Disease-specific Patient-reported Outcome Measures Research Articles

Mapping the Edmonton Symptom Assessment System-Revised: Renal to the EQ-5D-5L in patients with chronic kidney disease.

The Edmonton Symptom Assessment System-Revised: Renal (ESAS-r: Renal) is a disease-specific patient-reported outcome measure (PROM) that assesses symptoms common in chronic kidney disease (CKD). There is no preference-based scoring system for the ESAS-r: Renal or a mapping algorithm to predict health utility values. We aimed to develop a mapping algorithm from the ESAS-r: Renal to the Canadian EQ-5D-5L index scores. We used data from a multi-centre cluster randomized-controlled trial of the routine measurement and reporting of PROMs in hemodialysis units in Northern Alberta, Canada. In two arms of the trial, both the ESAS-r: Renal and the EQ-5D-5L were administered to CKD patients undergoing hemodialysis. We used data from one arm for model estimation, and data from the other for validation. We explored direct and indirect mapping models; model selection was based on statistical fit and predictive power. Complete data were available for 506 patient records in the estimation sample and 242 in the validation sample. All models tended to perform better in patients with good health, and worse in those with poor health. Generalized estimating equations (GEE) and generalized linear model (GLM) on selected ESAS-r: Renal items were selected as final models as they fitted the best in estimation and validation sample. When only ESAS-r: Renal data are available, one could use GEE and GLM to predict EQ-5D-5L index scores for use in economic evaluation. External validation on populations with different characteristics is warranted, especially where renal-specific symptoms are more prevalent.

Current Recommendations for Patient-Reported Outcome Measures Assessing Domains of Quality of Life in Neurofibromatosis Clinical Trials.

To review and recommend patient-reported outcome (PRO) measures assessing multidimensional domains of quality of life (QoL) to use as clinical endpoints in medical and psychosocial trials for children and adults with neurofibromatosis (NF) type 1, NF2, and schwannomatosis. The PRO working group of the Response Evaluation in Neurofibromatosis and Schwannomatosis (REiNS) International Collaboration used systematic methods to review, rate, and recommend existing self-report and parent-report PRO measures of generic and disease-specific QoL for NF clinical trials. Recommendations were based on 4 main criteria: patient characteristics, item content, psychometric properties, and feasibility. The highest-rated generic measures were (1) the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales for NF clinical trials for children or for children through adults, (2) the Functional Assessment of Cancer Therapy-General for adult medical trials, and (3) the World Health Organization Quality of Life-BREF for adult psychosocial trials. The highest-rated disease-specific measures were (1) the PedsQL NF1 Module for NF1 trials, (2) the NF2 Impact on Quality of Life Scale for NF2 trials, and (3) the Penn Acoustic Neuroma Quality of Life Scale for NF2 trials targeting vestibular schwannomas. To date, there are no disease-specific tools assessing multidimensional domains of QoL for schwannomatosis. The REiNS Collaboration currently recommends these generic and disease-specific PRO measures to assess multidimensional domains of QoL for NF clinical trials. Additional research is needed to further evaluate the use of these measures in both medical and psychosocial trials.

The Epworth Sleepiness Scale in epilepsy: Internal consistency and disease-related associations

