Disease-specific Patient-reported Outcome Measures Research Articles

Translation and validation of two disease-specific patient-reported outcome measures (Bladder Cancer Index and FACT-Bl-Cys) in Dutch bladder cancer patients

BackgroundThe Bladder Cancer Index (BCI) and Functional Assessment of Cancer Therapy-Bladder-Cystectomy (FACT-Bl-Cys) were developed to measure disease-specific health-related quality of life (HRQOL) in bladder cancer patients and patients treated with radical cystectomy, respectively. Both patient-reported outcome measures (PROMs) are frequently used in clinical practice, but are not yet validated according to the COSMIN criteria and not yet available in Dutch. Therefore, the aim of this study was to translate the BCI and FACT-Bl-Cys into Dutch and to evaluate their measurement properties according to the COSMIN criteria.MethodsThe BCI and FACT-Bl-Cys were translated into Dutch using a forward-backward method, and subsequently administered at baseline (pre-operatively) and 3 months post-operatively in bladder cancer patients who received a radical cystectomy. Validity (content and construct), reliability (internal consistency, test-retest reliability, and measurement error), floor and ceiling effects, and responsiveness were assessed according to the COSMIN criteria.ResultsForward-backward translation encountered no particular linguistic problems. In total 260 patients completed the baseline measurement, while 182 patients completed the three-month measurement. Only a ceiling effect was identified for the BCI. Hypotheses testing for construct validity was satisfying, as 67% and 92% of the hypothesized correlations were confirmed. Structural validity was moderate for both measures, as confirmatory factor analyses showed limited fit. Reliability of both PROMs was good. The intraclass correlation coefficient (ICC) of the BCI domains ranged from 0.47 to 0.93, minimal value of Cronbach’s α was 0.70, smallest detectable change on group level (SDC group) ranged from 1.9 to 8.6. The ICC of the FACT-Bl-Cys domains ranged from 0.43 to 0.83, minimal value of Cronbach’s α was 0.77, SDC group was around 1. Only the FACT-Bl-Cys total score was found to be responsive to changes in generic quality of life.ConclusionsThe Dutch versions of the BCI and FACT-Bl-Cys were shown to be reliable and have good content validity. Structural validity was limited for both measures. Only the FACT-Bl-Cys total score was responsive to changes in generic HRQOL. Despite some limitations, both PROMs seem suitable for use in clinical practice and research.

Qualitative exploration of the renal stone patients' experience and development of the renal stone-specific patient-reported outcome measure.

To investigate the experience of patients living with renal calculi via a qualitative methodology, aiming to develop and validate a disease-specific patient-reported outcome measure (PROM) for renal stones, the Cambridge Renal Stone PROM (CReSP). Patients with radiologically confirmed renal calculi who had undergone a range of management options were invited to focus groups or semi-structured interviews to elicit patient input and generate the PROM content. The developed renal stone PROM underwent validity studies included Cronbach's α for internal consistency, Spearman's and Pearson's correlation coefficients for test-retest reliability. Discriminant validity was assessed by Pearson's correlation coefficients vs the EuroQol five-dimensional five-level questionnaire (EQ-5D-5L). Our project has Health and Social Care Research Ethics Committee approval. A total of 106 subjects participated in creating the newly developed PROM. In all, 36 patients were invited to 22 semi-structured interviews and four focus groups, until reaching saturation. Major issues reported, and themes selected for the renal stone PROM included pain, anxiety, limitations to social life and tiredness, urinary symptoms, dietary changes' impacts, and gastrointestinal tract symptoms. Reliability analysis for 30 patients to determine internal consistency using Cronbach's α with a mean (range) of 0.91 (0.90-0.93) within domains and Cronbach's α between domains was 0.92. Average inter-item Pearson's and Spearman's correlation within domains was performed, with a Pearson's correlation mean (range) of 0.77 (0.73-0.85) and Spearman's correlation mean (range) of 0.72 (0.63-0.77). The test-retest Pearson's correlation mean (range) was 0.85 (0.57-0.95). Validity assessment was performed for 20 patients vs 20 controls. Pearson's correlation with EQ-5D-5L was -0.74, showing the newly developed PROM successfully discriminated patients with kidney stones. Our final renal stone PROM consists of 14 questions that are rated on a Likert scale; the higher the score, the worse the effect on a patient's quality of life. Although pain was the most frequent symptom, other health-related and social well-being issues significantly impacted patients' lives. Our validated patient-derived CReSP is a new instrument, specifically tailored to measure renal stone disease health outcomes from the patient's point of view.

