Longitudinal validity of the Hyperhidrosis Quality of Life Index (HidroQoL©) in a phase IIIb clinical trial population with hyperhidrosis: responsiveness and meaningful change.

BACKGROUND: The Hyperhidrosis Quality of Life Index (HidroQoL ©) is a well-developed patient-reported outcome measure assessing the quality of life (QoL) impacts in hyperhidrosis, which has proven very good measurement properties, such as structural validity and internal consistency. OBJECTIVES: We aimed to investigate responsiveness over time and estimate values for meaningful within-person change (MWPC) towards symptom improvement for different measurement time points (4 and 12 weeks), extending the existing validity evidence in patients with primary axillary hyperhidrosis. METHODS: Data (from a phase IIIb clinical trial) was collected at baseline, week 4, week 8, week 12, week 28, week 52, and week 72. For the assessment of responsiveness, HidroQoL change scores were correlated with corresponding change scores of the Hyperhidrosis Disease Severity Scale (HDSS), the Dermatology Life Quality Index (DLQI), and the gravimetric sweat production based on a-priori formulated hypotheses. Furthermore, it was tested whether the different HDSS change score groups differed significantly from each other over time and whether the HidroQoL was sensitive towards these group differences over time. This was extended by the calculation of matched-pair tests and effect sizes to test significance for each change group separately. For the estimation of MWPC thresholds towards symptom improvement, different anchor-based and integrated approaches were used. RESULTS: In total, the sample was composed of 357 patients with primary axillary hyperhidrosis. For the assessment of responsiveness, 5 out of 14 a-priori hypotheses regarding the correlation of the change scores could be confirmed, whereas the rejected hypotheses only marginally differed from the expected values. Furthermore, regarding responsiveness, the HidroQoL showed sensitivity towards symptom improvement at each measurement time point. Effect sizes were large as expected (d ≥ 0.806). MWPC thresholds towards symptom improvement were proposed for two measurement time points: 5 (week 4) and 6 (week 12). Increasing MWPC values over time were observed. CONCLUSION: This study extends the evidence for the longitudinal validity of the HidroQoL up to 72 weeks and proposed MWPC thresholds for different time intervals (4 and 12 weeks) after baseline, aiding interpretability. Results concur with findings from previous validation studies.

What do Hyperhidrosis Quality of Life Index (HidroQoL©) scores mean? Transferring science into practice by establishing a score banding system.

BACKGROUND: The Hyperhidrosis Quality of Life Index (HidroQoL©) is a measure of quality of life (QoL) impacts in hyperhidrosis (HH). OBJECTIVES: We aimed to establish score banding systems for the HidroQoL total score for specific contexts representing different severity/impact categories by using the Dermatology Life Quality Index (DLQI) and the Hyperhidrosis Disease Severity Scale (HDSS) as anchors, including data from 357 patients from a phase III clinical trial. METHODS: We used the HDSS, the established DLQI score bands and two single items (items 5 and 7) of the DLQI as anchors for the creation of banding systems for the HidroQoL. These anchors were chosen via consensus among an expert group according to relevance to patient experience. Due to the distribution of the HDSS and the single DLQI item 7, receiver operating characteristic curves were computed in order to create an optimal cut-off value of the HidroQoL total score. For the DLQI banding system and the single DLQI item 5, we created a banding system for the HidroQoL based on the distribution of their different categories. RESULTS: A score of 30 and greater is proposed as the cut-off value for sweating that 'always interferes in daily activities', based on the HDSS as anchor. In terms of overall skin QoL effects, score bands of 0-6, 7-18, 19-25, 26-32 and 33-36 represent 'no effect', 'small effect', 'moderate effect', 'very large effect' and 'extremely large effect' on the patient's life, respectively. CONCLUSIONS: In this study, we propose different banding systems for four different contexts: skin-specific QoL (DLQI banding), HH severity (HDSS), working and studying (single DLQI item 7) and social and leisure activities (single DLQI item 5). These banding systems and cut-off values can be used in clinical research and practice to place the patients in different severity categories.

Patient-reported outcome measures for acute rhinosinusitis in adults and children: a systematic review of the quality of existing instruments.