BackgroundThe Epworth Sleepiness Scale (ESS) is the most common instrument for measuring subjective sleep propensity in people with epilepsy but has not yet been validated in this population. Study objectivesWe aimed to systematically assess the validity, performance, and internal consistency of the ESS, as well as correlations between the ESS and disease-specific variables and patient-reported outcome measures in a cohort of adults with epilepsy (AWE). MethodsNinety-five AWE completed sleep and seizure diaries, in-laboratory polysomnography (PSG) and patient-reported outcome measures, including the ESS, Insomnia Severity Index (ISI), and the Beck Depression Inventory (BDI). Demographic information and data from 95 matched controls referred for PSG for suspected obstructive sleep apnea (OSA) was taken from the electronic medical record. Frequencies of high ESS item ratings (item score ≥2) were calculated for each group. Cronbach’s α and factor analysis were performed to assess the internal consistency and validity of the ESS within cases and controls. Multivariable linear models were used to assess the association between ESS and predictors of interest, adjusting for demographic and disease-specific variables, including seizure type, frequency, and anti-seizure medication (ASM) therapy. ResultsWhile suspected OSA controls had significantly greater mean ESS total scores (9.9 vs 7.9, p = 0.004) and proportion with ESS >10 (42% vs 25%, p = 0.014), there were no significant differences in the severity of item responses, with the exception of “lying down to rest in the afternoon when circumstances permit,” for which more controls rated as likely/very likely (79% vs 64%), p = 0.024). AWE with ESS >10 had higher mean standardized ASM dose (2.5 vs 1.7, p = 0.026). All ESS items were significantly correlated with the total score within each group. Cronbach’s α was 0.75 for cases and 0.85 for controls, indicating good internal consistency of the ESS for both groups. After adjusting for demographic and sleep characteristics, higher ESS scores were associated with greater insomnia scores on the ISI (p = 0.024) and depressive symptoms on the BDI (p = 0.018). ConclusionsThis study provides validity for the use of the ESS in adult populations with epilepsy.

Identification and categorisation of relevant outcomes for symptomatic uncomplicated gallstone disease: in-depth analysis to inform the development of a core outcome set

BackgroundMany completed trials of interventions for uncomplicated gallstone disease are not as helpful as they could be due to lack of standardisation across studies, outcome definition, collection and reporting. This...

P116 Development of a new patient-reported outcome measure for patients with complex cryptoglandular fistulas

Abstract Background Complex cryptoglandular fistulas (CCF) arise from infected anal glands and are often associated with substantial morbidity and healthcare utilization. Standarized patient-reported outcome measures (PROMs) for CCF are lacking. Thus, we aimed to develop a disease-specific PROM to assess the burden of symptoms and their impact on daily life in patients with CCF. Methods To develop a conceptual model, a targeted literature review was conducted to identify symptoms and impacts on daily life associated with CCF. Existing PROMs relating to anal fistulas were also sought. Semi-structured interviews with colorectal surgeons (n=5) assessed clinical perspectives on patient experience of CCF. A draft item pool was developed based on the refined conceptual model and using the Quality of Life in Patients with Anal Fistula Questionnaire as a reference. The tool was refined through concept elicitation interviews on the symptoms and impacts of CCF, and cognitive debriefing on the interpretation, understanding and response to each item. Interviews were conducted until concept saturation was achieved and patient feedback suggested no further refinements were required. Results The literature review identified discharge, pain, faecal incontinence and bleeding as the most prevalent symptoms, and embarrassment as the most prevalent impact on daily life. Results from surgeon interviews were then used to revise the initial conceptual model. Twenty US adults (60% female; mean age, 49 years) with clinically confirmed CCF participated in interviews (four waves of n=5). Patients identified 10 salient symptoms and 11 salient impacts on daily life (salient defined as mention by ≥50% of patients and mean disturbance rating ≥5 on a 10-point scale). The final conceptual model included the main symptoms and treatment-related effects, and impacts on daily life. The draft item pool was refined, resulting in a final PROM consisting of 14 items (covering frequency and severity) relating to symptom domains – discharge, incontinence, pain, irritation, odour, abscess – and 6 items relating to health-related quality of life domains – functional, physical, psychological, social health. The PROM features a 7-day recall period, with responses mostly given on a 5-point verbal rating scale. Cognitive debriefing confirmed that the PROM was clear, easy to understand and relevant to patients’ experiences. Conclusion The 20-item CCF questionnaire (CCFQ-20) is a new PROM that has been developed and tested for content validity, following expert guidance and regulatory best practices. It addresses a comprehensive set of salient symptoms and impacts experienced by patients with CCF. Psychometric testing is required to fully evaluate this PROM. Sponsor: Takeda Pharmaceuticals USA, Inc.

Patient-reported outcomes and patient-reported outcome measures in interstitial lung disease: where to go from here?

