECTIVES: To evaluate the responsiveness (sensitivity to change), smallest detectable change (SDC) and minimal important change (MIC) of the CADIS. METHODS: Parents and primary caregivers of 300 young children completed the CADIS and a global rating of their child's skin condition at baseline and a 4-week follow-up. Kruskal-Wallis tests, Wilcoxon tests and effect sizes were used to assess responsiveness. The SDC can be seen as a change beyond measurement error. Anchor-based and distribution-based methods, and an integration of both methods were used to estimate the MIC. RESULTS: In total, 270 families provided data at baseline and 228 at follow-up. The CADIS total change score and most of the domain scores had moderate-to-strong correlations with the skin change score. Patients were grouped according to the skin change score, which served as an anchor. Children whose parents noted an improvement of the skin showed lower CADIS scores at follow-up (P < 0·001). For the SDC we obtained score changes of 1·34 points on the total score and < 1·0 points on each domain score. All detected MIC values passed the SDC cut-off. CONCLUSIONS: The CADIS is sensitive to change towards improvement of quality of life. A change > 12% on the total score or each domain score very likely represents a clinically important change. What's already known about this topic? Atopic dermatitis reduces the quality of life of affected children and their parents. The Childhood Atopic Dermatitis Impact Scale (CADIS) has been evaluated and translated into two further languages. What does this study add? Further validation of the responsiveness of the CADIS, and whether it is sensitive to change in patients whose condition had changed. Calculation of the smallest detectable change. What are the clinical implications of this work? Estimation of the minimal important change in CADIS provides benchmarks for clinical practice.

TREatment of ATopic eczema (TREAT) Registry Taskforce: protocol for a European safety study of dupilumab and other systemic therapies in patients with atopic eczema.

BACKGROUND: A long-term prospective observational safety study is essential to characterize fully the safety profile of systemic immunomodulating therapies for patients with atopic eczema. The TREatment of ATopic eczema (TREAT) Registry Taskforce offers a large platform to conduct such research using national registries that collect the same data using a predefined core dataset. OBJECTIVES: To present a protocol for a safety study comparing dupilumab with other systemic immunomodulating therapies in children and adults with moderate-to-severe atopic eczema, to assess the long-term safety risk of these therapies in a routine clinical care setting. METHODS: We describe a registry-embedded international observational prospective cohort study. Adult and paediatric patients who start treatment with dupilumab or another systemic immunomodulating agent for their atopic eczema will be included. The primary end point is the incidence of malignancies (excluding nonmelanoma skin cancer) compared between the treatment groups. Secondary end points include other serious adverse events and adverse events of special interest, such as eye disorders and eosinophilia. CONCLUSIONS: This protocol delineates a safety study for dupilumab in adult and paediatric patients with atopic eczema, using a standardized methodological approach across several national registries. The protocol could also be used for other novel systemic immunomodulating therapies, and could provide licensing and reimbursement authorities, pharmaceutical companies and clinicians with safety evidence from a routine clinical care setting. What's already known about this topic? There is a need for long-term data on the safety of systemic immunomodulating therapies in patients with atopic eczema. Regulatory bodies, such as the European Medicines Agency, increasingly stipulate the collection of such data as part of the licensing agreement for new treatments, to assess the new agent's long-term safety profile against established therapies. Large numbers of patients with a long duration of follow-up are necessary in order to detect rare events like malignancies. What does this study add? The TREAT Registry Taskforce offers a platform to conduct such research with a network of multiple national atopic eczema research registries. We present a protocol for an investigator-initiated multicentre safety study comparing dupilumab with other systemic immunomodulating therapies in adults and subsequently adolescents and children with moderate-to-severe atopic eczema. This protocol can be used as a framework for similar studies for other novel systemic immunomodulating therapies across both adult and paediatric populations.