BACKGROUND: Acute rhinosinusitis (ARS) is a self-limiting inflammation of the nose and sinuses caused by viral or bacterial infections that requires primarily symptomatic treatment. Patient-reported outcome measures (PROMs) are suitable tools for the assessment of the effectiveness of remedies for ARS from the patient's perspective in clinical trials and real-world studies. Data regarding the quality of existing PROMs for ARS are limited. PURPOSE: To conduct a systematic review of the quality of existing disease-specific PROMs for use in adults and children with ARS according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology, and to derive recommendations for use of the identified instruments in future clinical studies. METHODS: We systematically searched PubMed, Web of Science and Embase for studies reporting on the development and/or validation of PROMs for ARS. We assessed the methodological quality of each included study, evaluated the quality of measurement properties per PROM and study, and graded the evidence. Based on the overall evidence, we derived recommendations for use of the instruments. RESULTS: We identified four studies on three PROMs measuring symptoms of ARS and quality of life in adults (Sinonasal Outcome Test-16, SNOT-16; Measurement of Acute Rhinosinusitis, MARS; Rhinosinusitis Quality-of-Life Assessment, RhinoQoL). For ARS in children, we identified two studies on two PROMs measuring symptoms of ARS (Pediatric Rhinosinusitis Symptom Scale, PRSS; Sinus Symptom Questionnaire, S5). Our assessment of measurement properties indicates that all instruments require further validation before they can be unrestrictedly recommended for use in future research (COSMIN category B). In particular, there were no content validity studies for any of the identified instruments, but also data on other important measurement properties, e.g., structural validity, are lacking. CONCLUSION: Currently, no PROM for ARS in adults and children can be unrestrictedly recommended based on the evaluation of their quality. Further validation is required for all identified PROMs. Content validation involving patients and experts should be given priority. SYSTEMATIC REVIEW REGISTRATION: OSF ( https://doi.org/10.17605/OSF.IO/VAP8U ).

Measurement properties of quality-of-life outcome measures for children and adults with eczema: A systematic review update 2.0.

A systematic review on measurement properties of patient-reported outcome measures (PROMs) for the assessment of quality of life (QoL) in children and adults with eczema was published in 2016 and updated in 2019. We now aimed to systematically assess the measurement properties of PROMs based on recently published development and validation studies regarding quality-of-life skin- or disease-specific PROMs using the COnsensus-based Standards for the selection of health Measurement Instruments (COSMIN) guidelines and integrate these findings with those from the 2019 update. A systematic literature search was conducted in PubMed and Embase. Eligible studies reported on measurement properties of QoL skin- or disease-specific PROMs for the assessment of QoL in children and adults with eczema. The methodological quality of the included studies was evaluated using the COSMIN Risk of Bias checklist. The methodological quality of the included PROMs was judged using updated criteria for good measurement properties, and the quality of evidence was graded. The new evidence was integrated into the results from the 2019 update. Finally, a recommendation for use of the identified PROMs was derived based on all evidence. We identified 12 studies fulfilling our inclusion criteria. Based on the newly generated evidence, the Childhood Atopic Dermatitis Impact Scale-short form (CADIS-SF) for infants and the Skindex for adults can be recommended for use. We identified three new PROMs, which have the potential to be used, but require further validation: the Pediatric Allergic Disease Quality of Life Questionnaire (PADQLQ), the Skindex-Mini, and the Scratch Intensity and Impact Scale (SIIS). All other assessed PROMs still have the opportunity to be recommended for use (except for the Dermatology Life Quality Index (DLQI)) but need further validation. Currently, two PROMs for the assessment of QoL in eczema can be recommended for use: the CADIS-SF for infants and the Skindex for adults. Many PROMs, including the recently developed and/or validated PROMs PADQLQ, Skindex-Mini and SIIS, need further validation.

Patient-reported outcome measures for uncomplicated urinary tract infections in women: a systematic review.

PURPOSE: To conduct a systematic review of the quality of existing patient-reported outcome measures (PROMs) for use in women with uncomplicated urinary tract infections (UTIs) applying the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology, and to derive recommendations for their use in future research. METHODS: A systematic literature search was performed in PubMed and Web of Science. Studies reporting on the development and/or validation of any PROMs for uncomplicated UTIs in women were considered eligible. We evaluated the methodological quality of each included study using the COSMIN Risk of Bias Checklist, and further applied predefined criteria for good measurement properties. Finally, we graded the evidence and derived recommendations for the use of the included PROMs. RESULTS: Data from 23 studies reporting on six PROMs were included. From those, the Acute Cystitis Symptom Score (ACSS) and the Urinary Tract Infection-Symptom and Impairment Questionnaire (UTI-SIQ-8) can be recommended for further use. Both instruments showed sufficient content validity. We further found high-quality evidence for sufficient internal consistency of the UTI-SIQ-8, while this criterion was not assessed for the ACSS due to a formative measurement model. All other PROMs have the potential to be recommended for use, but require further validation. CONCLUSION: The ACSS and the UTI-SIQ-8 have the potential to be recommended for use in women with uncomplicated UTIs in future clinical trials. For all included PROMs, further validation studies are indicated. SYSTEMATIC REVIEW REGISTRATION: PROSPERO.

Content validity of the Recap of atopic eczema (RECAP) instrument in Dutch, English and German to measure eczema control in young people with atopic eczema: a cognitive interview study.

