Disease-specific Questionnaire Research Articles

Scoping review of exposure questionnaires and surveys in interstitial lung disease

BackgroundMany interstitial lung diseases (ILDs) have clear causal relationships with environmental and occupational exposures. Exposure identification can assist with diagnosis, understanding disease pathogenesis, prognostication and prevention of disease progression and...

Measuring Physical Functioning Using Wearable Sensors in Parkinson Disease and Chronic Obstructive Pulmonary Disease (the Accuracy of Digital Assessment of Performance Trial Study): Protocol for a Prospective Observational Study.

Physical capacity and physical activity are important aspects of physical functioning and quality of life in people with a chronic disease such as Parkinson disease (PD) or chronic obstructive pulmonary disease (COPD). Both physical capacity and physical activity are currently measured in the clinic using standardized questionnaires and tests, such as the 6-minute walk test (6MWT) and the Timed Up and Go test (TUG). However, relying only on in-clinic tests is suboptimal since they offer limited information on how a person functions in daily life and how functioning fluctuates throughout the day. Wearable sensor technology may offer a solution that enables us to better understand true physical functioning in daily life. We aim to study whether device-assisted versions of 6MWT and TUG, such that the tests can be performed independently at home using a smartwatch, is a valid and reliable way to measure the performance compared to a supervised, in-clinic test. This is a decentralized, prospective, observational study including 100 people with PD and 100 with COPD. The inclusion criteria are broad: age ≥18 years, able to walk independently, and no co-occurrence of PD and COPD. Participants are followed for 15 weeks with 4 in-clinic visits, once every 5 weeks. Outcomes include several walking tests, cognitive tests, and disease-specific questionnaires accompanied by data collection using wearable devices (the Verily Study Watch and Modus StepWatch). Additionally, during the last 10 weeks of this study, participants will follow an aerobic exercise training program aiming to increase physical capacity, creating the opportunity to study the responsiveness of the remote 6MWT. In total, 89 people with PD and 65 people with COPD were included in this study. Data analysis will start in April 2024. The results of this study will provide information on the measurement properties of the device-assisted 6MWT and TUG in the clinic and at home. When reliable and valid, this can contribute to a better understanding of a person's physical capacity in real life, which makes it possible to personalize treatment options. ClinicalTrials.gov NCT05756075; https://clinicaltrials.gov/study/NCT05756075. DERR1-10.2196/55452.

“Quality of Life in Epidermolysis Bullosa” and “Epidermolysis Bullosa Burden of Disease”: Italian translation, cultural adaptation, and pilot testing of two disease-specific questionnaires

BackgroundInherited epidermolysis bullosa (EB) is a clinically and genetically heterogeneous group of skin fragility disorders characterized by blister formation following minor trauma. Four major types are distinguished based on the level of cleavage within the skin. Most EB forms present severely disabling cutaneous and systemic signs and symptoms. Management relies on daily time-consuming and distressing topical medications, and symptomatic treatment of systemic findings. Disease manifestations, symptoms, and daily care strongly affect patient and caregiver quality of life (QoL). To date, there are two validated EB-specific questionnaires, the “Quality of Life in Epidermolysis Bullosa” (QOLEB) and the “Epidermolysis Bullosa Burden of Disease” (EB-BoD) for the evaluation of patient and family disease burden, respectively. The aim of our study was to develop an Italian translation of the two questionnaires and to pilot-test them.MethodsThe guidelines for translation and cross-cultural adaptation of health-related QoL measures were followed. Initially, two separate translations were generated for each questionnaire, and subsequently reconciled by an expert committee. This was followed by a back-translation process. The original texts and all translations underwent revision by the expert committee, resulting in definitive versions. The final versions were then tested in a pilot study involving cognitive debriefing in a group of 17 families, representative of all EB major types.ResultsThe translation and reconciliation process led to minor changes to obtain semantic/idiomatic/cultural equivalence of the Italian versions with the original ones and to reconcile the questions with the answer options. The cognitive debriefing process showed a good understanding and did not require text modifications.ConclusionsThe Italian versions of the QOLEB and EB-BoD provide valuable tools in everyday clinical practice of reference centers, and they allow the participation in multicenter international real-life observational studies as well as in controlled clinical trials. They enable the identification of disease-specific psychological and socioeconomic challenges for EB patients and their families, guiding targeted interventions to ensure appropriate and timely care.