Patient-reported outcome measures (PROMs), tools to assess patient self-report of health status, are now increasingly used in research, care and policymaking. While there are two well-developed disease-specific PROMs for interstitial lung diseases (ILD) and idiopathic pulmonary fibrosis (IPF), many unmet and urgent needs remain. In December 2019, 64 international ILD experts convened in Erice, Italy to deliberate on many topics, including PROMs in ILD. This review summarises the history of PROMs in ILD, shortcomings of the existing tools, challenges of development, validation and implementation of their use in clinical trials, and the discussion held during the meeting. Development of disease-specific PROMs for ILD including IPF with robust methodology and validation in concordance with guidance from regulatory authorities have increased user confidence in PROMs. Minimal clinically important difference for bidirectional changes may need to be developed. Cross-cultural validation and linguistic adaptations are necessary in addition to robust psychometric properties for effective PROM use in multinational clinical trials. PROM burden of use should be reduced through appropriate use of digital technologies and computerised adaptive testing. Active patient engagement in all stages from development, testing, choosing and implementation of PROMs can help improve probability of success and further growth.

Patient-Reported Outcomes in Inflammatory Bowel Disease: A Measurement of Effect in Research and Clinical Care

The measurement of outcomes is key in evaluating healthcare or research interventions in inflammatory bowel disease (IBD). In patient-centred care, patient-reported outcome measures (PROMs) are central to this evaluation. In this review, we provide an overview of validated, adult disease-specific PROMs developed for use in IBD. Our aim is to assist clinicians and researchers in selection of PROMs to measure outcomes in their patient cohort. The Consensus-based Standards for the Selection of Health Measurement Instruments database of systematic reviews was the primary resource used to identify PROMs used in IBD. Search terms were ‘Crohn’s disease’, ‘ulcerative colitis’, and ‘IBD’. Seven systematic reviews were identified from this search. In addition, the publication by the IBD Core Outcome Set Working Group was used to identify further PROMs. Three systematic reviews were excluded as they did not meet the inclusion criteria. From the five included systematic reviews, we identified 21 PROMs and their shortened versions. In conclusion, it does not appear that any one PROM is entirely suitable for both research and clinical practice. Overall, the IBDQ-32 is most widely used in research but has the limitation of cost, whereas the IBD-Control has been recommended in the clinical core outcome set.

Evaluation of PROMIS’ Ability to Detect Immediate Postoperative Symptom Improvement Following Carpal Tunnel Release

We sought to determine whether subjective clinical improvement immediately after carpal tunnel release (CTR) was captured by Patient-Reported Outcomes Measurement Information System (PROMIS) Upper Extremity (UE), Physical Function (PF), Pain Interference (PI), and Depression. Between September 2018 and January 2020, patients presenting to a single academic medical center hand clinic were asked to complete PROMIS UE, PF, PI, and Depression computer adaptive tests. In addition, patients who had CTR were asked to answer the following at their first postoperative clinic visit: "Since my last clinic visit, my condition is: (1) much better; (2) mildly better; (3) no change; (4) mildly worse; (5) much worse." For each patient, the last clinic visit was the final preoperative visit. The PROMIS domain scores were compared before and after surgery using paired t tests. The percentage of patients subjectively reporting better symptoms was calculated. A total of 156 patients fit our inclusion criteria. The average number of days between the final preoperative visit and CTR was 7 (range, 0-30), and the average number of days between CTR and the first postoperative visit was 9 (range, 3-21). A total of 116 patients (74%) reported their carpal tunnel syndrome was better at their first postoperative visit. However, PROMIS UE, PF, and PI scores were significantly worse at the first postoperative visit, although not at clinically appreciable levels. There was no statistical or clinical difference in PROMIS Depression scores from pre- to postoperative time points. Nearly 75% of patients subjectively report their carpal tunnel syndrome is better at their first follow-up visit within 3 weeks of CTR; however, PROMIS does not capture this improvement. Hand surgeons evaluating patients shortly following CTR should be aware of the potential limitation of PROMIS to accurately capture immediate postoperative clinical outcomes. Disease-specific Patient-Reported Outcome Measures, such as the Boston Carpal Tunnel Questionnaire, may be preferred during this immediate postoperative timeframe.

