New patient reported outcome (PRO) measures are regularly developed to assess various aspects of the patients’ perspective on their disease and treatment. For these instruments to be useful in clinical research, they must undergo a proper psychometric validation, including demonstration of cross-sectional and longitudinal measurement properties. This quantitative evaluation requires a study to be conducted on an appropriate sample size. The aim of this research was to list and describe practices in PRO and proxy PRO primary psychometric validation studies, focusing primarily on the practices used to determine sample size. A literature review of articles published in PubMed between January 2009 and September 2011 was conducted. Three selection criteria were applied including a search strategy, an article selection strategy, and data extraction. Agreements between authors were assessed, and practices of validation were described. Data were extracted from 114 relevant articles. Within these, sample size determination was low (9.6%, 11/114), and were reported as either an arbitrary minimum sample size (n = 2), a subject to item ratio (n = 4), or the method was not explicitly stated (n = 5). Very few articles (4%, 5/114) compared a posteriori their sample size to a subject to item ratio. Content validity, construct validity, criterion validity and internal consistency were the most frequently measurement properties assessed in the validation studies. Approximately 92% of the articles reported a subject to item ratio greater than or equal to 2, whereas 25% had a ratio greater than or equal to 20. About 90% of articles had a sample size greater than or equal to 100, whereas 7% had a sample size greater than or equal to 1000. The sample size determination for psychometric validation studies is rarely ever justified a priori. This emphasizes the lack of clear scientifically sound recommendations on this topic. Existing methods to determine the sample size needed to assess the various measurement properties of interest should be made more easily available.

/pdf/sample-size-used-to-validate-a-scale-a-review-of-2jzle0qink.pdf

Sample size used to validate a scale: a review of publications on newly-developed patient reported outcomes measures

Summary
Background Accurate diagnosis and effective management of gastro-oesophageal reflux disease (GERD) can be challenging for clinicians and other health care professionals.

Aim  To develop a patient-centred, self-assessment questionnaire to assist health care professionals in the diagnosis and effective management of patients with GERD.

Methods  Questions from patient-reported GERD instruments, previously documented in terms of content validity and psychometric properties (RDQ, GSRS and GIS) and data on the diagnosis of GERD in primary and secondary care were used in the formal development of a diagnostic and management tool, the GerdQ, involving psychometric validation and piloting in patient focus groups.

Results  Analyses of data from over 300 primary care patients, moderated by patient input from qualitative interviews, were used to select specific items from the existing instruments to create a new six-item diagnostic and management tool (GerdQ). ROC analysis indicated a sensitivity for GerdQ of 65% and a specificity of 71% for the diagnosis of GERD, similar to that achieved by gastroenterologists.

Conclusion  The GerdQ is a potentially useful tool for family practitioners and other health care professionals in diagnosing and managing GERD without initial specialist referral or endoscopy.

https://onlinelibrary.wiley.com/doi/pdf/10.1111/j.1365-2036.2009.04142.x

Development of the GerdQ, a tool for the diagnosis and management of gastro-oesophageal reflux disease in primary care.

ACG Clinical Guideline: Upper Gastrointestinal and Ulcer Bleeding.

SummaryBackground
The diagnosis of gastro-oesophageal reflux disease (GERD) remains a challenge as both invasive methods and symptom-based strategies have limitations. The symptom-based management of GERD in primary care may be further optimised with the use of a questionnaire.

Aim
To assess the diagnostic validity of the GerdQ questionnaire in patients with symptoms suggestive of GERD.

Methods
Patients with symptoms suggestive of GERD without alarm features, underwent upper endoscopy, and if normal, pH-metry. Patients were followed for 4 weeks and GerdQ was completed blinded to the investigator at both visits. Reflux oesophagitis or pathological acid exposure was used as diagnostic references for GERD. The diagnostic accuracy for GERD on symptom response to proton pump inhibitor (PPI) was assessed.

Results
Among the 169 patients, a GerdQ cutoff ≥9 gave the best balance with regard to sensitivity, 66% (95% CI: 58–74), and specificity, 64% (95% CI: 41–83), for GERD. The high prevalence of reflux oesophagitis (81%) resulted in a high proportion of true positives, but at the same time a high proportion of false-negatives. Consequently, GerdQ had a high positive predictive value, 92% (95% CI: 86–97), but a low negative predictive value, 22% (95% CI: 13–34), for GERD. Symptom resolution on PPI therapy had high sensitivity, 76% (95% CI: 66–84), but low specificity, 33% (95% CI: 17–53), for GERD.