Impact of Air Pollution and Allergic Status on Health-Related Quality of Life among University Students in Northern Thailand.

Global awareness of ambient air pollution has heightened due to its detrimental impact on health, particularly in regions with elevated PM2.5 levels. Chiang Mai has emerged as an area experiencing the highest PM2.5 levels in Thailand. to examine the prevalence of respiratory allergies and assess the impact of air pollution on the health-related quality of life (QoL) among university students in Chiang Mai. Chiang Mai University (CMU) and Maejo University (MJU) students were recruited. The Global Asthma Network (GAN) questionnaire screened for respiratory allergies (RAs). The disease-specific QoL questionnaire (Rcq-36) was administered twice during low-PM2.5 and high-PM2.5 seasons to evaluate air pollution's impact on health-related QoL. Those showing potential RAs underwent a skin prick test (SPT) to investigate allergic sensitization. Out of 406 participants, 131 (32%) reported respiratory allergies. Among those undergoing SPT, a high rate (82.54%) had positive results. Across both universities, students reported significantly lower QoL in multiple domains, particularly respiratory, eye, sleep, and emotional well-being, during the high-PM2.5 season. This aligned with their poorer self-reported health on a visual analog scale (VAS; p-value < 0.01). PM2.5 levels significantly impacted social functioning for CMU students (p-value = 0.001) and role limitations for MJU students (p-value < 0.001). Notably, participants without respiratory allergies (non-RAs) were more significantly affected by PM2.5 than RA participants in almost all parameters, despite experiencing fewer baseline symptoms. Respiratory allergies, particularly allergic rhinitis, are prevalent among university students in Chiang Mai. This study underscores the substantial negative impact of ambient air pollution on QoL for both allergic and non-allergic students.

Validation of the Italian version of a patient-reported outcome measure for Hereditary Spastic Paraplegia.

Patient-reported outcome measures (PROMs) are recognized as valuable measures in the clinical setting. In 2018 we developed the Italian version of the "Hereditary Spastic Paraplegia-Self Notion and Perception Questionnaire" (HSP-SNAP), a disease-specific questionnaire that collects personal perception on motor symptoms related to HSP such as stiffness, weakness, imbalance, reduced endurance, fatigue and pain. In this study our primary aim was to assess the questionnaire validity and reliability. Our secondary aim was to characterize the symptoms "perceived" by patients with HSP and compare them with those "perceived" by age-matched healthy subjects. The 12-item HSP-SNAP questionnaire was submitted to 20 external judges for comprehensibility and to 15 external judges for content validity assessment. We recruited 40 subjects with HSP and asked them to fill the questionnaire twice for test-retest procedure. They also completed the Medical Outcome Survey Short Form (SF-36) and were evaluated by the Spastic Paraplegia Rating Scale and the Six-Minute Walk Test. We also recruited 44 healthy subjects who completed the HSP-SNAP once to test score variability. The HSP-SNAP content validity index was high (0.8±0.1) and the test-retest analysis showed high reliability (ICC = 0.94). The mean HSP-SNAP score (score range 0-48) of the HSP group was 22.2±7.8, which was significantly lower than healthy subjects (43.1±6.3). The most commonly perceived symptom was stiffness, followed by weakness and imbalance. Although HSP-SNAP does not investigate non-motor symptoms and we validated only its Italian version, it showed good validity and reliability and it could be used in combination with other objective outcome measures for clinical purposes or as endpoints for future clinical rehabilitation studies. Trial Registration: ClinicalTrial.gov, NCT04256681. Registered 3 February 2020.