Changes in quality of life (QoL) and other patient-reported outcome measures (PROMs) in living-donor and deceased-donor kidney transplant recipients and those awaiting transplantation in the UK ATTOM programme: a longitudinal cohort questionnaire survey with additional qualitative interviews

ObjectiveTo examine quality of life (QoL) and other patient-reported outcome measures (PROMs) in kidney transplant recipients and those awaiting transplantation.DesignLongitudinal cohort questionnaire surveys and qualitative semi-structured interviews using thematic analysis...

Outcome Measures and Quality of Life in Vestibular Schwannomas

This review summarizes existing vestibular schwannoma (VS) disease-specific and non-disease-specific patient-reported outcome measure (PROM) tools for quality of life (QoL) and audio-vestibular symptoms, compares QoL across treatment modalities, and discusses the potential role of psychological variables in QoL and recovery after surgical resection of VS. VS treatment success was previously assessed according to various factors such as the extent of tumor resection, facial nerve function, and hearing preservation. However, the literature demonstrates recent shifts away from using such physician-reported “objective” outcomes as benchmarks for success towards more patient-perceived “subjective” measures. A number of PROM tools have been developed to assess QoL and other measures in patients with VS. This has allowed us to better understand and quantify the impact of the disease and of the treatments on our patient population. However, there are important considerations to be made when applying PROM in clinical settings such as understanding the concept of Minimal Clinically Important Difference (MCID). The use of disease-specific PROMs, such as the PANQOL, as a primary outcome in VS research is encouraged. When doing so, PROM scales should incorporate the concept of MCID to distinguish statistically significant from clinically significant findings and should further investigate the role of non-medical variables, such as psychological variables, on QoL and recovery trajectories in patients with VS. Actual literature does not seem to indicate a clinically significant difference in patient-reported outcomes among treatment modalities.

Patient perceptions of health-related quality of life in giant cell arteritis: international development of a disease-specific patient-reported outcome measure.

ObjectivesGCA is a large vessel vasculitis (LVV) presenting with headache, jaw claudication, musculoskeletal and visual involvement. Current treatment is glucocorticoids and anti-IL-6 tocilizumab in refractory disease. The objective of this study was to explore the impact of GCA and its treatment on people’s health-related quality of life (HRQoL), to inform the development of a disease-specific patient-reported outcome measure (PROM) for use in clinical trials and practice.MethodsParticipants from the UK and Australia, with biopsy- or imaging-confirmed GCA, were interviewed to identify salient aspects of HRQoL in relation to GCA and its treatment. Purposive sampling included a range of demographic and disease features (cranial, LVV-GCA and visual involvement). Inductive analysis identified individual themes of importance, then domains. Candidate questionnaire items were developed from the individual themes, refined by piloting, cognitive interviews and a linguistic translatability assessment.ResultsThirty-six interviews were conducted to saturation with participants with GCA from the UK (25) and Australia (11). Mean age was 74 years, 23 (63.9%) were female, 13 (36.1%) had visual loss and 5 (13.9%) had LVV-GCA. Thirty-nine individual themes within five domains were identified: physical symptoms; activity of daily living and function; participation; psychological impact; and impact on sense of self and perception of health. Sixty-nine candidate items were developed from individual themes; piloting and refinement resulted in a 40-item draft questionnaire.ConclusionThis international qualitative study underpins the development of candidate items for a disease-specific PROM for GCA. The draft questionnaire is now ready for psychometric testing.

