Asthma Daytime Symptom Diary (ADSD) and Asthma Nighttime Symptom Diary (ANSD): Measurement Properties of Novel Patient-Reported Symptom Measures

Abstract Objectives Existing measures of inflammatory bowel disease (IBD) symptoms are not well suited to self-report, inadequate in measurement properties, insufficiently specific, or burdensome for brief or repeated administration. We aimed to develop a patient-reported outcome measure to assess a broader range of IBD symptoms. Methods The IBD Symptoms Inventory (IBDSI) was developed by adapting symptom items from existing clinician-rated or diary-format inventories; after factor analysis, 38 items were retained on 5 subscales: bowel symptoms, abdominal discomfort, fatigue, bowel complications, and systemic complications. Participants completed the IBDSI and other self-report measures during a clinic visit. A nurse administered the Harvey Bradshaw Index (HBI) for Crohn’s disease (CD) or the Powell-Tuck Index (PTI) for ulcerative colitis (UC), and a gastroenterologist completed a global assessment of disease severity (PGA). Results The 267 participants with CD (n = 142) or UC (n = 125), ages 18 to 81 (M = 43.4, SD = 14.6) were 58.1% female, with a mean disease duration of 13.9 (SD = 10.5) years. Confirmatory factor analysis supported the 5 subscales. The total scale and subscales showed good reliability and significant correlations with self-report symptom and IBD quality of life measures, the HBI, PTI, and PGA. Conclusions The IBDSI showed strong measurement properties: a supported factor structure, very good internal consistency, convergent validity, and excellent sensitivity and specificity to clinician-rated active disease. Self-report HBI and PTI items, when extracted from this measure, produced scores comparable to clinician-administered versions. The 38-item IBDSI, or 26-item short form, can be used as a brief survey of common IBD symptoms in clinic or research settings.

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Validation of a menstrual pictogram and a daily bleeding diary for assessment of uterine fibroid treatment efficacy in clinical studies

Journal of Patient-Reported Outcomes ◽

10.1186/s41687-020-00263-0 ◽

2020 ◽

Vol 4 (1) ◽

Author(s):

Claudia Haberland ◽

Anna Filonenko ◽

Christian Seitz ◽

Matthias Börner ◽

Christoph Gerlinger ◽

...

Keyword(s):

Uterine Fibroid ◽

Full Range ◽

Intraclass Correlation ◽

Phase Iii ◽

Measurement Properties ◽

Retest Reliability ◽

Response Options ◽

Patient Global Impression ◽

Patient Reported ◽

Test Retest Reliability

Abstract Background To evaluate the psychometric and measurement properties of two patient-reported outcome instruments, the menstrual pictogram superabsorbent polymer-containing version 3 (MP SAP-c v3) and Uterine Fibroid Daily Bleeding Diary (UF-DBD). Test-retest reliability, criterion, construct validity, responsiveness, missingness and comparability of the MP SAP-c v3 and UF-DBD versus the alkaline hematin (AH) method and a patient global impression of severity (PGI-S) were analyzed in post hoc trial analyses. Results Analyses were based on data from up to 756 patients. The full range of MP SAP-c v3 and UF-DBD response options were used, with score distributions reflecting the cyclic character of the disease. Test-retest reliability of MP SAP-c v3 and UF-DBD scores was supported by acceptable intraclass correlation coefficients when stability was defined by the AH method and Patient Global Impression of Severity (PGI-S) scores (0.80–0.96 and 0.42–0.94, respectively). MP SAP-c v3 and UF-DBD scores demonstrated strong and moderate-to-strong correlations with menstrual blood loss assessed by the AH method. Scores increased in monotonic fashion, with greater disease severities, defined by the AH method and PGI-S scores; differences between groups were mostly statistically significant (P < 0.05). MP SAP-c v3 and UF-DBD were sensitive to changes in disease severity, defined by the AH method and PGI-S. MP SAP-c v3 and UF-DBD showed a lower frequency of missing patient data versus the AH method, and good agreement with the AH method. Conclusions This evidence supports the use of the MP SAP-c v3 and UF-DBD to assess clinical efficacy endpoints in UF phase III studies replacing the AH method.