BACKGROUND: Recap of atopic eczema (RECAP) is a patient-reported outcome measure assessing eczema control. This instrument has been developed and validated in the UK. There are self-reported and proxy-reported versions in English, Dutch and German. However, it is unclear whether the self-reported version shows adequate content validity when completed by young people (8-16 years) in these languages. OBJECTIVES: To assess the content validity (comprehensibility, relevance and comprehensiveness) of the English, German and Dutch versions of the self-reported RECAP in young people with atopic eczema and to identify the most appropriate age cutoff for self-completion. METHODS: We conducted 23 semistructured cognitive interviews with young people aged 8-16 years, using the 'think-aloud' method. In Germany and the Netherlands, participants were recruited in dermatology clinics and in the UK through social media and existing mailing lists. Interviews were audio recorded, transcribed verbatim and analysed in the three languages, using a problem-focused coding manual. Transcripts were coded by two independent reviewers in each country. Themes were translated into English and compared across the three countries. RESULTS: Significant age-related comprehensibility issues with the last three items of the questionnaire occurred with young people aged 8-11 years, causing difficulties completing RECAP without help. However, older children had only minor problems and were able to complete the questionnaire by themselves. The self-reported version of RECAP has sufficient content validity for self-completion in young people aged 12 years and above. However, the German version with some translational adaptations may be appropriate for children from the age of 8 years. There may be some situations where the proxy version is needed for older children too. CONCLUSIONS: The self-reported version of RECAP is appropriate for use from the age of 12 years. The proxy version can be used in children younger than 12 years. Other measurement properties should be further investigated. What is already known about this topic? Recap of atopic eczema (RECAP) is an instrument recommended by the Harmonising Outcome Measures for Eczema initiative for the core outcome domain of long-term control of atopic eczema. Content validity of RECAP for self-completion by adults and of the proxy version has been assessed. What does this study add? In this study, content validity (comprehensibility, relevance and comprehensiveness) of the self-reported version of RECAP among young people (aged 8-16 years) with atopic eczema across the UK, Germany and the Netherlands is assessed. Based on these findings, key recommendations on how to measure eczema control in young people with atopic eczema are formulated. What are the clinical implications of this work? The Dutch, English and German self-completion versions of RECAP are recommended for use in adolescents from the age of 12 years. The proxy version could be used in children younger than 12 years or where children are cognitively or physically incapable of reporting their experience of eczema control. Caregivers should be encouraged to complete RECAP together with their child where possible.

Recommended measurement instruments for genitourinary symptoms associated with menopause: the COMMA (Core outcomes in menopause) consortium.

OBJECTIVE: The aim of the study is to identify appropriate definitions and patient-reported outcome measures (PROMs) for each of the eight core outcomes previously selected for genitourinary symptoms associated with menopause: pain with sex, vulvovaginal dryness, vulvovaginal discomfort or irritation, discomfort or pain when urinating, change in most bothersome symptom, distress, bother or interference of genitourinary symptoms, satisfaction with treatment, and side effects. METHODS: We conducted a systematic review to identify possible definitions and PROMs, including their measurement properties. Identified definitions and relevant PROMs with acceptable measurement properties were entered into an international consensus process involving 28 participants from 10 countries to achieve final recommendations for each core outcome. RESULTS: A total of 87 publications reporting on 34 PROMs were identified from 21,207 publications screened. Of these 34 PROMs, 29 were not considered to sufficiently map onto the core outcomes, and 26 of these also had insufficient measurement properties. Therefore, only five PROMs corresponding to two core outcomes were considered for recommendation. We recommend the PROMIS Scale v2.0 - Sexual Function and Satisfaction: Vaginal Discomfort with Sexual Activity to measure the outcome of "pain with sexual activity" and the Day-to-Day Impact of Vaginal Aging (DIVA) Questionnaire to measure "distress, bother or interference" from genitourinary symptoms. Six definitions of "side effects" were identified and considered. We recommend that all trials report adverse events in study participants, which is a requirement of Good Clinical Practice. CONCLUSIONS: Suitable PROMs and definitions were identified to measure three of eight core outcomes. Because of the lack of existing measures, which align with the core outcomes and have evidence of high-quality measurement properties, future work will focus on developing or validating PROMs for the remaining five core outcomes.

Recommended measurement instruments for menopausal vasomotor symptoms: the COMMA (Core Outcomes in Menopause) consortium.