Conclusions
GerdQ is a useful complementary tool for the diagnosis of gastro-oesophageal reflux disease in primary care. The implementation of GerdQ could reduce the need for upper endoscopy and improve resource utilisation. Symptom resolution on proton pump inhibitor did not predict gastro-oesophageal reflux disease.

https://onlinelibrary.wiley.com/doi/pdf/10.1111/apt.12204

Validation of the GerdQ questionnaire for the diagnosis of gastro-oesophageal reflux disease

Numerous questionnaires with a wide variety of characteristics have been developed for the assessment of gastroesophageal reflux disease (GERD) Four well-defined dimensions are noticeable in these GERD questionnaires, which are symptoms, response to treatment, diagnosis, and burden on the quality of life of GERD patients The aim of this review is to develop a complete overview of all available questionnaires, categorized per dimension of the assessment of GERD A systematic search of the literature up to January 2013 using the Pubmed database and the Embase database, and search of references and conference abstract books were conducted A total number of 65 questionnaires were extracted and evaluated Thirty-nine questionnaires were found applicable for the assessment of GERD symptoms, three of which are generic gastrointestinal questionnaires For the assessment of response to treatment, 14 questionnaires were considered applicable Seven questionnaires with diagnostic purposes were found In the assessment of quality of life in GERD patients, 18 questionnaires were found and evaluated Twenty questionnaires were found to be used for more than one assessment dimension, and eight questionnaires were found for GERD assessment in infants and/or children A wide variety of GERD questionnaires is available, of which the majority is used for assessment of GERD symptoms Questionnaires differ in aspects such as design, validation and translations Also, numerous multidimensional questionnaires are available, of which the Reflux Disease Questionnaire is widely applicable We provided an overview of GERD questionnaires to aid investigators and clinicians in their search for the most appropriate questionnaire for their specific purposes

Systematic review: questionnaires for assessment of gastroesophageal reflux disease

Summary

Background Although gastro-oesophageal reflux disease (GERD) has a well-established pathophysiology, the impact of GERD symptoms on the patients’ quality of life can be profound. Therefore, health-related quality of life (HRQOL) questionnaires have become standard instruments to evaluate the effect of treatment in clinical trials.



Aim  To evaluate the reliability, validity and responsiveness of available GERD-specific HRQOL.



Methods  A systematic literature search using the Medical Subject Headings terms: ‘Gastro-oesophageal reflux’, ‘Heartburn’ and ‘Oesophagitis’ with ‘quality of life’ and ‘questionnaires’ was preformed to identify the available HRQOL questionnaires. To analyse the psychometric properties of the respective tools, the actual guidelines for the use of patient-reported outcomes in clinical trials were applied.



Results  We identified five GERD-specific HRQOL questionnaires but none of them fulfilled all quality criteria; either they did not meet the actual standards for psychometric properties (HBQOL, GERD-HRQL, Jasani et al.Aust Fam Physician 1999; 28: 515), or were impracticable to use in clinical trials. The generic and hybrid instruments lack specificity and sensitivity and were not designed for treatment evaluation in GERD patients.



Conclusion  There is need for a new evaluative tool in the assessment of HRQOL during GERD therapy.

https://onlinelibrary.wiley.com/doi/pdf/10.1111/j.1365-2036.2008.03683.x

Systematic review: health-related quality of life (HRQOL) questionnaires in gastro-oesophageal reflux disease.

Background Controlled pantoprazole data in peptic ulcer bleeding are few. Aim To compare intravenous (IV) pantoprazole with IV ranitidine for bleeding ulcers. Methods After endoscopic haemostasis, 1256 patients were randomized to pantoprazole 80 mg+8 mg/h or ranitidine 50 mg+13 mg/h, both for 72 h. Patients underwent second-look endoscopy on day 3 or earlier, if clinically indicated. The primary endpoint was an overall outcome ordinal score: no rebleeding, rebleeding without/with subsequent haemostasis, surgery and mortality. The latter three events were also assessed separately and together. Results There were no between-group differences in overall outcome scores (pantoprazole vs. ranitidine: S0: 91.2 vs. 89.3%, S1: 1.5 vs. 2.5%, S2: 5.4 vs. 5.7%, S3: 1.7 vs. 2.1%, S4: 0.19 vs. 0.38%, P = 0.083), 72-h clinically detected rebleeding (2.9% [95% CI 1.7, 4.6] vs. 3.2% [95% CI 2.0, 4.9]), surgery (1.9% [95% CI 1.0, 3.4] vs. 2.1% [95% CI 1.1, 3.5]) or day-3 mortality (0.2% [95% CI 0, 0.09] vs. 0.3% [95% CI 0, 1.1]). Pantoprazole significantly decreased cumulative frequencies of events comprising the ordinal score in spurting lesions (13.9% [95% CI 6.6, 24.7] vs. 33.9% [95% CI 22.1, 47.4]; P = 0.01) and gastric ulcers (6.7% [95% CI 4, 10.4] vs. 14.3% [95% CI 10.3, 19.2], P = 0.006). Conclusions Outcomes amongst pantoprazole and ranitidine-treated patients were similar; pantoprazole provided benefits in patients with arterial spurting and gastric ulcers.