RESULTS FROM A NATIONAL CROSS-SECTIONAL STUDY OF DISEASE BURDEN IN CROHN’S DISEASE

Abstract Background Patients with Crohn’s disease experience a wide variety of clinical symptoms that affect how they feel and function. As therapeutic trials are planned for patients with Crohn’s disease, it is important to better understand the symptoms that have the greatest impact on Crohn’s disease patient’s lives. Objective To identify the most common and important disease manifestations in Crohn’s disease in a large population of patients. To determine the modifying factors that are associated with these symptoms. Methods We conducted a national cross-sectional study of 415 patients from the IBD Partners patient registry sponsored by the Crohn’s & Colitis Foundation to identify the prevalence and relative importance of 148 individual symptoms across 17 unique symptomatic themes. These themes were previously identified through 16 semi-structured qualitative interviews with Crohn’s patients. Results Crohn’s disease participants provided over 55,000 symptom rating responses. The symptomatic themes with the highest prevalence in Crohn’s disease were gastrointestinal issues (93.0%), fatigue (86.4%), dietary restrictions (77.9%), impaired sleep or daytime sleepiness (75.6%), and inability to do activities (72.3%). Symptomatic theme prevalence was widely associated with having above the median number of stools per day, having above the median number of bowel movements per day, having perianal disease, having to miss work, and unemployment. Discussion/Conclusion: Crohn’s disease symptoms, some under-recognized, vary based on disease characteristics and demographic features. These symptoms represent targets for future therapeutic interventions and are potential areas of interest for an upcoming disease-specific patient-reported outcome measure for this population. Acknowledgements Funding for this project was provided by UR Ventures. Research activities were conducted in collaboration with the Crohn’s & Colitis Foundation.

The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment.

BackgroundPatient-reported outcome measures (PROMs) are used in health technology assessment (HTA) to measure patient experiences with disease and treatment, allowing a deeper understanding of treatment impact beyond clinical endpoints. Developing and administering PROMs for rare diseases poses unique challenges because of small patient populations, disease heterogeneity, lack of natural history knowledge, and short-term studies.ObjectiveThis research aims to identify key factors to consider when using different types of PROMs in HTA for rare disease treatments (RDTs).MethodsA scoping review of scientific and grey literature was conducted, with no date or publication type restrictions. Information on the advantages of and the challenges and potential solutions when using different types of PROMs for RDTs, including psychometric properties, was extracted and synthesized.ResultsOf 79 records from PubMed, 32 were included, plus 12 records from the grey literature. PROMs for rare diseases face potential data collection and psychometric challenges resulting from small patient populations and disease heterogeneity. Generic PROMs are comparable across diseases but not sensitive to disease specificities. Disease-specific instruments are sensitive but do not exist for many rare diseases and rarely provide the utility values required by some HTA bodies. Creating new PROMs is time and resource intensive. Potential solutions include pooling data (multi-site/international data collection), using computer-assisted technology, or using generic and disease-specific PROMs in a complementary way.ConclusionsPROMs are relevant in HTA for RDTs but pose a number of difficulties. A deeper understanding of the potential advantages of and the challenges and potential solutions for each can help manage these difficulties.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40271-020-00493-w.

Development of a Patient-Reported Outcome Measure for Non-Alcoholic Steatohepatitis (NASH-CHECK): Results of a Qualitative Study

BackgroundNon-alcoholic steatohepatitis (NASH) is a progressive form of non-alcoholic fatty liver disease characterized by excessive liver fat accumulation, inflammation, cell injury, and fibrosis. It is viewed as largely asymptomatic in its earlier (non-cirrhotic) stages, and information on the patient-perceived impact of NASH is scarce.ObjectiveThis study aimed to develop a NASH-specific patient-reported outcome (PRO) measure (NASH-CHECK) for use as a trial endpoint, using methods compliant with regulatory expectations.MethodsA NASH conceptual model was developed based on the literature and clinical/patient expert review. The model guided concept elicitation (CE) interviews in patients with non-cirrhotic NASH recruited via a US tertiary care center. NASH-CHECK content was generated via thematic analysis of CE data and review by clinical/patient experts. Cognitive debriefing (CD) interviews with US patients evaluated content validity.ResultsThe literature review confirmed that NASH impacts on functioning and health-related quality of life (HRQoL). Overall, 23 CE and 20 CD interviews were conducted. Key symptoms reported in CE interviews included pain in the upper-right abdomen (n = 14), fatigue (n = 18), poor sleep quality (n = 12), impaired memory (n = 13), and reduced focus (n = 11); key HRQoL impacts included impaired physical functioning, reduced ability to conduct daily living tasks, reduced quality of relationships, low mood, anxiety, and self-consciousness. The 52-item first-draft NASH-CHECK was reduced to 31 items based on patient feedback on item relevance, acceptability, and comprehension.ConclusionsThe interviews revealed key symptoms and broad HRQoL impacts of NASH. As a disease-specific PRO measure assessing symptoms and HRQoL, the NASH-CHECK is relevant, comprehensive, and acceptable to patients and clinicians.