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Apathy Measures in Older Adults and People with Dementia: A Systematic Review of Measurement Properties Using the COSMIN Methodology

Dementia and Geriatric Cognitive Disorders ◽

10.1159/000515678 ◽

2021 ◽

pp. 1-13

Author(s):

Clare Burgon ◽

Sarah Elizabeth Goldberg ◽

Veronika van der Wardt ◽

Catherine Brewin ◽

Rowan H. Harwood

Keyword(s):

Systematic Review ◽

Older Adults ◽

Rating Scale ◽

Measurement Properties ◽

Evaluation Procedure ◽

Measurement Instruments ◽

Health Measurement ◽

Eligibility Criteria ◽

People With Dementia ◽

Patient Reported

Background: Apathy is highly prevalent in dementia and is also seen in mild cognitive impairment and the general population. Apathy contributes to failure to undertake daily activities and can lead to health problems or crises. It is therefore important to assess apathy. However, there is currently no gold standard measure of apathy. A comprehensive systematic review of the measurement properties of apathy scales is required. Methods: A systematic review was registered with PROSPERO (ID: CRD42018094390). MEDLINE, Embase, PsycINFO, and CINAHL were searched for studies that aimed to develop or assess the validity or reliability of an apathy scale in participants over 65 years, living in the community. A systematic review was conducted in line with the COnsensus-based Standards for the selection of health Measurement INstruments procedure for reviewing patient-reported outcome measures. The studies’ risk of bias was assessed, and all relevant measurement properties were assessed for quality. Results were pooled and rated using a modified Grading of Recommendations Assessment, Development, and Evaluation procedure. Results: Fifty-seven publications regarding 18 measures and 39 variations met the eligibility criteria. The methodological quality of individual studies ranged from inadequate to very good and measurement properties ranged from insufficient to sufficient. Similarly, the overall evidence for measurement properties ranged from very low to high quality. The Apathy Evaluation Scale (AES) and Lille Apathy Rating Scale (LARS) had sufficient content validity, reliability, construct validity, and where applicable, structural validity and internal consistency. Conclusion: Numerous scales are available to assess apathy, with varying psychometric properties. The AES and LARS are recommended for measuring apathy in older adults and people living with dementia. The apathy dimension of the commonly used Neuropsychiatric Inventory should be limited to screening for apathy.

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The German RECAP questionnaire: linguistic validation and cognitive debriefing in German adults with self-reported atopic eczema and parents of affected children

Journal of Patient-Reported Outcomes ◽

10.1186/s41687-021-00285-2 ◽

2021 ◽

Vol 5 (1) ◽

Author(s):

Michaela Gabes ◽

Christina Tischer ◽

Anne Herrmann ◽

Laura Howells ◽

Christian Apfelbacher

Keyword(s):

Atopic Eczema ◽

Content Validity ◽

Qualitative Content Analysis ◽

Recall Period ◽

Measurement Properties ◽

German Population ◽

German Version ◽

Health Measurement ◽

Response Options ◽

Patient Reported

Abstract Background Recap of atopic eczema (RECAP) is a patient-reported outcome measure (PROM) assessing eczema control. Long-term control of eczema is one of the four core outcome domains for atopic eczema trials. This instrument has been recently developed in the UK. Objective This study aimed to translate the English RECAP into German and test its content validity in a German population with self-reported atopic eczema. Methods A six-step procedure including two forward and one backward translations, two consensus decisions and an expert review was performed to obtain a German version of RECAP. We conducted semi-standardized cognitive interviews with adults with atopic eczema (n = 7) and parents having children affected by this disease (n = 5). A “think-aloud” method was used and aspects of comprehensibility, comprehensiveness and relevance according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) criteria were examined. Interviews were coded using qualitative content analysis. Results No particular linguistic problems were encountered during forward-backward translation. Minor wording changes were made as required. The title was adjusted to a more familiar German term of the disease (which is ‘Neurodermitis’). The recall period was rephrased from ‘over the last week’ to ‘over the last seven days’ since there was a different cultural understanding of the time frame. Regarding content validity, the items of the German RECAP were considered to be comprehensible, comprehensive and relevant for the participants and parents of affected children. The participants understood the instruction and considered the one-week recall period and the response options as appropriate. Conclusions A German version of RECAP that is linguistically equivalent to the original version is now available but further assessment of its measurement properties is needed.