Symptoms and Dietary Impact in Hypertriglyceridemia-Associated Pancreatitis: Development and Content Validity of Two New Measures.

BackgroundSevere hypertriglyceridemia (sHTG) is a rare condition, complicated by episodes of acute pancreatitis (AP), which can cause pain and/or life-threatening multi-organ dysfunction. Currently, there are no disease-specific patient-reported outcome (PRO) measures evaluating symptoms or dietary impact for this condition.ObjectiveThe objective of this study was to explore patient-reported symptoms and impacts of sHTG and AP and develop new measures to capture the symptoms and dietary impacts of this condition using patient language.MethodsIn-depth, semi-structured concept elicitation interviews were conducted with 12 US-based participants to explore their experience and identify key symptoms and impact on dietary behavior, both during and between episodes of AP. Participants had a range of AP severity with a previous triglyceride reading > 1000 mg/dL, and at least one attack of AP within the last 12 months. Transcripts were coded using thematic analysis.ResultsQualitative data analysis revealed the substantial burden of AP associated with sHTG. Participants reported experiencing symptoms, especially abdominal pain, both during and between attacks of AP, and discussed considerable diet changes to prevent or minimize future attacks. A conceptual model was refined, based on patient input, and reviewed by clinical experts to determine key concepts for inclusion within two PRO measures, one evaluating symptoms and another evaluating impact on dietary behavior. Items were drafted using patient-derived language. A 19-item symptoms measure [Hypertriglyceridemia and Acute Pancreatitis Symptom Scale (HAP-SS)] and a 6-item dietary impact measure (Hypertriglyceridemia and Acute Pancreatitis Dietary Behavior (HAP-DB) measure) were developed, both with a 24-h recall period.ConclusionsThe qualitative analysis confirmed the substantial burden of AP associated with sHTG. This research resulted in development of two disease-specific PRO measures for use during and between attacks of AP. These measures are being utilized in a clinical trial, which will confirm content, structure, and psychometric properties.Electronic supplementary materialThe online version of this article (10.1007/s41669-019-0155-y) contains supplementary material, which is available to authorized users.

Decompression alone vs. decompression plus fusion for claudication secondary to lumbar spinal stenosis