OBJECTIVE: The aim of the study is to identify suitable definitions and patient-reported outcome measures (PROMs) to assess each of the six core outcomes previously identified through the COMMA (Core Outcomes in Menopause) global consensus process relating to vasomotor symptoms: frequency, severity, distress/bother/interference, impact on sleep, satisfaction with treatment, and side effects. METHODS: A systematic review was conducted to identify relevant definitions for the outcome of side-effects and PROMs with acceptable measurement properties for the remaining five core outcomes. The consensus process, involving 36 participants from 16 countries, was conducted to review definitions and PROMs and make final recommendations for the measurement of each core outcome. RESULTS: A total of 21,207 publications were screened from which 119 reporting on 40 PROMs were identified. Of these 40 PROMs, 36 either did not adequately map onto the core outcomes or lacked sufficient measurement properties. Therefore, only four PROMs corresponding to two of the six core outcomes were considered for recommendation. We recommend the Hot Flash Related Daily Interference Scale to measure the domain of distress, bother, or interference of vasomotor symptoms and to capture impact on sleep (one item in the Hot Flash Related Daily Interference Scale captures interference with sleep). Six definitions of "side effects" were identified and considered. We recommend that all trials report adverse events, which is a requirement of Good Clinical Practice. CONCLUSIONS: We identified suitable definitions and PROMs for only three of the six core outcomes. No suitable PROMs were found for the remaining three outcomes (frequency and severity of vasomotor symptoms and satisfaction with treatment). Future studies should develop and validate PROMs for these outcomes.

Psychometric evaluation of the German Day-to-Day Impact of Vaginal Aging questionnaire using data from two intervention studies.

OBJECTIVE: We aimed to further validate the German version of the Day-to-Day Impact of Vaginal Aging (DIVA) questionnaire. METHODS: Data were collected in the context of two intervention studies. Forty-two women participated in clinical trial VMP-03/2018 (NCT04222647) and 79 women in clinical trial VFCrC-01/2021 (NCT05211505). Internal consistency was calculated using Cronbach α . Correlations with other outcome measures such as a subjective assessment of symptoms and dyspareunia, the Vaginal Health Index, and the Vaginal Maturation Index were calculated regarding construct validity. A priori hypotheses were formulated for construct validity. Responsiveness was assessed after 43 (±3) and after 38 (±1) days in the two clinical trials. RESULTS: Strong internal consistency in all of the DIVA domains was found ( α ≥ 0.80). Regarding construct validity (at baseline and over time), many hypotheses were confirmed. Furthermore, all of the DIVA domains were able to detect changes over time ( P ≤ 0.006). Moderate to strong effect sizes were found (≥0.460). The data supported the responsiveness of the DIVA. CONCLUSIONS: Our findings from two independent intervention studies support internal consistency, construct validity, and responsiveness of the German version of the DIVA (domains).

Hyperhidrosis quality of life index (HidroQoL): further validation by applying classical test theory and item response theory using data from a phase III clinical trial.

BACKGROUND: The Hyperhidrosis Quality of Life Index (HidroQoL ©) is a well-developed and validated patient-reported outcome measure assessing the quality-of-life impacts in hyperhidrosis with 18 items. Our aim was to extend the already existing validity evidence for the HidroQoL, especially in relation to structural validity. Especially Rasch analysis has not been applied to the final 18-item HidroQoL before. METHODS: Data from a phase III clinical trial were used. Confirmatory factor analysis was conducted to confirm the two a priori HidroQoL scales within classical test theory. Furthermore, the assumptions of the Rasch model (model fit, monotonicity, unidimensionality, local independence) and Differential Item Functioning (DIF) were assessed using item response theory. RESULTS: The sample included 529 patients with severe primary axillary hyperhidrosis. The two-factor structure could be confirmed by the confirmatory factor analysis (SRMR = 0.058). The item characteristic curves showed mainly optimally functioning response categories, indicating monotonicity. The overall fit to the Rasch model was adequate and unidimensionality for the HidroQoL overall scale could be confirmed, since the first factor had an eigenvalue of 2.244 and accounted for 18.7%. Local independence was below assumed thresholds (residual correlations ≤ 0.26). DIF analysis, controlling for age or gender, was critical for four and three items, respectively. However, this DIF could be explained. CONCLUSION: Using classical test theory and item response theory/Rasch analyses, this study provided further evidence for the structural validity of the HidroQoL. This study confirmed several specific (measurement) properties of the HidroQoL questionnaire in patients with physician-confirmed severe primary axillary hyperhidrosis: the HidroQoL is a unidimensional scale allowing the summation of scores to generate a single score, and simultaneously it has a dual structure, also allowing the calculation of separate domain scores for daily activities and psychosocial impacts. With this study, we provided new evidence of the structural validity of the HidroQoL in the context of a clinical trial. Trial registration The study was registered (ClinicalTrials.gov identifier: NCT03658616, 05 September 2018, https://clinicaltrials.gov/ct2/show/NCT03658616?term=NCT03658616&draw=2&rank=1 ).