Skin Biopsy Detection of Phosphorylated α-Synuclein in Patients With Synucleinopathies

Finding a reliable diagnostic biomarker for the disorders collectively known as synucleinopathies (Parkinson disease [PD], dementia with Lewy bodies [DLB], multiple system atrophy [MSA], and pure autonomic failure [PAF]) is an urgent unmet need. Immunohistochemical detection of cutaneous phosphorylated α-synuclein may be a sensitive and specific clinical test for the diagnosis of synucleinopathies. To evaluate the positivity rate of cutaneous α-synuclein deposition in patients with PD, DLB, MSA, and PAF. This blinded, 30-site, cross-sectional study of academic and community-based neurology practices conducted from February 2021 through March 2023 included patients aged 40 to 99 years with a clinical diagnosis of PD, DLB, MSA, or PAF based on clinical consensus criteria and confirmed by an expert review panel and control participants aged 40 to 99 years with no history of examination findings or symptoms suggestive of a synucleinopathy or neurodegenerative disease. All participants completed detailed neurologic examinations and disease-specific questionnaires and underwent skin biopsy for detection of phosphorylated α-synuclein. An expert review panel blinded to pathologic data determined the final participant diagnosis. Skin biopsy for detection of phosphorylated α-synuclein. Rates of detection of cutaneous α-synuclein in patients with PD, MSA, DLB, and PAF and controls without synucleinopathy. Of 428 enrolled participants, 343 were included in the primary analysis (mean [SD] age, 69.5 [9.1] years; 175 [51.0%] male); 223 met the consensus criteria for a synucleinopathy and 120 met criteria as controls after expert panel review. The proportions of individuals with cutaneous phosphorylated α-synuclein detected by skin biopsy were 92.7% (89 of 96) with PD, 98.2% (54 of 55) with MSA, 96.0% (48 of 50) with DLB, and 100% (22 of 22) with PAF; 3.3% (4 of 120) of controls had cutaneous phosphorylated α-synuclein detected. In this cross-sectional study, a high proportion of individuals meeting clinical consensus criteria for PD, DLB, MSA, and PAF had phosphorylated α-synuclein detected by skin biopsy. Further research is needed in unselected clinical populations to externally validate the findings and fully characterize the potential role of skin biopsy detection of phosphorylated α-synuclein in clinical care.

Confounders Effecting Quality of Life of COPD Patients in European Union: A Systematic Review

Introduction: Chronic Obstructive Pulmonary Disease (COPD) is a respiratory disease characterized by restriction in expiratory airflow. It is among one of the major causes of illness and death globally. COPD causes severe negative effects on physical and mental health. It has a drastic effect upon the health-related quality of life of patients. Numerous factors contribute towards morbidity of COPD that include smoking, obesity, air pollutants and comorbid conditions (cardiovascular diseases, diabetes mellitus, arterial hypertension, infections). Aims and Objectives: The current systematic review was conducted with the aim to determine the health-related quality of life of COPD patients as well as the confounders of quality of life. Methodology: A systematic review was conducted of 22 studies. The electronic databases used to search the articles were Pubmed, Scopus, Science Direct, ProQuest, Web of Science. Total 1880 studies were found, out of which only 22 studies met the inclusion criteria. All the studies included are within 2017-2022. Patients met inclusion criteria have less than 70% FEV1/FVC ratio and diagnosis of patients was according to the GOLD staging system. Majority study design were observational, some were cross sectional, experimental and randomized clinical trials. Mean age of patients observed was 40-85 years. To assess the quality of life of COPD patient specific questionnaires has been used like SGRQ, CAT, CCQ, EQ-5D-5L. Results: Included studies shows that the quality of life of COPD is compromised physically as well as mentally. It is due to less physical activities, smoking habits, psychological issues, comorbidities like diabetes mellitus, cardiovascular diseases, arterial hypertension. Conclusion: All the studies concluded that COPD patients diagnosed on basis of GOLD criteria and their quality of life is assessed via disease specific questionnaires that majorly includes SGRQ, CAT, CCQ, EQ-5D-5L. And it shows that quality of life of COPD decreases overall. However, the major confounders of reduced quality of life in COPD patients were: Smoking, raised BMI, high cholesterol, Depression/ anxiety, traffic related air pollutants, malnutrition and co-morbid conditions such as hypertension, asthma, anemia and diabetes mellitus.

Evaluation of disease-specific quality of life and its influencing factors in Bulgarian patients with Graves' orbitopathy.