BACKGROUNDDegenerative lumbar spinal stenosis is a common condition, predominantly affecting middle-aged and elderly people. This study focused on patients with neurogenic claudication secondary to lumbar stenosis without spondylolisthesis or deformity. PURPOSETo determine whether the addition of fusion to decompression resulted in improved clinical outcomes at 3, 12, and 24 months postsurgery. STUDY DESIGN/SETTINGThe Canadian Spine Outcomes and Research Network (CSORN) prospective database that includes pre- and postoperative data from tertiary care hospitals. PATIENT SAMPLEThe CSORN database was queried for consecutive spine surgery cases of degenerative lumbar stenosis receiving surgical decompression for neurogenic claudication or radiculopathy. Neurogenic claudication patients with baseline and 2-year follow-up data, from four sites, formed the study sample (n=306). The sample was categorized into two groups: (1) those that had decompression alone, and (2) those that underwent decompression plus fusion. OUTCOME MEASURESChange in modified Oswestry Disability Index (ODI), numerical rating scale for back/leg pain, the EuroQol EQ5D, the SF-12 physical, and mental component scores. The primary outcome measure was the ODI at 2 years postoperative. METHODSWe conducted a multicenter, ambispective review of consecutive spine surgery patients enrolled between October 2012 and January 2018. RESULTSBaseline characteristics were comparable between groups except for female sex and multilevel pathology (both with greater proportion in the decompression plus fusion group). The decompression plus fusion group had clinically meaningfully more operative time, blood loss, rate of perioperative complication, and length of hospital stay (p<.05). These differences were preserved following adjustment for baseline differences between the groups.Both decompression and decompression plus fusion had a large clinically meaningful impact on generic and disease-specific patient-reported outcome measures within 3 months of surgery which was maintained out to 24-month follow-up. At any follow-up time point, there was no statistical evidence of a difference in these effects favoring decompression plus fusion over decompression alone. CONCLUSIONSThe addition of fusion to decompression did not result in improved outcomes at 3-, 12-, or 24-month follow-up. The addition of fusion to decompression provides no advantage to decompression alone for the treatment of patients with neurogenic claudication secondary to lumbar stenosis without spondylolisthesis or deformity.

Patient-reported outcome measures in subarachnoid hemorrhage: A systematic review.

ObjectivePatient-reported outcomes (PROs) are aspects of a patient's health status and are considered important for stimulating patient-centered care. Current outcome measures in clinical care for patients with aneurysmal subarachnoid hemorrhage (aSAH) are insufficient to capture PROs. In this systematic review, we aimed to summarize the evidence regarding the quality of patient-reported outcome measures (PROMs) in aSAH patients.MethodsWe performed a systematic review of the literature published from inception until October 29, 2018, in PubMed, the Cochrane Central Register of Controlled Trials, and EMBASE. Eligible studies had to evaluate measurement properties and capture PROs in aSAH patients. The quality of the studies and measurement properties were assessed using the consensus-based standards for the selection of health status measurement instruments (COSMIN) checklist. The review protocol was registered with PROSPERO (CRD42018058566).ResultsWe identified 9 articles that reported the assessment of 7 different disease-specific and generic PROMs used for aSAH patients, including 5 that focused on the Stroke-Specific Quality of Life Scale (SS-QoL). The methodologic quality of the validation processes used was generally doubtful. None of the PROMs complied with current standards for content validity.ConclusionsDue to the low quality of evidence for the measurement properties, the evidence base for selecting a suitable PROM for use with aSAH patients is insufficient. Given the specific long-term consequences of aSAH, we consider a disease-specific PROM the most appropriate, with SS-QoL the most suitable PROM currently available.

Validation and reliability of a disease-specific quality-of-life measure in patients with cutaneous lupus erythematosus.

Cutaneous lupus erythematosus (CLE) is a potentially disfiguring, chronic autoimmune disease with variable skin manifestations, negatively affecting patients' quality of life (QoL). Patient-reported outcome (PRO) measures assessing QoL in patients with CLE have been generic or developed without input from patients. To demonstrate the reliability and validity of a disease-specific QoL measure for CLE - the cutaneous lupus erythematosus quality of life (CLEQoL). One hundred and one patients with CLE were recruited, and each patient was asked to complete the CLEQoL. Internal consistency was used as a measure of reliability. Validity was measured in two ways - structural validity via exploratory factor analysis and convergent validity via Spearman correlations between CLEQoL and the Short Form 36 (SF-36), visual analogue scales and clinical variables. Patient demographic and disease characteristics were collected. The mean ± SD age of patients with CLE was 48 ± 13 years, with discoid lupus (n = 72; 71.3%) being the most predominant CLE subtype. Patients were mostly female (n = 88; 87·1%) and African American/Black (n = 59; 58·4%). Internal consistency ranged from 0·67 to 0·97. Five domains (functioning, emotions, symptoms, body image/cosmetic effects and photosensitivity) were extracted with a total explained variance of 71·1%. CLEQoL-related domains correlated with SF-36 domains (r range -0·39 to -0·65). The CLEQoL was found to be a valid and reliable PRO measure for assessing QoL in patients with CLE. Demonstrating that the CLEQoL has strong psychometric properties is an important step towards the development of a disease-specific PRO measure that future clinical trials can use.