I-WISh: A wish list for immune thrombocytopenia quality of life indicators becomes reality.

I-WISh: A wish list for immune thrombocytopenia quality of life indicators becomes reality.

Readability analysis of pediatric otolaryngology patient-reported outcome measures

Objective(s): Patient-reported outcome measures (PROMs) are tools that allow patients to directly share information about their health with their healthcare provider. Health literacy experts recommend that health information, such as PROMs, be written at a 6th grade level to ensure patients can read and comprehend it. As the readability of PROMs used in pediatric otolaryngology has yet to be studied, our goal was to analyze the readability of these PROMs and assess their compliance with readability recommendations. MethodsThe Gunning Fog, the Simple Measure of Gobbledygook (SMOG), and the FORCAST readability formulas were used to determine the readability of disease-specific PROMs for pediatric otolaryngology. ResultsFourteen PROMs were reviewed in the study. Most were intended for caregiver completion (n = 13, 92.9%). Ten PROMs when measured by Gunning Fog (71.4%), 2 PROMs when measured by SMOG (14.3%), and 0 PROMs when measured by FORCAST (0.0%) were at or below the 6th grade reading level. ConclusionMost PROMs available for use in pediatric otolaryngology are above the recommended 6th grade reading level when measured by FORCAST, the most appropriate metric for assessing questionnaires. The high reading grade level needed to complete these PROMs can contribute to health disparities among underserved and vulnerable populations, such as children. Pediatric otolaryngology PROMs developed in the future should take readability into account in order to ensure equity in the delivery of care.

Rhinologic disease and its impact on sleep: a systematic review.

Rhinologic disease can be responsible for systemic symptoms affecting mood, cognition, and sleep. It is unclear whether sleep disturbance in specific rhinologic disorders (chronic rhinosinusitis [CRS], rhinitis, and nasal septal deviation [NSD]) is an obstructive phenomenon or due to other mechanisms. In this review we examine the impact of CRS, rhinitis, and NSD on objective and subjective sleep outcome metrics and draw comparisons to normal controls and patients with known obstructive sleep apnea (OSA). A systematic review of 4 databases (PubMed, Scopus, Cochrane Library, and Web of Science) was performed. Studies reporting on objective (apnea-hypopnea index [AHI], respiratory disturbance index [RDI], oxygen nadir) and subjective (Epworth Sleepiness Scale [EpSS], Pittsburgh Sleep Quality Index [PSQI], Fatigue Severity Scale [FSS]) sleep parameters and disease-specific patient-reported outcome measures (PROMs; 22-item Sino-Nasal Outcome Test [SNOT-22], Rhinoconjunctivitis Quality of Life Questionnaire [RQLQ], Nasal Obstruction Symptom Evaluation [NOSE]) were included. The database search yielded 1414 unique articles, of which 103 were included for analysis. Baseline PROMs were at the high end of normal to abnormal for all 3 conditions: EpSS: CRS (9.8 ± 4.0), rhinitis (9.7 ± 4.3), and NSD (8.9 ± 4.6); and PSQI: CRS (11.0 ± 4.5), rhinitis (6.1 ± 3.7), and NSD (8.6 ± 3.5). Objective measures demonstrated a mild to moderate OSA in the studied diseases: AHI: CRS (10.4 ± 11.5), rhinitis (8.6 ± 8.8), and NSD (13.0 ± 6.9). There were significant differences when compared with reported norms in all measured outcomes (p < 0.001). Sleep quality is impacted by rhinologic (CRS, rhinitis, NSD) disease. There is likely a mild obstructive component contributing to poor sleep, but other contributing factors may be involved.