Graves' orbitopathy (GO) profoundly affects patients' quality of life (QoL). Our aim was to assess QoL in patients with different forms of GO and to search for predictors of QoL. This was a cross-sectional study involving 221 consecutive unselected GO patients (77.4% females, mean age 52 ± 11.6) referred to our clinic in the period 2017-2021. Detailed medical history was obtained from all patients, followed by hormonal and immunological testing and comprehensive ocular status. QoL was assessed by a disease-specific questionnaire (GO-QoL), consisting of two scales-one related to visual functioning (QoL-VF) and the other-to physical appearance (QoL-AP). The results were calculated by formula and expressed as percentages. Mild GO patients had significantly higher scores on both scales of GO-QoL compared to moderate-to-severe and sight-threatening GO patients (82.0 vs. 54.6% and 27.3% for QoL-VF; 70.1 vs. 54.4% and 36.9% for QoL-AP). Patients with active GO had significantly poorer QoL-VF (56.6 vs. 76.6%) and QoL-AP (53.1 vs. 67.5%) in comparison to patients with inactive GO. The stepwise linear regression analysis showed that the variables with major predictive value for QoL-VF were: CAS, diplopia score, visual acuity and severity of the symptoms (R2 = 0.44), whereas gender, CAS, diplopia score and proptosis best predicted QoL-AP (R2 = 0.39). The impact of GO on patients' QoL depends on the severity and activity of the disease. The clinical predictors of the impairment of QoL should be taken into account when considering the optimal adjunctive treatment approaches aiming to improve patients' QoL.

Knowledge gaps in health-related quality of life research performed in children with bleeding disorders - A scoping review.

Bleeding disorders (BDs) may influence health-related quality of life (HRQoL) in children and caregivers. Measuring HRQoL gives insight into domains requiring support and provides an opportunity to evaluate the effects of novel therapies. To gain insight in the current body of literature on HRQoL in children with BDs in order to identify knowledge gaps for research and further development of this field. Scoping review. We included 53 articles, describing studies mainly performed in Europe and North-America (60.4%) and mostly within the last ten years. Only 32% studies included children<4 years. Almost all studies (47/53, 88.7%) were performed in boys with haemophilia, pooling haemophilia A and B (n=21) and different disease severities (n=20). Thirteen different generic and five disease-specific HRQoL-questionnaires were applied; all questionnaires were validated for haemophilia specifically. Six (11,3%) combined generic and disease-specific questionnaires. Self-reports were most frequently applied (40/53, 75.5%), sometimes combined with proxy and/or parent-reports (17/53, 32.1%). Eleven studies used a reference group (20.8%). Statistical analyses mostly consisted of mean and SD (77.4%). HRQoL-research is mainly performed in school-aged boys with haemophilia, treated in developed countries. Pitfalls encountered are the pooling of various BDs, subtypes and severities, as well as the application of multiple generic questionnaires prohibiting comparison of results. More attention is needed for broader study populations including other BDs, young children, feminine bleeding issues and platelet disorders, as well as the use of HRQoL as an effect-measurement tool for medical interventions, and more thorough statistical analysis.

Diagnostic intervention improved health-related quality of life among teenagers with food allergy.

The aim was to examine if a diagnostic intervention set up to assess current food allergy to cow's milk, hen's egg, fish, or wheat among teenagers had an impact on generic and disease specific health-related quality of life (HRQL). The study compared HRQL scoring before and two years after the intervention, and in relation to age matched controls without reported food allergy. The study was performed within the Obstructive Lung Disease in Northern Sweden (OLIN) studies where a cohort study on asthma and allergic diseases among 8-year-old schoolchildren was initiated in 2006. At age 12 years, the 125/2612 (5%) children who reported allergy to cow's milk, hen's egg, fish, or wheat were invited to a diagnostic intervention including clinical examination, blood tests and evaluation by a pediatric allergist. Of 94 participants, 79 completed generic and disease specific HRQL questionnaires. Additionally, a random sample of 200 (62% of invited) children without food allergy from the OLIN cohort answered the generic HRQL questionnaire. The respondents of the HRQL questionnaires were re-examined two years later and 57 teenagers with and 154 without reported allergy participated. There were no significant differences in generic HRQL scores between teenagers with and without reported food allergy at study entry, or after the intervention. Among those with reported food allergy, we found a significant improvement in disease specific HRQL after the intervention (mean values: 3.41 vs 2.80, p<0.001). Teenagers with only food allergy had better disease specific HRQL compared to those with one, two or three concomitant allergic diseases, both before and after the intervention. Children with only food allergy significantly improved their HRQL after the intervention, 1.84 vs. 2.87 (p<0.001) but this association was not seen in children with one other allergic disorder (3.16 vs. 3.65, p = 0.121) or those with two or more allergic disorders (3.72 vs. 3.90, p = 0.148). The diagnostic intervention showed a long-term improvement of disease specific HRQL but not generic HRQL.