Health Impairment From Nasal Airway Obstruction and Changes in Health Utility Values From Septorhinoplasty.

The association of nasal airway obstruction with health is significant, and the health care resources utilized in open septorhinoplasty need to be included in health economic analyses. To describe the association of nasal airway obstruction and subsequent open septorhinoplasty with patient health. A prospective case series study was conducted from September 30, 2009, to October 29, 2015, at 2 tertiary rhinologic centers in Sydney, Australia, among 144 consecutive adult patients (age, ≥18 years) with nasal airway obstruction from septal and nasal valve disorders. Open septorhinoplasty. Patients were assessed before undergoing open septorhinoplasty and then 6 months after the procedure. Health utility values (HUVs) were derived from the 36-Item Short Form Health Survey. Nasal obstruction severity was also measured using the Nasal Obstruction Symptom Evaluation (NOSE) questionnaire and the Sino-Nasal Outcome Test 22 questionnaires. A total of 144 patients (85 women and 59 men; mean [SD] age, 38 [13] years) were assessed. The baseline mean (SD) HUV for patients in this study was 0.72 (0.09), which was below the weighted mean (SD) Australian norm of 0.81 (0.22). After open septorhinoplasty, the mean (SD) HUV improved to 0.78 (0.12) (P < .001). Improvements in HUV were associated with changes in disease-specific patient-reported outcome measures, including Nasal Obstruction Symptom Evaluation scores (r = -0.48; P = .01) and Sino-Nasal Outcome Test 22 scores (r = -0.68; P = .01). Patients with nasal airway obstruction reported baseline HUVs that were lower than the Australian norm and similar to those in individuals with chronic diseases with significant health expenditure. There was a clinically and statistically significant improvement in HUVs after open septorhinoplasty that was associated with a reduction in Nasal Obstruction Symptom Evaluation and Sino-Nasal Outcome Test 22 scores. Outcomes from this study may be used for health economic analyses of the benefit associated with open septorhinoplasty. 4.

Characterizing the Health-Related Quality of Life Burden of Overactive Bladder Using Disease-Specific Patient-Reported Outcome Measures: A Systematic Literature Review.

IntroductionThe objective was to identify the most commonly used patient-reported outcome (PRO) instruments for overactive bladder (OAB), determine which are the most useful for measuring burden in OAB and characterize the findings of recent studies that have employed PRO instruments to assess OAB symptoms and the effects of treatment.MethodsA systematic search of OAB literature published between January 2006 and November 2017 using Medline/PubMed and EMBASE databases.ResultsOf 3425 abstracts and 500 full-text articles reviewed, 58 studies (both clinical trials and observational studies) were included in the review. The most commonly used PRO instruments were the OAB Questionnaire (OAB-q; 64%), followed by the King’s Health Questionnaire (KHQ; 31%) and the Patient Perception of Bladder Condition (PCBC; 21%). Synthesis of data from studies using the OAB-q showed that OAB treatment with antimuscarinics, mirabegron and onabotulinumtoxinA all improve health-related quality of life (HRQoL) and symptoms beyond the benefits observed with placebo. The OAB-q could detect dose–response relationships in some studies and demonstrated there were no significant differences across therapies from different drug classes.ConclusionThe HRQoL burden of OAB and response to treatment can be reliably measured by PRO instruments, and the OAB-q is the most commonly used instrument in OAB, particularly in clinical trials of OAB interventions. These data will be useful to provide benchmarks of burden levels for PRO scores obtained among those on contemporary therapies for comparison with outcomes from patients managed with emerging treatments.FundingAstellas Pharma Global Development, Inc.Electronic supplementary materialThe online version of this article (10.1007/s12325-019-0880-8) contains supplementary material, which is available to authorized users.