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Measurement properties of patient-reported outcome measures (PROMs) in hyperhidrosis: a systematic review

Quality of Life Research ◽

10.1007/s11136-021-02958-3 ◽

2021 ◽

Author(s):

Michaela Gabes ◽

Helge Knüttel ◽

Gesina Kann ◽

Christina Tischer ◽

Christian J. Apfelbacher

Keyword(s):

Systematic Review ◽

Clinical Trials ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Measurement Properties ◽

High Quality ◽

High Quality Evidence ◽

Patient Reported ◽

Quality Evidence

Abstract Purpose To critically appraise, compare and summarize the quality of all existing PROMs that have been validated in hyperhidrosis to at least some extend by applying the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) methodology. Thereby, we aim to give a recommendation for the use of PROMs in future clinical trials in hyperhidrosis. Methods We considered studies evaluating, describing or comparing measurement properties of PROMs as eligible. A systematic literature search in three big databases (MEDLINE, EMBASE and Web of Science) was performed. We assessed the methodological quality of each included study using the COSMIN Risk of Bias checklist. Furthermore, we applied predefined quality criteria for good measurement properties and finally, graded the quality of the evidence. Results Twenty-four articles reporting on 13 patient-reported outcome measures were included. Three instruments can be further recommended for use. They showed evidence for sufficient content validity and moderate- to high-quality evidence for sufficient internal consistency. The methodological assessment showed existing evidence gaps for eight other PROMs, which therefore require further validation studies to make an adequate decision on their recommendation. The Hyperhidrosis Disease Severity Measure-Axillary (HDSM-Ax) and the short-form health survey with 36 items (SF-36) were the only questionnaires not recommended for use in patients with hyperhidrosis due to moderate- to high-quality evidence for insufficient measurement properties. Conclusion Three PROMs, the Hyperhidrosis Quality of Life Index (HidroQoL), the Hyperhidrosis Questionnaire (HQ) and the Sweating Cognitions Inventory (SCI), can be recommended for use in future clinical trials in hyperhidrosis. Results obtained with these three instruments can be seen as trustworthy. Nevertheless, further validation of all three PROMs is desirable. Systematic review registration PROSPERO CRD42020170247

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The ENDOPAIN 4D Questionnaire: A New Validated Tool for Assessing Pain in Endometriosis

Journal of Clinical Medicine ◽

10.3390/jcm10153216 ◽

2021 ◽

Vol 10 (15) ◽

pp. 3216

Author(s):

Anne Puchar ◽

Pierre Panel ◽

Anne Oppenheimer ◽

Joseph Du Cheyron ◽

Xavier Fritel ◽

...

Keyword(s):

Minimal Clinically Important Difference ◽

Patient Reported Outcome ◽

Measurement Properties ◽

Dimensional Structure ◽

Clinically Important Difference ◽

Urinary Tract Symptoms ◽

New Instrument ◽

Patient Reported ◽

Good Internal Consistency ◽

Pain Symptoms

Objectives: To study the measurement properties, the responsiveness and the minimal clinically important difference of the ENDOPAIN-4D: a new questionnaire for assessing pain in endometriosis. Methods: A prospective, observational, multicentre study was conducted including all women ≥18 years consulting for symptomatic proven endometriosis between 1 January 2017 and 30 June 2018 and volunteering to participate. Each patient had to answer a new self-administered patient-reported outcome (PRO) questionnaires (the ENDOPAIN-4D) at inclusion (T0) and 12 months after medical or surgical treatment (T1). Criteria defined by COSMIN were used to validate the questionnaire's measurement properties. The minimal clinically important difference was estimated by the anchor-based method. Results: The study included 199 women. The ENDOPAIN-4D score had a four dimensional structure with good internal consistency (measured by Cronbach α): I) pain-related disability (α = 0.79), II) painful bowel symptoms (α = 0.80), III) dyspareunia (α = 0.83), and IV) painful urinary tract symptoms (α = 0.77). They produced four subscores that can be summed to obtain a single score (α = 0.61). The ENDOPAIN-4D total score ranged from 0 to 94.00 (mean ± SD: 46.7 ± 22). The total score was significantly correlated with the PROs used in endometriosis. Sensitivity to change was good with large effect sizes (ES) (mean of the differences: 36.3 p = 1.8 10−7, ES 0.76). The minimal clinically important difference of the global score was determined to be 10.9. Conclusions: The ENDOPAIN-4D questionnaire is easy to use, valid, and effective in assessing patient reported pain symptoms in women treated for endometriosis. This new instrument can be used as the primary outcome for future clinical trials and as a tool for routine patient follow-up.