Home mechancial ventilation: quality of life and the final stage of life

The evaluation of health-related quality of life (HRQL) has gained importance in recent years. Disease-specific questionnaires are available for respiratory insufficiency, which enable the assessment of HRQL. The Severe Respiratory Insufficiency (SRI) questionnaire, which was specially developed for patients with respiratory insufficiency, is ideal for quantifying the quality of life in patients receiving home mechanical ventilation (HMV). Studies using the SRI questionnaire demonstrated that the physical functioning of patients with chronic lung diseases of various etiologies is significantly impaired, but frequently without significant impairment of psychological well-being. Therefore, severity of the disease and HRQL do not necessarily correlate with each other.Both invasive and non-invasive mechanical ventilation can improve quality of life. Co-morbidity, and above all advanced age, have the highest negative predictive value with regard to in-hospital mortality and weaning failure. The number of patients older than 80 years who remain dependent on invasive HMV after prolonged weaning in Germany is increasing significantly. High dependence on invasive HMV is often associated with a loss of quality of life and autonomy. Thus, ethical issues of the continued treatment of ventilated patients at the end of life are discussed increasingly.After weaning failure, the indication for invasive HMV should be critically examined and should focus on potentially severely reduced quality of life and poor prognosis, as well as the patient's wishes. If previously agreed treatment goals can no longer be achieved during the course of invasive HMV, changing the therapy goals should be discussed within the clinical team, with the patient, their relatives and, if necessary, with legal representatives. A case involving all those involved in providing care is sometimes necessary.In order to avoid overtreatment and unnecessary patient suffering during invasive HMV, advanced care planning should be started as early as possible and if necessary accompanied by palliative medical measures.

Cross-Cultural Adaptation and Measurement of Psychometric Properties of the Lithuanian Version of the Western Ontario Shoulder Instability Index (WOSI).

Background and Objectives: The Western Ontario Shoulder Instability Index (WOSI) is a disease-specific self-administered questionnaire which is designed to measure health-related quality of life for patients with shoulder instability. The objective of this study was to translate and adapt the WOSI questionnaire for the Lithuanian-speaking population and investigate the psychometric properties of the Lithuanian version of the WOSI questionnaire (WOSI-LT): validity, reliability, and responsiveness. Materials and Methods: The WOSI scale was translated into Lithuanian using D. E. Beaton's systematic and standardized guidelines for cross-cultural adaptation of patient-administered scales. Subsequently, the psychometric properties of the Lithuanian version of the scale (WOSI-LT) were investigated. The study involved 40 patients who reported shoulder instability and underwent surgical treatment. All patients completed the WOSI-LT, QuickDASH, and SF-12 scales. A subset of 10 patients was selected for the reproducibility and responsiveness evaluation. Based on the obtained data, the reliability, validity, and responsiveness of WOSI-LT were examined using statistical analysis methods. Results: The Lithuanian adaptation of the WOSI questionnaire exhibited a high degree of internal consistency, evidenced by a Cronbach's alpha of 0.93. Its reproducibility was commendable with an intraclass correlation coefficient (ICC) value of 0.90. When assessing correlations, WOSI-LT demonstrated a stronger relationship with QuickDASH (r = 0.64) than with SF-12 (physical component score (PCS) 0.61, mental component score (MCS) 0.33). Six months post-operation, the responsiveness of the WOSI-LT was particularly notable, with a standardized response mean (SRM) of 0.91, the highest among the three scales. Furthermore, no floor or ceiling effects were identified in the scores of the Lithuanian WOSI. Conclusions: WOSI-LT is a valid, reliable, and responsive questionnaire that correlates excellently with the original English version of the scale. This scale can be used in Lithuanian medical institutions to assess the severity of patients' shoulder instability and evaluate their progress during treatment.