The requirement for a disease-specific patient-reported outcome measure of dysphagia in oculopharyngeal muscular dystrophy.

There is no patient-reported outcome (PRO) questionnaire specifically designed to assess oropharyngeal dysphagia in oculopharyngeal muscular dystrophy (OPMD). To select a suitable questionnaire, content validity of the existing questionnaires must be assessed. This study sought (1) to identify dysphagia-related symptoms in OPMD and (2) to assess content validity of currently available PRO for the assessment of dysphagia severity in OPMD. A two-step literature review was conducted of dysphagia-related symptom identification and oropharyngeal dysphagia-related PRO. Symptoms were validated with an expert panel by using a Delphi survey. Content validity of PRO questionnaires was documented through content analysis. Ten PRO questionnaires were identified. None of the questionnaires cover the entire symptom spectrum in OPMD and thus lack content validity. The development and validation of a new PRO questionnaire to assess dysphagia in OPMD is required to establish the importance of symptomatic relief from new treatments. Muscle Nerve 59:445-450, 2019.

Outcomes using the T-14 symptom score for tonsillectomy in an Australian paediatric population

Background: Quality of life (QoL) outcomes afterpaediatric tonsillectomy can be assessed with disease-specific patient reportedoutcome measures. Our aim was to assess a modified T-14 Paediatric ThroatDisorders Outcome Test in an Australian paediatric population undergoing tonsillectomy. Methods: A consecutive case series wasconducted in children (aged 1–16 years) undergoing tonsillectomy over a periodof 15 months. T-14 questionnaires administered to the caregivers of the childpre-operatively and post-operatively (n=45) within 6–8 weeks of the procedurewere included for statistical analysis. Pre- and post-operative T-14 scoreswere compared and analysed according to surgical indication [either sleepdisordered breathing (SDB), recurrent tonsillitis, or both] and complicationswith non-parametric tests. Results: Post-operative scores demonstrated aglobal improvement for SDB (n=22), recurrent tonsillitis (n=12) and for both(n=11) indications (P<0.0001 each). High compliance with the T-14 wasobtained with parents completing the questionnaire pre- and post-operatively.T-14 scores did not improve in 2 patients who had rhinitis and otitis media atthe time of post-operative testing. Conclusions: The modified T-14 questionnaire issuitable for measuring parental reported health outcomes for paediatric throatdisorders in patients undergoing tonsillectomy in the Australian population.The T-14 questionnaire demonstrates improvement in parental perception of theirchild’s symptoms after tonsillectomy. It is easy to administer producing a highcompliance rate.

Evaluation of the effect of urethroplasty for anterior urethral strictures by a validated disease-specific patient-reported outcome measure.

To better understand our urethroplasty outcome, we translated the English version of a urethral stricture surgery patient-reported outcome measure (USS-PROM) into Japanese and used it to evaluate the effect of urethroplasty for anterior urethral strictures. The PROM quantifies lower urinary tract symptoms (LUTS) and health-related quality of life (EQ-5D), and it evaluates overall satisfaction by asking patients to choose "very satisfied", "satisfied", "unsatisfied", or "very unsatisfied". 93 Japanese-speaking male patients with anterior urethral stricture who underwent urethroplasty completed it before (baseline) and 6months after urethroplasty. The psychometric criteria evaluated in the present study were the PROM's internal consistency, test-retest reliability, criterion validity, and responsiveness. Qmax was negatively correlated with the LUTS-total scores (r = - 0.61). Cronbach's alpha was 0.80 and the test-retest intraclass correlation coefficient for the LUTS-total score was 0.82. 83 patients (89.2%) achieved stricture-free, which was defined as no re-stricture on cystoscopy and no need for additional treatment. The mean total LUTS-score improved from 12.4 at baseline to 3.7 postoperatively (p < 0.0001). The mean EQ-5D visual analogue scores and EQ-5D index improved from 61.2 and 0.76, respectively, at baseline to 77.9 and 0.89 postoperatively (p < 0.0001, p < 0.0001). 55 patients (59.1%) were "very satisfied" with the outcome of their urethroplasty and 33 (35.5%) were "satisfied". The Japanese version of the USS-PROM has adequate psychometric properties. Urethroplasty improved not only objective data but also voiding symptoms and health-related QOL, and it resulted in a high rate of patient satisfaction.

