Research Appraisals
Evidence-based critical appraisals of the latest medical research, systematically evaluated using Oxford CEBM methodology.
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International journal of medical informatics
Harmonizing patient-reported outcome measures for nasal complaints using traditional and machine learning methods
BACKGROUND: Nasal obstruction measurement instruments are widely used in the field of nasal surgery. There are various scales that measure nasal obstruction and they differ regarding the number of items, their wording, and the type of response options. In order to pool the data and analyze it together, it is necessary to harmonize it so that we can compare participants' nasal obstruction scores irrespective of instrument they filled in. Data harmonization is still not used in the field of nasal obstruction assessment. THE AIM: The aim of this study was to find the best harmonization method in terms of predicting the scores on a target instrument based on the scores from another instrument as precise as possible in the case of four different nasal complaints instruments. A method was sought to find a transformation of scores on the NOSE, Utrecht-Q and SCHNOS that makes them equivalent to ENFAS scores. METHODS: A total of 1324 unique patients completed all four measurement instruments. We tried linear equating, Item Response Theory (IRT), and the following machine learning methods: linear regression, random forest regression, support vector machine regression, and neural network. We used the root-mean-square error (RMSE) of differences between predicted and observed scores to evaluate the quality of harmonization in 5-fold cross-validation. RESULTS: The ML methods gave overall the best results (the lowest RMSEs) and outperformed IRT (which is considered as a common choice for data harmonization in psychometrics). CONCLUSION: The ML methods led to the best quality of the results, confirming their strong potential for data harmonization. This study shows that next to linear equating and IRT that are commonly used for data harmonization, we can also use ML methods for the same purpose and, by doing so, to even increase the quality of the harmonization in certain use cases.
6 May 2026
Read appraisal →The British journal of ophthalmology
Use of patient-reported outcomes in ophthalmology clinical trials between 2014 and 2023.
BACKGROUND/AIMS: Patient-reported outcomes (PROs) evaluate health and functional status, and PRO measures (PROMs) are standardised tools for measuring PROs. Together, they provide valuable insights into treatment efficacy, safety and practicality not captured by traditional clinical endpoints. This cross-sectional analysis with a systematic search component aims to investigate the use, interpretation and reporting of PROs and PROMs in ophthalmic randomised controlled trials (RCTs). METHODS: Ophthalmic RCTs published in the top 10 highest impact factor ophthalmic and medical journals between 2014 and 2023 were systematically reviewed. The frequency of PRO inclusion and adherence to Consolidated Standards of Reporting Trials (CONSORT) PRO guidelines was assessed. The relationship between PRO utilisation and study-level and journal-level characteristics was explored with multivariable regression. RESULTS: Among 9436 records screened, 333 RCTs met eligibility criteria. Of these, 87 (26.1%) included PROs, and 28 (8.4%) used them as primary outcomes. Most studies (83/87, 95.4%) leveraged PROMs, with ophthalmology-specific tools predominating (73.5%). Minimal important differences (MIDs) were rarely used (2.3%) for PRO interpretation. At least 8/13 CONSORT PRO Extension items were reported in 33.3% studies, and trials with primary PRO endpoints had better adherence (p<0.001). PRO utilisation was less likely in trials with lower 5-year journal impact factor (adjusted OR (aOR) 0.99, 95% CI 0.98 to 1.00, p=0.037) and pharmaceutical compared with health service interventions (aOR 0.15, 95% CI 0.03 to 0.67, p=0.013). CONCLUSIONS: PRO integration and interpretation remain limited in high-impact ophthalmic RCTs, despite offering a meaningful adjunct to objective endpoints. Future trials should adopt validated and condition-specific PROMs and establish MIDs to enhance interpretation.
26 Apr 2026
Read appraisal →Sleep & breathing = Schlaf & Atmung
Health-related quality of life in patients with obstructive sleep apnea - a systematic review update assessing the quality of measurement properties of patient-reported outcome measures
PURPOSE: The purpose of this systematic review update was to identify existing health-related quality of life (HRQOL) patient-reported outcome measures (PROMs) for obstructive sleep apnea (OSA). Building upon a 2016 systematic review, which recommended four PROMs with the highest evidence, this investigation was warranted to synthesize new evidence and evaluate new PROMs that have emerged since then via the Consensus based Standards for the Selection of Health Measurement Instruments (COSMIN). METHODS: We searched Medline via PubMed, Embase and the Cochrane Library for HRQOL PROM development and validation studies published in English or German. Studies focusing on cross-cultural validation, those in which PROMs were assessed as secondary outcomes, and studies limited to specific demographic subgroups or single HRQOL domains were excluded. For risk of bias and quality of measurement properties (MP) assessment, we used the COSMIN methodology to facilitate comparability with the previous review, as well as the GRADE approach for evidence level evaluation. RESULTS: We identified two new PROMs, the Patient-Reported Apnea Questionnaire (PRAQ) and the Symptoms, Tiredness, Alertness, Mood and Psychosocial instrument (STAMP), which were validated in two studies and a single study, respectively. Overall, the PRAQ displays an moderate risk of bias with excellent quality of MP and high evidence for content validity in particular, whereas the STAMP shows a higher risk of bias and low quality of MP. CONCLUSION: The PRAQ has demonstrated the strongest evidence among PROMs since 2016. In total, including the previous review, five PROMs are currently recommended for assessing HRQOL in individuals with OSA. REVIEW REGISTRATION: The review was registered with PROSPERO under the registration number CRD42024602020.
24 Apr 2026
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