Reliability, Validity, Modification and Expansion of the Chinese Version of the Disease-Specific Anxiety Questionnaire for Chronic Obstructive Pulmonary Disease.

To translate a disease-specific anxiety questionnaire on chronic obstructive pulmonary disease (COPD) and test its reliability and validity in China. The German version of the revised COPD Anxiety Questionnaire (CAF-R) was initially validated using step-by-step translation, back-translation, and cross-cultural adaptation. The reliability and validity of the Chinese version of the CAF-R (CAF-R-CN) were tested among 448 patients with COPD (mean age =71.42±9.33 years, 17.2% female) from four medical institutions in Suzhou, Jiangsu Province, using convenience sampling, from April 2022 to June 2023. The CAF-R-CN included six dimensions with a total of 25 items. The item-level content validity index was 0.860-1.000; the scale-level content validity index was 0.920. The structural validity χ2/df was 2.326, the root mean square error of approximation was 0.077, the comparative fit index was 0.924, and the Tucker-Lewis index was 0.912. The six-dimensional internal consistency index Cronbach's α coefficient was 0.696-0.910, and the test-retest reliability was 0.949. An optimal cut-off score of 50.5 was selected with a sensitivity of 0.786 and specificity of 0.870. The CAF-R-CN had satisfactory reliability and validity and can be used to identify and assess anxiety in COPD patients with a Chinese cultural background.

Pre-treatment expectations and their influence on subjective symptom change in Crohn's disease

BackgroundTreatment expectations reportedly shape treatment outcomes, but have not been studied in the context of multimodal therapy in Crohn's disease (CD). Therefore, the current study investigated the role of treatment expectations for subjective symptom changes in CD patients who have undergone an integrative multimodal therapy program. MethodsValidated questionnaires were completed at the start of the treatment program and post intervention. Pre-treatment expectations and experienced symptom change were assessed with the Generic Rating Scale for Previous Treatment Experiences, Treatment Expectations, and Treatment Effects (GEEE); stress levels were quantified with the Perceived Stress Scale (PSS-10) and disease specific quality of life was quantified with the disease-specific Inflammatory Bowel Disease Questionnaire (IBDQ). We performed multiple linear and Bayesian regression to determine how expectations related to symptom change. ResultsN = 71 CD patients (66.2% female) were included. Stronger expectations regarding symptom improvement (b = 0.422, t = 3.70, p < .001) were associated with higher experienced symptom improvement. Additionally, Bayesian analysis provided strong evidence for including improvement expectations as a predictor of improvement experience (BFinclusion = 13.78). ConclusionsIn line with research in other disorders, we found that positive treatment expectations were associated with experienced symptom improvement. In contrast, we found no indication that an experience of symptom worsening was associated with positive or negative baseline treatment expectations. Induction of positive expectations might be a potential avenue for improving treatment outcomes in CD therapy.

Evaluation of the health-related quality of life in dogs following intracranial meningioma resection using a specifically developed questionnaire.

As advanced treatments are becoming increasingly feasible in veterinary medicine, the evaluation of the health-related quality of life (HRQOL) of treated animals is becoming more relevant. We evaluated owner-perceived HRQOL of 10 dogs that underwent craniotomy for meningioma resection between 2002 and 2022 at our institution through telephone interview. For this purpose, we developed a disease-specific questionnaire containing 52 items (mostly of scoring nature) patterned after previously validated instruments and organised into eight domains. Approval by the Human Ethical Committee and respondents' consent were obtained. We analysed the scores for all domains and dogs. The effect of different variables on the HRQOL score was determined via log-rank test and Pearson correlation. Scores for all included dogs (range, 0-235 points) were totaled, with a higher number of points indicating a better HRQOL. The dogs included in this study yielded a mean score of 200.6 points (range, 176-227 points), implying a good overall quality of life. There were no significant associations between individual parameters and outcomes. Our questionnaire represents a structured tool for the specific evaluation of postoperative HRQOL in dogs with meningioma, placing a minimal burden on respondents. Few instruments have been developed to assess animal welfare in a disease-specific context. Implementing these tools, however, is essential to accurately evaluate not only the impact of treatments on biologic parameters, but also their implications on patient welfare. Thus, treatment plans may consider HRQOL for a more comprehensive clinical decision-making process.