Impact of Carotid Artery Stenosis on Quality of Life: A Systematic Review.

The aim of this study was to identify themes that determine health-related quality of life (HRQoL) in patients with carotid artery stenosis and identify the patient-reported outcome measures (PROMs) that best cover the identified themes. A systematic review of the main six databases from inception to September 2018 was undertaken to identify primary qualitative studies reporting on the HRQoL of patients with carotid artery stenosis. The quality of studies was assessed using the Critical Appraisal Skills Programme (CASP) criteria. Findings from the included studies were analysed using framework analysis methodology. The identified themes were mapped against the items/domains from the PROMs used previously in patients with carotid artery stenosis. The systematic review identified four papers that fulfilled the inclusion criteria. The included papers reported the views of 62 patients with symptomatic carotid artery stenosis; 24 of the patients were awaiting assessment for intervention, 26 had carotid endarterectomy, and 12 were turned down for intervention and received best medical therapy. The overall quality of the included studies was good based on CASP criteria. Framework analysis identified 16 themes that were divided into five main domains: anxiety, impact on personal roles and activities, effect on independence, psychological impact, and symptoms. The best-fit generic and disease-specific PROMs were the Medical Outcomes Study 36-Item Short Form (SF-36®) and the Carotid Stenosis Specific Outcome Measure (CSSOM), respectively. None of the PROMs covered all the themes identified in the qualitative systematic review. The findings from the review identified the important themes that affect patients with carotid stenosis disease. The current generic and disease-specific PROMs do not cover all themes that impact the HRQoL of patients suffering with this disease. The proposed themes can be used to develop a new disease-specific PROM to measure HRQoL.

Patient-Reported Outcome Measures in Neurosurgery: A Review of the Current Literature.

Patient-reported outcome measures (PROMs) play an important role in the evaluation of health outcomes, quality of life, and satisfaction, and have been successfully utilized in many areas of clinical medicine and surgical practice. The prevalence of PROMs in neurosurgery is not known. To review the PROMs that have been utilized in the published neurosurgery literature to date. Articles were searched in MEDLINE, EMBASE, HMIC Health Management Information Consortium, PsycARTICLES, and PsycINFO using search terms related to neurosurgery and PROMs, published from 1806 to August 2016. A total of 268 articles were identified that were stratified by the inclusion and exclusion criteria leading to a total of 137 articles. Twenty-six PROMs, involving both adult and pediatric populations, were identified. A large number of generic and disease-specific PROMs are used in the neurosurgical literature. Generic PROMs are usually nonspecific measures of health status. Disease-specific PROMs may not address issues relevant to neurosurgical procedures. There are very few neurosurgery-specific PROMs that take into account the impact of a neurosurgical procedure on a specific condition. PROMs that currently feature in the neurosurgical literature may not address the specific outcomes relevant to neurosurgical practice. There is an emergent need for generic and disease-specific PROMs to be validated in neurosurgical patients and neurosurgery-specific PROMs developed to address unmet needs of patients undergoing neurosurgical procedures.