https://onlinelibrary.wiley.com/doi/pdf/10.1111/j.1365-2036.2008.03904.x

Clinical trial: intravenous pantoprazole vs. ranitidine for the prevention of peptic ulcer rebleeding: a multicentre, multinational, randomized trial.

Background A clinical need exists for a means of assessing symptom control in patients with gastro-oesophageal reflux disease. The ReQuest questionnaire has been extensively validated for symptom assessment in both erosive and non-erosive gastro-oesophageal reflux disease but was designed for research purposes. We derived a shorter version (ReQuest in Practice) that would be more convenient for clinical practice.

Aim  To validate ReQuest in Practice in patients suffering from gastro-oesophageal reflux disease.

Methods  Multicentre, non-interventional, crossover comparison. Patients completed ReQuest in Practice followed by ReQuest or vice versa. Before and after a planned endoscopy, patients completed the health-related quality of life questionnaire GERDyzer. Internal consistency and the Intraclass Correlation Coefficient were calculated. Construct validity was evaluated by correlation with ReQuest and GERDyzer.

Results  There was high internal consistency of ReQuest in Practice (Cronbach’s alpha: 0.9) and a high Intraclass Correlation Coefficient of 0.99. The measurement error of ReQuest in Practice was 4.1. High correlation between ReQuest in Practice and ReQuest (Spearman correlation coefficient: 0.9) and GERDyzer (Spearman correlation coefficient: 0.8) demonstrated construct validity.

Conclusions  ReQuest in Practice was proven to be valid and reliable. Its close correlation with ReQuest makes it a promising tool to guide the clinical management of patients across the full spectrum of both erosive and non-erosive gastro-oesophageal reflux disease.

https://onlinelibrary.wiley.com/doi/pdf/10.1111/j.1365-2036.2008.03641.x

Validation of a brief symptom questionnaire (ReQuest in Practice) for patients with gastro-oesophageal reflux disease.

Summary


Background Although erosive gastro-oesophageal reflux disease (GERD) is a highly prevalent condition, there is no specific, valid, reliable and sensitive questionnaire that allows evaluating treatment-induced changes in health-related quality of life (HRQoL).



Aim  To design a self-administered questionnaire, the GERD Analyzer (GERDyzer), for use in clinical studies.



Methods  The GERDyzer comprises 10 dimensions each illustrated by pictogram-like drawings, simplifying communication with the patients. Self-assessment is performed by 100 mm Visual Analogue Scales. For validation, a 5-week clinical trial involving 395 patients (per-protocol) with oesophagitis was conducted. Patients were treated with pantoprazole (40 mg o.d.) for 28 days. Psychometric analyses included internal consistency, test–retest reliability, responsiveness and construct validity.



Results  Factor analysis showed consistency of the dimensions and no reduction was necessary. Validation of GERDyzer indicated high internal consistency (Cronbach’s α = 0.95) and test–retest reliability (intraclass correlation coefficient =0.91). Responsiveness of the total score expressed by nonparametric effect size was 1.38. Comparison of scores with other questionnaires resulted in logical correlation levels depending on the respected concepts measured.



Conclusions  GERDyzer proved to be highly valid, reproducible and responsive. It allows reliably assessing treatment-induced changes in HRQoL in erosive GERD.

https://onlinelibrary.wiley.com/doi/pdf/10.1111/j.1365-2036.2008.03922.x

International validation of a health-related quality of life questionnaire in patients with erosive gastro-oesophageal reflux disease.

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Papers

Systematic review: health-related quality of life (HRQOL) questionnaires in gastro-oesophageal reflux disease.

Clinical trial: intravenous pantoprazole vs. ranitidine for the prevention of peptic ulcer rebleeding: a multicentre, multinational, randomized trial.

Validation of a brief symptom questionnaire (ReQuest in Practice) for patients with gastro-oesophageal reflux disease.

International validation of a health-related quality of life questionnaire in patients with erosive gastro-oesophageal reflux disease.