The Necessity of Interoperability to Uncover the Full Potential of Digital Health Devices.

Personalized health care can be optimized by including patient-reported outcomes. Standardized and disease-specific questionnaires have been developed and are routinely used. These patient-reported outcome questionnaires can be simple paper forms given to the patient to fill out with a pen or embedded in digital devices. Regardless of the format used, they provide a snapshot of the patient's feelings and indicate when therapies need to be adjusted. The advantage of digitizing these questionnaires is that they can be automatically analyzed, and patients can be monitored independently of doctor visits. Although the questions of most clinical patient-reported outcome questionnaires follow defined standards and are evaluated by clinical trials, these standards do not exist for data processing. Interoperable data formats and structures would benefit multilingual and cross-study data exchange. Linking questionnaires to standardized terminologies such as the Systematized Nomenclature of Medicine Clinical Terms (SNOMED CT) and Logical Observation Identifiers, Names, and Codes (LOINC) would improve this interoperability. However, linking clinically validated patient-reported outcome questionnaires to clinical terms available in SNOMED CT or LOINC is not as straightforward as it sounds. Here, we report our approach to link patient-reported outcomes from health applications to SNOMED CT or LOINC codes. We highlight current difficulties in this process and outline ways to minimize them.

Can a generic patient reported outcome measure substitute a condition specific measure at assessing care effectiveness in low back pain?

Quality of care assessments beside the provider level should also include the patient perspective, however, there aren't widespread solutions for this approach. One possibility is to apply a generic, thus, widely applicable Patient Reported Outcomes Measure (PROM) to assess care outcomes. Taking this notion into consideration, this study aimed to investigate if a generic questionnaire can reliably substitute a disease-specific questionnaire when measuring care effectiveness with patient-reported outcome measures among patients with low back pain. Between January and December 2019, we conducted a before and after survey in three Hungarian hospitals. Adults with confirmed low back pain expecting spinal surgery were eligible to enter the study. SF-36 Health Survey (SF-36) and Roland‐Morris Disability Questionnaire (RMDQ) were used. Multivariate linear regression analyses were conducted to explore the relationship between the results of the two questionnaires and how service provider, sex, and education level could explain the differences in the calculated PROM-based performance measures. During the pre-intervention survey, 11 individuals decided to either not sign the consent form or complete the questionnaire. As a result, 116 individuals participated in the first – baseline – survey and 86 in the second – follow-up – survey. The drop-out rate varied by providers: the lowest rate was 15%, and the highest 39%. Of the SF-36 subscale-based performance measures, role limitations due to physical health and physical functioning significantly correlated with the RMDQ performance measure. Considering the necessary minimum clinically important difference, the explanatory analysis showed that the SF-36 physical functioning subscale-based and the RMDQ-based performance measures established the same performance rank-order among the participating hospitals. The physical functioning subscale of the SF-36 provided similar results to the RMDQ regarding care effectiveness. Thus, the SF-36 may be able to measure and compare care effectiveness among providers in low-back pain. If future studies investigating other health conditions come to the same conclusion, then the SF-36 could be used by itself to incorporate the patient perspective into health care quality assessments, thereby increasing comparability and lowering administrative costs.