PRS76 Selection of Health-Related Quality of Life Measures for Food Allergy

Food allergy (FA) poses a substantial burden on patients and their families, with reported physical and emotional impacts that can significantly influence daily life. Multiple disease-specific patient-reported outcome measures (PROMs) have been developed to measure the impact of FA on health-related quality of life (HRQoL). This review aimed to identify suitable PROMs to enable the assessment of interventions on the HRQoL impacts of patients with FAs. A literature search was performed in Medline and Embase from Jan 2010-April 2020. Key words like “food allergy” and “HRQoL” identified PROMs developed or used in patients with FA. Psychometric properties including content validity, reliability, validity, responsiveness and interpretability, were evaluated in accordance with the FDA PRO Guidance (2009). A total of 1028 abstracts were identified and 241 were selected for full-text review resulting in a list of 46 PROMs. They were classified as generic (n=22), allergy specific (n=2), FA-specific (n=19), and treatment satisfaction-specific (n=3). From the FA-specific PROMs, only 7 were conceptually relevant for further evaluation— Food Allergy Quality of Life Questionnaire (FAQLQ), Food Allergy Independent Measure (FAIM), Food-Pet-Allergy in Children Questionnaire (PFAC), Food Allergy Quality of Life Assessment Tool for Adolescents (FAQL-teen), You and Your Food Allergy, Food Intolerance Quality of Life Questionnaire (FIQLQ) and Pediatric Food Allergy Quality of Life Questionnaire (PFA-QL). You and Your Food Allergy, FAQLQ, PFAC, FAQL-teen involved patient’s qualitative inputs to establish content validity. All measures showed internal consistency (reliability) to be above 0.70 threshold; in terms of validity, PROMs moderately (0.30-0.70) correlated with existing measures. Only the FAQLQs showed both responsiveness to change and interpretability, key attributes for use in clinical trials. The FAQLQs validated for measuring HRQoL in FA have relevant conceptual coverage and adequate psychometric strength. Therefore, FAQLQs are suitable for further exploration of the impact of interventions within FA clinical trials.

Prospective Analysis of an Evidence-Based Symptom Set in Superior Canal Dehiscence Syndrome.

Superior canal dehiscence syndrome (SCDS) is characterized by a defect in the bone overlying the superior semicircular canal. This third mobile window generates an abnormal low-impedance pathway for sound/pressure transmission into the inner ear resulting in the characteristic symptoms. To perform a prospective analysis of symptoms in patients with SCDS. An aggregated symptom set was studied prospectively and compared with a healthy control group. The 31 items inquired about the presence and severity of symptoms. Initial comparison between both groups was performed using the Kolmogorov-Smirnov test and nonparametric Mann-Whitney U test. For analysis of the internal consistency, the Cronbach's alpha coefficient was determined. Finally, the prevalence of symptoms obtained in the present study was compared to the prevalence of symptoms obtained as part of a recent retrospective systematic review. Responses from the 29 patients with SCDS in the case group and the 58 healthy controls were not normally distributed. Those with SCDS had a higher prevalence and more severe symptoms for almost all items. The Cronbach's alpha coefficient of 0.969 indicates an excellent internal consistency. The prospective prevalence of most symptoms was higher than the retrospective prevalence reported earlier. SCDS causes a variety of vestibular and auditory symptoms. This prospective study demonstrates that their prevalence and severity are higher than reported previously. This symptom set demonstrates excellent internal consistency and will serve as a foundation toward developing a disease-specific patient-reported outcome measure for SCDS. NA.

Choosing the right survey-patient reported outcomes in esophageal surgery.

Patient reported outcomes (PROs) fulfill a crucial and unique niche in patient management, providing health-care providers a glimpse into their patients’ health experience. This is of utmost importance in patients with benign and malignant disorders of esophagus requiring surgery, which carries significant morbidity, in part due to a high burden of symptoms affecting health-related quality of life (HRQOL). There are a variety of generic and disease-specific patient reported outcome measures (PROMs) available for use in esophageal surgery. This article provides a broad overview of commonly used HRQOL instruments in esophageal surgery, including their utility in comparative effectiveness research, prognostication and shared decision-making for patients undergoing surgery for benign and malignant disorders of the esophagus.