Dyspnea in Amyotrophic Lateral Sclerosis: Rasch-Based Development and Validation of a Patient-Reported Outcome (DALS-15)

ContextDyspnea is a cardinal but often underestimated symptom in amyotrophic lateral sclerosis (ALS). The lack of a satisfying assessment tool leads to diagnostic uncertainty and bears the risk that established life-prolonging and symptom-relieving therapeutic options will not be adequately applied. ObjectivesThe objective of this study was to develop and validate a German language disease-specific patient-reported outcome measure to assess dyspnea in ALS by combination of a qualitative and quantitative approach using Rasch analysis. MethodsBased on input from clinical experts and patients, a preliminary 35-item questionnaire was developed and completed by 94 patients with ALS having dyspnea. Data were subjected to Rasch analysis and tested for required measurement issues such as appropriate response categories, the absence of differential item functioning, local independence, and unidimensionality. ResultsAfter iterative Rasch analyses, the final 15-item Dyspnea-ALS-Scale (DALS-15) was obtained. The scale satisfies the axioms of the Rasch model with good fit statistics, the absence of local dependency, and differential item functioning as well as acceptable unidimensionality. The DALS-15 is optimally targeted and suitable for group and individual use. It shows excellent test-retest reliability and convergent validity. ConclusionThe DALS-15 satisfies strictest modern measurement criteria and has interval scale properties. It fills an important gap in assessment and could be most helpful to optimize symptom management in patients with ALS.

Patient-reported outcome measurements in clinical routine of trauma, spine and craniomaxillofacial surgeons: between expectations and reality: a survey among 1212 surgeons

ObjectiveTo gain information about the advantages/disadvantages of an implementation of patient-reported outcome measures (PROM) into the clinical routine of trauma/orthopaedic surgeons, and to identify the technical constraints confronting a successful...

Lack of cross-cultural validity of the Endometriosis Health Profile-30.

Introduction:The Endometriosis Health Profile-30 is a disease-specific patient-reported outcome measure of health-related quality of life. Cross-cultural validation of the Endometriosis Health Profile-30 has been performed for several translated versions. The aim of this study was to evaluate the measurement properties of a Norwegian version Endometriosis Health Profile-30.Methods:This study was designed as a cross-sectional anonymous postal questionnaire study. A total of 157 women with endometriosis were included during a period from 2012 to 2013. Women aged 18–45 years were recruited from the Norwegian Endometriosis Association. Principal components analysis with varimax rotation was used to assess construct validity. Short Form-36 was used to determine convergent validity. Cronbach’s alpha was used to measure internal consistency. Intraclass correlation coefficients and paired t-tests were used to evaluate test–retest reliability. Floor and ceiling effects were estimated.Results:Factor analysis resulted in a three and five-factor model for the core and modular questionnaire, respectively. Factor analysis could not support construct validity of the scales self-image and treatment. The Norwegian version Endometriosis Health Profile-30 demonstrated acceptable internal consistency and test–retest reliability, except for the scale relationship with children. Floor effects were observed for the scales self-image (20.1%), work life (33.9%), relationship with children (34.2%), and medical profession (20.5%).Conclusion:The construct self-image does not seem to be measured appropriately by the Norwegian version Endometriosis Health Profile-30, suggesting a lack of cross-cultural validity of the Endometriosis Health Profile-30. With multinational studies increasing, adequate translation, cross-cultural adaptation, and cross-cultural validation of instruments are essential to ensure equivalence in languages and cultures other than the original.