Cross-walk of the Chronic Liver Disease Questionnaire for Nonalcoholic Steatohepatitis (CLDQ-NASH) and the EuroQol EQ-5D-5L in patients with NASH

BackgroundNonalcoholic steatohepatitis (NASH) is a chronic progression of nonalcoholic fatty liver disease, which can negatively impact the health-related quality of life (HRQoL) of affected individuals. HRQoL in NASH has been assessed using the disease-specific Chronic Liver Disease Questionnaire for NASH (CLDQ-NASH) and the generic EuroQol EQ-5D-5L. As the performance of these instruments relative to each other is unknown, we performed a cross-walk analysis of CLDQ-NASH to EQ-5D-5L using data from a real-world NASH population.MethodsData were drawn from the Adelphi Real World 2019 NASH Disease Specific Programme, a cross-sectional survey of physicians and their patients in the United States. Patients with physician-diagnosed NASH completed a questionnaire that included the CLDQ-NASH and EQ-5D-5L. Mapping from CLDQ-NASH to EQ-5D-5L was done using tenfold cross-validation; performance was assessed using root-mean squared error as accuracy measure. Subgroup analyses compared performance of the models in obese versus non-obese patients and patients with versus without type 2 diabetes (T2D).ResultsData from 347 patients were included in this analysis. Overall, 2172 models were tested for predicting EQ-5D-5L index score from CLDQ-NASH score. The best model for this mapping was a generalized linear model using Gaussian distribution and a power link. The best model for mapping from CLDQ-NASH domains to the EQ-5D-5L was a fractional logistic model. Models performed better at predicting upper versus lower values of EQ-5D-5L, for non-obese versus obese patients, and for patients without versus with T2D.ConclusionWe describe a scoring algorithm for cross-walking the CLDQ-NASH to the EQ-5D-5L enabling health status comparisons of HRQoL across studies.

QoL, CIs, QALYs, and Individualized Rehabilitation: The Clinical and Practical Benefits of Regularly Assessing the Quality of Life of Adult Cochlear Implant Recipients.

This study aimed to report quality of life (QoL) scores in unilateral cochlear implant (CI) users and to generate guidance for clinicians on using QoL measures to individualize CI counselling and rehabilitation and to increase access to CIs as a mode of rehabilitation. Participants (n = 101) were unilateral CI users with single-sided deafness (SSD; n = 17), asymmetrical hearing loss (AHL; n = 26), or bilateral hearing loss (Uni; n = 58). Generic QoL was assessed via the Health Utilities Index (HUI-3), and disease-specific QoL was assessed via the Speech, Spatial, and Qualities of Hearing scale (SSQ12) and Nijmegen CI Questionnaire (NCIQ) at preimplantation and at 6 and 12 months of CI use. All groups had significantly increased HUI-3 scores at both intervals. The SSD group showed significant benefit on the SSQ12 at visit 3, the AHL group showed significant benefit on the SSQ12 and most NCIQ subdomains at both intervals, and the Uni group showed significant benefit with both tests at both intervals. Unilateral CI recipients demonstrate improved QoL within the first 12 months of device use. Regular assessment with generic and disease-specific questionnaires has the potential to play an important role in personalizing treatment and possibly in increasing access to CI provision.

An Exploration of Quality of Life Perceptions of Cochlear Implant Recipients and Their Frequent Communication Partners

Purpose: Quality of life (QoL) after cochlear implantation has been investigated using a variety of QoL questionnaires. However, few studies have examined the perceived QoL of cochlear implant (CI) recipients compared between the recipient and a frequent communication partner (FCP) using both hearing-related (hearing-specific) and health-general (health-general) measures. Method: For this multiple case study, five participant pairs were recruited, consisting of five adult recipients who were implanted at least 6 months prior to the time of evaluation and five adult FCPs who lived with the recipient at the time of evaluation. All 10 participants (five pairs) completed a health-general questionnaire (Medical Outcome Study Short-Form Survey 12-Item [SF-12]), and eight participants (four pairs) completed a disease specific questionnaire (Nijmegen Cochlear Implant Questionnaire [NCIQ]). Results: Both recipients and partners tended to score similarly to each other on both measures. However, both groups scored higher on the SF-12 than the NCIQ. Results also revealed a better interrater agreement between pairs on the SF-12 compared to the NCIQ. Conclusions: Overall, scores for recipients and partners were higher for the health-general SF-12 compared to the hearing-related NCIQ. Additionally, rater agreement was better for the SF-12 compared to the NCIQ, suggesting that differences in QoL perceptions exist for more hearing-specific challenges. These results highlight potential differences in perception of recipient QoL postimplantation as illustrated through the use of multiple QoL measures. Results speak to the importance of open communication between recipients and partners.