Małgorzata Farnik

Instrument development and evaluation for patient-related outcomes assessments

Patient-related outcomes measures could provide important information for the current state of the art in medical care and even have an impact on macrodecisions in the health care system. Patient-related outcomes were initially defined as subjective health indicators that allow disability and illness to be assessed, based on patient, caregiver, or physician self-reports. As illness involves psychological and behavioral complex processes of care, a multidisciplinary approach in measuring patient-reported outcomes should be recommended, such as quality of life questionnaires. Patient-related outcomes measures should correspond to specific clinical situations and bring opportunities to improve quality of care. Objective measurements enable quantitative data to be collected and analyzed. Depending on the aim of the research, investigators can use existing methods or develop new tools. This publication presents a methodology for developing patient-related outcomes measures, based on a multistage procedure. The proper definition of specific study objectives and the methodology of instrument development are crucial for successfully transferring the study concept. The model of instrument development is the process of starting from the preliminary phase and includes questionnaire design and scaling, pilot testing (cognitive debriefing), revision of the preliminary version, evaluation of the new tool, and implementation. Validation of the new instrument includes reliability, reproducibility, internal consistency, and responsiveness. The process of designing the new tool should involve a panel of experts, including clinicians, psychologists (preliminary phase), and statisticians (scale development and scoring), and patients (cognitive debriefing). Implementation of a new tool should be followed by evaluation study – assessment of the tool’s usefulness in clinical practice. An instrument must show not only the expected methodological properties and performance but also a positive contribution to care. The necessity of implementation of direct patient-reporting methods has been highlighted by both the Food and Drug Administration and the European Medicines Agency.

Quality of life protocol in the early asthma diagnosis in children

Asthma is considered an important problem in children and influences on their everyday functioning. This study was concerned with the impact of asthma on the health‐related quality of life (HRQOL) of newly recognized pediatric asthma patients and their parents.

Underdiagnosis of childhood asthma: A comparison of survey estimates to clinical evaluation

ObjectivesDiagnostic patterns play a role in asthma prevalence estimates and could have implications for disease management. We sought to determine the extent to which questionnaire-derived estimates of childhood asthma reflect the disease’s true occurrence.Materials and MethodsChildren aged 6–12 years from Katowice, Poland, were recruited from a cross-sectional survey (N = 1822) via primary schools. Students were categorized into three mutually exclusive groups based on survey responses: “Asthma” (previously diagnosed asthma); “Respiratory symptoms” (no previous diagnosis of asthma and one or more respiratory symptoms during last year), “No respiratory symptoms” (no previous diagnosis of asthma or respiratory symptoms). A sample of children from each group (total N = 456) completed clinical testing to determine asthma presence according to GINA recommendations.ResultsBased on the survey, 5.4% of children were classified with asthma, 27.9% with respiratory symptoms, and 66.7% with no respiratory symptoms or asthma. All previously known 41 cases of asthma were confirmed. New diagnoses of asthma were made in 21 (10.9%) and 8 (3.6%) of subjects from the “Respiratory symptoms” (N = 192) and “No respiratory symptoms” (N = 223) groups, respectively. The overall prevalence of childhood asthma, incorporating the results of clinical examination, was 10.8% (95% CI: 9.4–12.2), compared to the questionnaire-derived figure of 5.4% (95% CI: 4.4–6.5%) and affected females more than males.ConclusionsAsthma prevalence was underestimated in this population possibly resulting from under-presentation or under-diagnosis. This could have potential implications for proper management and well-being of children. Questionnaire estimates of prevalence should be considered carefully in the context of regional diagnostic patterns.

Distribution and characteristics of COPD phenotypes – results from the Polish sub-cohort of the POPE study

Background This study aimed to examine the distribution of predefined phenotypes, demographic data, clinical outcomes, and treatment of patients who were included in the Polish cohort of the Phenotypes of COPD in Central and Eastern Europe (POPE) study. Patients and methods This was a sub-analysis of the data from the Polish cohort of the POPE study, an international, multicenter, observational cross-sectional survey of COPD patients in Central and Eastern European countries. The study included patients aged >40 years, with a confirmed diagnosis of COPD, and absence of exacerbation for at least 4 weeks before study inclusion. A total of seven Polish centers participated in the study. Results Among the 430 Polish COPD patients enrolled in the study, 61.6% were non-exacerbators (NON-AE), 25.3% were frequent exacerbators with chronic bronchitis (AE CB), 7.9% were frequent exacerbators without chronic bronchitis (AE NON-CB), and 5.1% met the definition of asthma-COPD overlap syndrome (ACOS). There were statistically significant differences among these phenotypes in terms of symptom load, lung function, comorbidities, and treatment. Patients with the AE CB phenotype were most symptomatic with worse lung function, and more frequently reported anxiety and depression. Patients with the ACOS phenotype were significantly younger and were diagnosed with COPD earlier than those with other COPD phenotypes; those with the ACOS phenotype were also more often atopic and obese. Conclusion There is significant heterogeneity among COPD patients in the Polish population in terms of phenotype and clinical outcome. The non-exacerbator phenotype is observed most frequently in Poland, while the frequent exacerbator with chronic bronchitis phenotype is the most symptomatic.

Association of anxiety sensitivity-physical concerns and FVC with dyspnea severity in sarcoidosis

OBJECTIVE The purpose of the study was to evaluate the relationship of an objective functional lung parameter (FVC) and a subjective psychological factor (physical symptom concerns) with dyspnea in sarcoidosis. Dyspnea constitutes one of the most common and burdensome symptoms in sarcoidosis, yet little is known about its mechanisms and, in particular, psychological. METHOD A total of 107 hospitalized sarcoidosis patients (Female=50, Mage=45.3years) volunteered to take part in the correlational research study. Participants underwent spirometry and completed the MRC Dyspnea Scale and the Anxiety Sensitivity Index-3 (ASI) questionnaire. Linear hierarchical regression analysis was used to determine the relationship between the studied predictors and dyspnea severity. RESULTS The best fitting model predicted 18% of variance in dyspnea severity. Physical symptom concerns subscale of ASI (β=0.24) and FVC (β=-0.23) were significantly related to dyspnea MRC severity, but only physical concerns remained significantly related to dyspnea when both predictors were in the model. CONCLUSIONS The current results suggest that both psychological and physiological factors should be taken into account when explaining subjective dyspnea severity in sarcoidosis. More specifically, these findings call for including cognitive vulnerability factors related to anxiety (physical symptom concerns) into the diagnostic procedures and management of dyspnea in sarcoidosis.