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A systematic review of the measurement properties of patient reported outcome measures used for adults with an ankle fracture

Journal of Patient-Reported Outcomes ◽

10.1186/s41687-019-0159-5 ◽

2019 ◽

Vol 3 (1) ◽

Cited By ~ 1

Author(s):

Rebecca McKeown ◽

David R. Ellard ◽

Abdul-Rasheed Rabiu ◽

Eleni Karasouli ◽

Rebecca S. Kearney

Keyword(s):

Systematic Review ◽

Construct Validity ◽

Outcome Measures ◽

Ankle Fracture ◽

Methodological Quality ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Measurement Properties ◽

Health Measurement ◽

Patient Reported

Abstract Background Ankle fractures are painful and debilitating injuries that pose a significant burden to society and healthcare systems. Patient reported outcome measures (PROMs) are commonly used outcome measures in clinical trials of interventions for ankle fracture but there is little evidence on their validity and reliability. This systematic review aims to identify and appraise evidence for the measurement properties of ankle specific PROMs used in adults with an ankle fracture using Consensus Based Standards for the Selection of Health Measurement Instrument (COSMIN) methodology. Methods We searched MEDLINE, Embase and CINAHL online databases for evidence of measurement properties of ankle specific PROMs. Articles were included if they assessed or described the development of the PROM in adults with ankle fracture. Articles were ineligible if they used the PROM to assess the measurement properties of another instrument. Abstracts without full articles and conference proceedings were ineligible, as were articles that adapted the PROM under evaluation without any formal justification of the changes as part of a cross-cultural validation or translation process. Two reviewers completed the screening. To assess methodological quality we used COSMIN risk of bias checklist and summarised evidence using COSMIN quality criteria and a modified Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach. Two reviewers assessed the methodological quality and extracted the data for a sample of articles. Results The searches returned a total of 377 articles. From these, six articles were included after application of eligibility criteria. These articles evaluated three PROMs: A-FORM, OMAS and AAOS. The A-FORM had evidence of a robust development process within the patient population, however lacks post-formulation testing. The OMAS showed sufficient levels of reliability, internal consistency and construct validity. The AAOS showed low quality evidence of sufficient construct validity. Conclusions There is insufficient evidence to support the recommendation of a particular PROM for use in adult ankle fracture research based on COSMIN methodology. Further validation of these outcome measures is required in order to ensure PROMs used in this area are sufficiently valid and reliable to assess treatment effects. This would enable high quality, evidenced-based management of adults with ankle fracture.

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Patient-Reported Outcome Measures for Post-mastectomy Breast Reconstruction: A Systematic Review of Development and Measurement Properties

Annals of Surgical Oncology ◽

10.1245/s10434-020-08736-8 ◽

2020 ◽

Vol 28 (1) ◽

pp. 386-404 ◽

Cited By ~ 1

Author(s):

C. F. Davies ◽

R. Macefield ◽

K. Avery ◽

J. M. Blazeby ◽

S. Potter

Keyword(s):

Systematic Review ◽

Breast Reconstruction ◽

Content Validity ◽

Patient Reported Outcome Measures ◽

Patient Reported Outcome ◽

Measurement Properties ◽

Future Studies ◽

Patient Reported ◽

Eortc Qlq

Abstract Background Breast reconstruction (BR) is performed to improve outcomes for patients undergoing mastectomy. A recently developed core outcome set for BR includes six patient-reported outcomes that should be measured and reported in all future studies. It is vital that any instrument used to measure these outcomes as part of a core measurement set be robustly developed and validated so data are reliable and accurate. The aim of this systematic review is to evaluate the development and measurement properties of existing BR patient-reported outcome measures (PROMs) to inform instrument selection for future studies. Methods A PRISMA-compliant systematic review of development and validation studies of BR PROMs was conducted to assess their measurement properties. PROMs with adequate content validity were assessed using three steps: (1) the methodological quality of each identified study was assessed using the COSMIN Risk of Bias checklist; (2) criteria were applied for assessing good measurement properties; and (3) evidence was summarized and the quality of evidence assessed using a modified GRADE approach. Results Fourteen articles reported the development and measurement properties of six PROMs. Of these, only three (BREAST-Q, BRECON-31, and EORTC QLQ-BRECON-23) were considered to have adequate content validity and proceeded to full evaluation. This showed that all three PROMs had been robustly developed and validated and demonstrated adequate quality. Conclusions BREAST-Q, BRECON-31, and EORTC QLQ-BRECON-23 have been well-developed and demonstrate adequate measurement properties. Work with key stakeholders is now needed to generate consensus regarding which PROM should be recommended for inclusion in a core measurement set.

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