Quality of life and functional assessment of facial palsy patients: A questionnaire study

BackgroundFacial palsy leads to functional and aesthetic deficits, which impair the quality of life of affected patients. General health-related and disease-specific questionnaires are available for quality of life assessment. In this study, observer-based analysis of facial function (Sunnybrook Facial Grading Scale) was compared patient-based to facial palsy-specific gradings (Facial Clinimetric Evaluation Scale and Facial Disability Index), and general health-related quality of life questionnaires (SF-36). We hypothesized that only facial palsy-specific instruments capture functional and social impairments of affected patients. MethodsThirty facial palsy patients treated at a tertiary referral centre were included in this study. Inclusion criteria were unilateral facial palsy with stable facial function, age over 18 years and fluency in German. Facial function was assessed with general and disease-specific patient-reported outcome measures and subsequently evaluated by the treating facial plastic surgeon. Statistical analysis included descriptive statistics for all assessed measurements. Correlations were calculated to compare general and facial palsy-specific instruments, as well as observer-based grading. ResultsObserver-based evaluation of facial function correlated well to the patients-based assessment of physical function, however social subscores did not correlate demonstrating the limited correlation of patient distress and facial nerve impairment. Physical function scores of disease-specific instruments did not correlate with general health assessment scores, while social function scores showed moderate to good correlations. ConclusionValidated disease-specific instruments are essential for the assessment of facial palsy patients. Patient-reported outcome measures like the FaCE Scale and the Facial Disability Index should be applied in addition to standardized observer-based ratings to capture the patients' perspective on functional and social impairments associated with facial palsy to fully assess the burden of disease.

Patient-Reported Outcome Measures in Urethral Reconstruction.

Treatment of anterior urethral stricture disease (USD) has shifted from endoscopic approaches to urethroplasty with significantly higher success rates among reconstructive urologists. This academic stance has led to a critical evaluation of "success" and developing disease-specific instruments to assess surgical outcomes focusing on patients' satisfaction rather than the historical goal of avoiding secondary procedures. Many disease non-specific and/or non-validated patient-reported outcome measures (PROMs) have been utilized to evaluate the voiding symptoms and sexual of function of patients after urethroplasty in the literature. Urethral Stricture Surgery PROM (USS PROM) is the first validated, disease-specific PROM for anterior USD which has been designed in 2001. Urethral Stricture Symptoms and Impact Measure (USSIM) is a comprehensive PROM and is currently being validated at multiple institutions. This article reviews the tools used to assess success after urethroplasty and elaborates the need to develop a comprehensive USD-specific PROM.

Themes that Determine Quality of Life in Patients with Peripheral Arterial Disease: A Systematic Review.

The aim of this study was to identify domains that determine quality of life in patients with peripheral arterial disease and find the patient-reported outcome measures that can examine the identified themes. A systematic review of all the main six databases was undertaken to identify primary qualitative studies reporting on the health and/or quality of life of patients with peripheral arterial disease. The quality of studies was assessed using the Critical Appraisal Skills Program criteria. Findings from the included studies were analysed using framework analysis methodology. The identified themes were mapped against the items/domains of validated patient-reported outcome measures used in patients with peripheral arterial disease. The systematic review identified eight papers that fulfilled the inclusion criteria. The included papers reported the views of 186 patients with peripheral arterial disease including patients with intermittent claudication, critical ischaemia and amputation secondary to peripheral arterial disease. The overall quality of the included studies was good based on Critical Appraisal Skills Program criteria. Framework analysis identified 35 themes that were divided into six main groups: symptoms, impact on physical functioning, impact on social functioning, psychological impact, financial impact and process of care. The best-fit generic and disease-specific patient-reported outcome measures were the Nottingham Health Profile and the Vascular Quality of Life Questionnaire, respectively. None of the patient-reported outcome measures covered all the themes important to patients with peripheral arterial disease. The findings from the review identified the important domains that affect patients living with peripheral arterial disease. None of the current generic and disease-specific patient-reported outcome measures provide a comprehensive measure for all themes that impact the daily living of patients with peripheral arterial disease.

Validation of the ANCA-associated vasculitis patient-reported outcomes (AAV-PRO) questionnaire

ObjectivesTo finalise and validate a disease-specific patient-reported outcome (PRO) measure: the ANCA-associated vasculitis patient-reported outcome (AAV-PRO) questionnaire. Using a 35-item candidate questionnaire developed following 50 qualitative interviews in the UK,...